BioVie Inc Aktienkurs
Ist BioVie Inc eine Topscorer-Aktie nach der Dividenden-, High-Growth-Investing- oder Levermann-Strategie?
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📘 Marktkapitalisierung
📈 Was ist das?
Die Marktkapitalisierung zeigt, wie viel ein Unternehmen laut Börse aktuell wert ist.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Sie hilft Unternehmen in Größenklassen (Large, Mid, Small Cap) einzuordnen und gibt Hinweise auf Marktmacht und Stabilität.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Große Unternehmen gelten als stabiler, zahlen oft Dividenden, wachsen aber langsamer.
- Kleine Firmen können stärker wachsen, sind aber schwankungsanfälliger.
- Die Marktkapitalisierung ist ein guter Indikator für Unternehmensgröße, aber kein Maß für Unter- oder Überbewertung.
📘 Enterprise Value (Unternehmenswert)
📈 Was ist das?
Der Enterprise Value (EV) zeigt, was ein Unternehmen tatsächlich kostet, wenn man es komplett übernehmen würde – inklusive Schulden und abzüglich Cash.
🧮 Wie wird es berechnet?
(= Marktkapitalisierung + Nettoverschuldung)
🏛️ Wofür ist es wichtig?
Der EV ist eine realistischere Bewertungsbasis als die Marktkapitalisierung, da er die Kapitalstruktur berücksichtigt. Er ist Grundlage für Kennzahlen wie EV/FCF oder EV/Sales.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Der Enterprise Value zeigt, was ein Unternehmen tatsächlich wert ist – unabhängig davon, wie es finanziert ist.
- Er ist besonders wichtig für professionelle Investoren, da er eine objektivere Grundlage für Bewertungsvergleiche bietet als die Marktkapitalisierung allein.
- Ein Unternehmen mit hoher Verschuldung erscheint im EV teurer, eines mit viel Cash günstiger – auch wenn sie an der Börse gleich viel wert sind.
📘 Nettoverschuldung
📈 Was ist das?
Die Nettoverschuldung zeigt, wie viele Schulden nach Abzug des verfügbaren Cashs tatsächlich verbleiben.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Sie zeigt, wie stark ein Unternehmen von Fremdkapital abhängig ist – und wie gut es in der Lage ist, seine Schulden kurzfristig zu bedienen.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Eine niedrige oder negative Nettoverschuldung bedeutet hohe finanzielle Stabilität.
- Unternehmen mit viel Cash und geringer Verschuldung sind besser gerüstet für Krisen.
- Eine hohe Nettoverschuldung erhöht das Risiko – besonders bei steigenden Zinsen oder konjunkturellen Schwächen.
📘 Cash
📈 Was ist das?
Der Cashbestand zeigt, wie viele liquide Mittel einem Unternehmen sofort zur Verfügung stehen.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Er gibt Auskunft über die finanzielle Flexibilität: Ein hoher Cashbestand ermöglicht Investitionen, Rückkäufe oder Krisenresistenz.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hoher Cashbestand zeigt finanzielle Stärke und Handlungsspielraum.
- Cash kann für Investitionen, Schuldentilgung oder Aktienrückkäufe genutzt werden.
- Allerdings: Zu viel ungenutztes Kapital kann auch auf mangelnde Investitionsideen hinweisen.
📘 Anzahl ausstehender Aktien
📈 Was ist das?
Die Anzahl ausstehender Aktien gibt an, wie viele Aktien eines Unternehmens aktuell im Umlauf sind und von Investoren gehalten werden.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Sie ist die Grundlage für viele Kennzahlen wie Gewinn je Aktie (EPS), Marktkapitalisierung oder KGV.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Je weniger Aktien im Umlauf sind, desto höher fällt z. B. der Gewinn je Aktie aus – wichtig für Bewertung und Dividendenrendite.
- Aktienrückkäufe verringern die Anzahl ausstehender Aktien – und steigern den Wert je Aktie.
- Kapitalerhöhungen haben den gegenteiligen Effekt: mehr Aktien → Verwässerung der bestehenden Anteile.
📘 Kurs-Gewinn-Verhältnis (KGV)
📈 Was ist das?
Das KGV zeigt, wie oft der Gewinn pro Aktie im aktuellen Aktienkurs enthalten ist – also wie „teuer“ eine Aktie im Verhältnis zum Gewinn ist.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Das KGV gehört zu den bekanntesten Bewertungskennzahlen. Es hilft Anlegern einzuschätzen, ob eine Aktie im Vergleich zu ihrem Gewinn eher günstig oder teuer erscheint.
🧮 Berechnung
📊 KGV (TTM) = bezogen auf den Gewinn der letzten 12 Monate (Trailing Twelve Months):🎯 Was bedeutet das für Anleger?
- Ein niedriges KGV kann auf eine günstige Bewertung hindeuten – oder auf Probleme im Geschäftsmodell.
- Ein hohes KGV kann Wachstumserwartungen widerspiegeln – oder eine überbewertete Aktie.
📘 Kurs-Umsatz-Verhältnis (KUV)
📈 Was ist das?
Das KUV zeigt, wie viel Anleger für 1 € Umsatz eines Unternehmens zahlen – unabhängig vom Gewinn.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Das KUV ist besonders bei wachstumsstarken oder noch nicht profitablen Unternehmen hilfreich. Es zeigt, wie hoch der Umsatz an der Börse bewertet wird.
🎯 Was bedeutet das für Anleger?
- Ein niedriges KUV kann auf Unterbewertung hindeuten – oder auf schwache Margen.
- Ein hohes KUV kann hohe Erwartungen widerspiegeln – oder übermäßigen Optimismus.
- Besonders sinnvoll bei Wachstumsunternehmen, bei denen der Gewinn oder Free Cashflow (noch) keine Aussagekraft hat.
📘 Unternehmenswert zu Umsatz (EV/Sales)
📈 Was ist das?
EV/Sales zeigt, wie viel Anleger für 1 € Umsatz eines Unternehmens zahlen, wenn man auch Schulden und Cash berücksichtigt – es ist eine kapitalstrukturbereinigte Version des KUV.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Diese Kennzahl eignet sich besonders für den Vergleich von Unternehmen mit unterschiedlicher Verschuldung – sie zeigt, wie teuer ein Unternehmen tatsächlich im Verhältnis zum Umsatz ist.
🎯 Was bedeutet das für Anleger?
- EV/Sales ist neutral gegenüber der Kapitalstruktur und eignet sich gut für Unternehmensvergleiche.
- Ein niedriges Verhältnis kann auf eine günstig bewertete Aktie hindeuten – ein hohes Verhältnis auf hohe Erwartungen oder Überbewertung.
- Besonders nützlich bei wachstumsstarken, noch nicht profitablen Firmen.
📘 Unternehmenswert zu Free Cashflow (EV/FCF)
📈 Was ist das?
EV/FCF zeigt, wie viele Jahre es dauern würde, bis ein Unternehmen seinen Unternehmenswert durch freien Cashflow „zurückverdient”.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Diese Kennzahl hilft, Unternehmen auf Basis ihrer tatsächlichen Cash-Erträge zu bewerten – unabhängig von Bilanzierungsregeln oder buchhalterischem Gewinn.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein niedriges EV/FCF deutet auf eine günstige Bewertung bei starker Cashgenerierung hin.
- Ein hohes EV/FCF kann entweder auf Optimismus oder auf temporär schwachen Cashflow hindeuten.
- Besonders hilfreich bei reifen, profitablen Unternehmen mit stabilen Cashflows.
📘 Kurs-Buchwert-Verhältnis (KBV)
📈 Was ist das?
Das KBV zeigt, wie hoch der Marktwert eines Unternehmens im Verhältnis zu seinem bilanziellen Eigenkapital ist.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Das KBV ist besonders bei Substanzwerten (z. B. Banken, Industrie) relevant. Es hilft Anlegern zu erkennen, ob ein Unternehmen unter oder über seinem buchhalterischen Vermögen bewertet ist.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein KBV unter 1 kann auf Unterbewertung oder schwache Rentabilität hindeuten.
- Ein KBV über 1 zeigt, dass der Markt dem Unternehmen Mehrwert über den Buchwert hinaus zuschreibt (z. B. Marken, Patente, Wachstum).
- Das KBV eignet sich besonders gut für Unternehmen mit stabilen, materiellen Vermögenswerten.
📘 Eigenkapitalquote
📈 Was ist das?
Die Eigenkapitalquote zeigt, wie hoch der Anteil des Eigenkapitals an der Bilanzsumme eines Unternehmens ist – also wie stark es sich aus eigenen Mitteln finanziert.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Eine hohe Eigenkapitalquote steht für finanzielle Stabilität, Krisenfestigkeit und gute Bonität. Sie ist besonders relevant bei der Beurteilung der Verschuldung.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Eine hohe Eigenkapitalquote signalisiert finanzielle Stabilität – besonders in Krisenzeiten.
- Ein niedriger Wert kann auf ein höheres Risiko oder eine aggressive Verschuldung hinweisen.
- Wichtig: Die Eigenkapitalquote sollte immer gemeinsam mit der Eigenkapitalrendite betrachtet werden. Nur so lässt sich beurteilen, ob ein Unternehmen nicht nur solide, sondern auch effizient wirtschaftet.
📘 Eigenkapitalrendite (ROE)
📈 Was ist das?
Die Eigenkapitalrendite zeigt, wie effizient ein Unternehmen mit dem Kapital seiner Aktionäre arbeitet – also wie viel Gewinn es pro Euro Eigenkapital erwirtschaftet.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Die Eigenkapitalrendite ist eine zentrale Rentabilitätskennzahl. Sie hilft Anlegern zu erkennen, ob das Unternehmen eine attraktive Verzinsung auf das eingesetzte Eigenkapital erwirtschaftet.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Eine hohe Eigenkapitalrendite spricht für ein starkes, effizientes Geschäftsmodell.
- Besonders interessant ist sie bei kapitalintensiven Firmen oder solchen mit hoher Eigenkapitalquote.
- Wichtig: Ein sehr hoher ROE kann auch auf hohe Schulden hinweisen – daher sollte sie immer im Kontext mit der Eigenkapitalquote betrachtet werden.
📘 Return on Capital Employed (ROCE)
📈 Was ist das?
ROCE misst die Gesamtrentabilität eines Unternehmens – also wie effizient es das eingesetzte Kapital (Eigen- und Fremdkapital) zur Gewinnerzielung nutzt.
🧮 Wie wird es berechnet?
Das eingesetzte Kapital ist das gesamte betriebsnotwendige Kapital, unabhängig von der Finanzierungsquelle.
🏛️ Wofür ist es wichtig?
ROCE eignet sich besonders gut für den Vergleich unterschiedlich finanzierter Unternehmen. Es zeigt, wie effektiv ein Unternehmen Kapital investiert – unabhängig von der Kapitalstruktur.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hoher ROCE zeigt, dass ein Unternehmen sein Kapital effizient einsetzt – unabhängig davon, ob es durch Eigen- oder Fremdkapital finanziert ist.
- Je höher der ROCE im Vergleich zu ähnlichen Unternehmen, desto mehr Wert schafft das Unternehmen mit seinem investierten Kapital.
- Besonders wichtig ist der ROCE bei Firmen mit hohen Investitionen – z. B. in Industrie, Energie oder Infrastruktur.
📘 Return on Invested Capital (ROIC)
📈 Was ist das?
ROIC zeigt, wie effizient ein Unternehmen das Kapital investiert, das langfristig im operativen Geschäft gebunden ist – unabhängig davon, ob es aus Eigen- oder Fremdkapital stammt.
🧮 Wie wird es berechnet?
- NOPAT = „Net Operating Profit After Taxes“
- Investiertes Kapital = operatives Vermögen abzüglich nicht-verzinster Schulden
🏛️ Wofür ist es wichtig?
ROIC ist eine der präzisesten Kennzahlen zur Bewertung der Kapitalrendite – besonders im Vergleich zur Eigenkapitalrendite, weil es Verzerrungen durch Schulden vermeidet. Er zeigt, ob ein Unternehmen Mehrwert für alle Kapitalgeber schafft.
🎯 Was bedeutet das für Anleger?
- Ein hoher ROIC zeigt, wie gut ein Unternehmen mit dem tatsächlich investierten (betriebsnotwendigen) Kapital wirtschaftet.
- Im Unterschied zu ROCE wird nur Kapital betrachtet, das wirklich zur Finanzierung operativer Aktivitäten dient – und verzinst werden muss.
- Besonders hilfreich, um die Kapitalrendite von Unternehmen mit viel „überschüssigem“ Kapital oder zinsfreien Verbindlichkeiten realistisch zu vergleichen.
📘 Verschuldungsgrad (Leverage Ratio)
📈 Was ist das?
Der Verschuldungsgrad zeigt, wie stark ein Unternehmen durch verzinsliche Schulden (z. B. Kredite und Anleihen) im Verhältnis zum Eigenkapital finanziert ist.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Die Kennzahl hilft, das finanzielle Risiko und die Abhängigkeit von Fremdkapital zu beurteilen. Ein hoher Verschuldungsgrad kann die Eigenkapitalrendite steigern – birgt aber auch erhöhte Risiken bei Zinsanstiegen oder Liquiditätsengpässen.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein niedriger Verschuldungsgrad steht für finanzielle Stabilität und Unabhängigkeit.
- Ein hoher Wert kann auf erhöhte Risiken hinweisen – insbesondere bei schwankenden Zinsen oder konjunkturellen Schwächen.
- Wichtig: Immer im Kontext zur Branche und Kapitalintensität bewerten.
📘 Umsatz
📈 Was ist das?
Der Umsatz zeigt, wie viel ein Unternehmen insgesamt mit seinen Produkten und Dienstleistungen verdient – also den Bruttoerlös vor Abzug von Kosten.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Der Umsatz ist eine der zentralen Kennzahlen zur Einschätzung der Unternehmensgröße, Marktstellung und Wachstumskraft.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein wachsender Umsatz zeigt eine steigende Nachfrage und kann ein guter Frühindikator für Gewinnsteigerungen sein.
- Vergleiche von aktuellem und erwartetem Umsatz geben Hinweise auf das Marktumfeld und Analystenerwartungen.
- Wichtig: Starker Umsatz allein genügt nicht – auch Margen und Profitabilität zählen.
📘 EBITDA
📈 Was ist das?
EBITDA steht für „Earnings Before Interest, Taxes, Depreciation and Amortization“ – also Gewinn vor Zinsen, Steuern und Abschreibungen. Es zeigt das operative Ergebnis eines Unternehmens, bereinigt um bilanztechnische und finanzierungsbedingte Effekte.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
EBITDA ist eine verbreitete Kennzahl zur Beurteilung der operativen Leistungsfähigkeit – insbesondere bei kapitalintensiven Unternehmen oder im internationalen Vergleich.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hohes oder wachsendes EBITDA spricht für starke operative Erträge – unabhängig von Bilanzierung oder Steuerlast.
- EBITDA ist besonders nützlich, um Unternehmen branchenübergreifend zu vergleichen.
- Wichtig: EBITDA ist keine offizielle Gewinnkennzahl – Abschreibungen und Finanzierungskosten werden ausgeklammert.
📘 EBIT
📈 Was ist das?
EBIT steht für „Earnings Before Interest and Taxes“ – also Gewinn vor Zinsen und Steuern. Es zeigt das operative Ergebnis eines Unternehmens nach Abschreibungen, aber vor Finanzierungs- und Steueraufwand.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
EBIT ist eine zentrale Kennzahl zur Beurteilung der Profitabilität aus dem Kerngeschäft – unabhängig von Kapitalstruktur oder Steuersystem.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hohes EBIT deutet auf ein profitables Kerngeschäft hin – vor Zinslasten oder steuerlichen Effekten.
- Es erlaubt objektivere Vergleiche zwischen Unternehmen mit unterschiedlicher Finanzierung.
- Im Vergleich mit EBITDA zeigt EBIT bereits den Einfluss von Abschreibungen auf das operative Ergebnis.
📘 Nettogewinn
📈 Was ist das?
Der Nettogewinn ist der verbleibende Jahresüberschuss (oder -fehlbetrag) eines Unternehmens – nach Abzug aller Kosten, Steuern, Zinsen und Abschreibungen
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Der Nettogewinn ist die zentrale Erfolgskennzahl – er zeigt, wie profitabel ein Unternehmen nach allen Kosten tatsächlich arbeitet.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein steigender Nettogewinn zeigt, dass das Unternehmen effizient wirtschaftet – trotz aller Kosten.
- Die Entwicklung des Gewinns beeinflusst z. B. direkt das KGV und weitere Kennzahlen.
- Im Zeitverlauf lässt sich ablesen, wie stabil und profitabel ein Geschäftsmodell wirklich ist.
📘 Free Cashflow (FCF)
📈 Was ist das?
Der Free Cashflow gibt Aufschluss über die echte finanzielle Stärke eines Unternehmens – unabhängig von Bilanzierungsregeln. Er zeigt, wie viel Spielraum für Dividenden, Aktienrückkäufe oder Schuldenabbau besteht.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
FCF reflects a company’s real financial strength – regardless of accounting profits. It shows how much flexibility a company has for dividends, share buybacks, or debt reduction.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hoher Free Cashflow bedeutet, dass ein Unternehmen echte Finanzkraft besitzt – unabhängig vom bilanzierten Gewinn.
- Er ist oft die solideste Grundlage für nachhaltige Dividenden und Aktienrückkäufe.
- Sinkender FCF kann ein Warnsignal sein – auch wenn der Gewinn stabil aussieht.
📘 Umsatzwachstum
📈 Was ist das?
Das Umsatzwachstum zeigt, wie stark sich die Erlöse eines Unternehmens im Vergleich zum Vorjahr verändert haben – tatsächlich (TTM) und auf Prognosebasis (erwartet).
🧮 Wie wird es berechnet?
Erwartet = (Umsatz erwartet ÷ Umsatz Vorjahr − 1) × 100
Erwartetes Wachstum basiert auf Analystenschätzungen für das laufende Geschäftsjahr.
🏛️ Wofür ist es wichtig?
Ein wachsender Umsatz ist ein zentrales Signal für steigende Nachfrage, Geschäftsausweitung und Marktanteilsgewinne – besonders bei Wachstumsunternehmen.
🎯 Was bedeutet das für Anleger?
- Wachstum ist der Motor langfristiger Wertsteigerung – besonders bei Technologie- und Wachstumsaktien.
- Wichtig ist nicht nur das aktuelle Wachstum, sondern auch dessen Nachhaltigkeit.
- Prognosen zeigen, ob Analysten weiteres Potenzial erwarten – oder eine Verlangsamung.
📘 EBITDA-Wachstum
📈 Was ist das?
Das EBITDA-Wachstum zeigt, wie stark das operative Ergebnis eines Unternehmens vor Zinsen, Steuern und Abschreibungen im Vergleich zum Vorjahr gestiegen oder gesunken ist.
🧮 Wie wird es berechnet?
Erwartet = (erwartetes EBITDA ÷ EBITDA Vorjahr − 1) × 100
Erwartetes Wachstum basiert auf Analystenschätzungen für das laufende Geschäftsjahr.
🏛️ Wofür ist es wichtig?
Ein steigendes EBITDA ist ein Zeichen für verbesserte operative Ertragskraft – unabhängig von Finanzierungsstruktur oder Abschreibungen.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Starkes EBITDA-Wachstum signalisiert operative Effizienz und Skalierung – besonders relevant in Wachstumsphasen.
- EBITDA-Wachstum ist ein Frühindikator für Margen- und Gewinnentwicklung – sollte aber stets im Zusammenhang mit Umsatz und EBIT betrachtet werden.
📘 EBIT Wachstum
📈 Was ist das?
Das EBIT-Wachstum zeigt, wie stark das operative Ergebnis eines Unternehmens (nach Abschreibungen, aber vor Zinsen und Steuern) im Vergleich zum Vorjahr gewachsen ist.
🧮 Wie wird es berechnet?
Erwartet = (erwartetes EBIT ÷ EBIT Vorjahr − 1) × 100
Erwartetes Wachstum basiert auf Analystenschätzungen für das laufende Geschäftsjahr.
🏛️ Wofür ist es wichtig?
Das EBIT-Wachstum ist ein direkter Indikator für die wirtschaftliche Entwicklung des operativen Geschäfts – unter Berücksichtigung der Kapitalintensität (Abschreibungen).
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Steigendes EBIT signalisiert wachsende operative Rentabilität – auch unter Berücksichtigung von Abschreibungen.
- Das EBIT-Wachstum ist ein wichtiges Maß zur Beurteilung von Geschäftsmodellen mit hohen Investitionskosten.
- Im Zusammenspiel mit Umsatz- und EBITDA-Wachstum ergibt sich ein umfassendes Bild zur operativen Entwicklung.
📘 Nettogewinn-Wachstum
📈 Was ist das?
Das Nettogewinn-Wachstum zeigt, wie stark der Jahresüberschuss eines Unternehmens gegenüber dem Vorjahr gestiegen oder gesunken ist – sowohl tatsächlich (TTM) als auch auf Basis von Prognosen (erwartet).
🧮 Wie wird es berechnet?
Erwartet = (erwarteter Nettogewinn ÷ Nettogewinn Vorjahr − 1) × 100
Der erwartete Wert basiert auf Analystenschätzungen für das laufende Geschäftsjahr.
🏛️ Wofür ist es wichtig?
Der Gewinn ist die entscheidende Ergebnisgröße für ein Unternehmen. Ein wachsender Nettogewinn deutet auf steigende Effizienz, stabile Kostenkontrolle und nachhaltige Ertragskraft hin.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Wachsender Nettogewinn stärkt die Bewertung, Dividendenfähigkeit und Kursfantasie.
- Stagnierender oder rückläufiger Gewinn trotz Umsatzwachstum kann auf Margendruck hinweisen.
📘 Free Cashflow-Wachstum
📈 Was ist das?
Das Free-Cashflow-Wachstum zeigt, wie sich der freie Mittelzufluss eines Unternehmens im Vergleich zum Vorjahr verändert hat – also der Betrag, der nach allen operativen Ausgaben und Investitionen übrig bleibt.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Free Cashflow ist der echte, verfügbare Geldzufluss. Wachstum in diesem Bereich ist ein Zeichen für finanzielle Stärke und steigende Flexibilität bei Dividenden, Rückkäufen oder Investitionen.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Sinkender Free Cashflow kann auf steigende Investitionen, höhere Kosten oder stagnierende operative Erträge hindeuten.
- Besonders bei Dividendenwerten ist das FCF-Wachstum wichtig – denn Dividenden werden letztlich aus dem verfügbaren Cash gezahlt.
- Ein negativer Trend sollte genauer analysiert werden – er ist nicht zwangsläufig schlecht, aber potenziell ein Warnsignal.
📘 Bruttomarge
📈 Was ist das?
Die Bruttomarge zeigt, wie viel vom Umsatz nach Abzug der direkten Herstellungskosten (Material, Produktion) als Bruttogewinn übrig bleibt – also der „Rohgewinn“ eines Unternehmens.
🧮 Wie wird es berechnet?
Auch: Bruttomarge = Bruttogewinn ÷ Umsatz × 100
🏛️ Wofür ist es wichtig?
Die Bruttomarge gibt Aufschluss über die Profitabilität eines Produkts oder Geschäftsmodells vor Fixkosten, Steuern und Zinsen. Sie zeigt, wie effizient ein Unternehmen produzieren oder einkaufen kann.
🎯 Was bedeutet das für Anleger?
- Eine hohe Bruttomarge deutet auf starke Preissetzungsmacht und effiziente Herstellung hin.
- Sinkende Bruttomargen können auf Kostensteigerungen oder Preisdruck hindeuten.
- Besonders im Vergleich zu Wettbewerbern liefert die Bruttomarge wertvolle Einblicke in die Geschäftsqualität.
📘 EBITDA-Marge
📈 Was ist das?
Die EBITDA-Marge zeigt, wie viel vom Umsatz als operativer Gewinn vor Zinsen, Steuern und Abschreibungen (EBITDA) übrig bleibt. Sie misst die operative Effizienz – ohne Verzerrungen durch Finanzierung oder Buchwerte.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Die EBITDA-Marge hilft zu verstehen, wie viel operativer Gewinn ein Unternehmen aus jedem Euro Umsatz erzielt – unabhängig von Kapitalstruktur oder steuerlichem Umfeld.
🎯 Was bedeutet das für Anleger?
- Eine hohe EBITDA-Marge zeigt starke operative Ertragskraft – unabhängig von Bilanzierungseffekten.
- Die Marge ermöglicht gute Vergleiche zwischen Unternehmen und Branchen.
- Ein stabiler oder wachsender Wert kann auf effiziente Kostenkontrolle und Skalierbarkeit hindeuten.
📘 EBIT-Marge
📈 Was ist das?
Die EBIT-Marge zeigt, wie viel Prozent des Umsatzes als operativer Gewinn nach Abschreibungen, aber vor Zinsen und Steuern übrig bleiben.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Die EBIT-Marge misst die operative Ertragskraft eines Unternehmens unter Berücksichtigung der Kapitalintensität (z. B. Maschinen, Anlagen). Sie eignet sich gut zum Vergleich von Geschäftsmodellen mit unterschiedlich hohen Abschreibungen.
🎯 Was bedeutet das für Anleger?
- Eine hohe EBIT-Marge zeigt, dass ein Unternehmen auch nach Abschreibungen effizient arbeitet.
- Sie ist besonders relevant in kapitalintensiven Branchen.
- Langfristig stabile oder steigende Margen sind ein Zeichen wirtschaftlicher Stärke und Preissetzungsmacht.
📘 Nettomarge
📈 Was ist das?
Die Nettomarge zeigt, wie viel vom Umsatz am Ende als „Reingewinn“ übrig bleibt – also nach Abzug aller Kosten, Zinsen, Steuern und Abschreibungen.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Die Nettomarge gibt an, wie effizient ein Unternehmen über alle Stufen hinweg wirtschaftet. Sie zeigt, wie viel Gewinn tatsächlich je Euro Umsatz übrig bleibt.
🎯 Was bedeutet das für Anleger?
- Eine hohe Nettomarge zeigt, dass ein Unternehmen nicht nur operativ stark ist, sondern auch seine Finanzierung und Steuerbelastung im Griff hat.
- Vergleiche mit Wettbewerbern geben Einblicke in die wirtschaftliche Qualität.
- Sinkende Nettomargen trotz Umsatzwachstum können ein Warnsignal sein – etwa für steigende Kosten oder sinkende Effizienz.
📘 Free Cashflow Marge
📈 Was ist das?
Die Free-Cashflow-Marge zeigt, wie viel vom Umsatz nach Abzug aller operativen Ausgaben und Investitionen tatsächlich als freier Mittelzufluss übrig bleibt.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Diese Marge misst die echte Liquidität, die ein Unternehmen erwirtschaftet – unabhängig von Bilanzierungsregeln oder Abschreibungen. Sie ist besonders relevant für Dividenden, Rückkäufe und Investitionen.
🎯 Was bedeutet das für Anleger?
- Eine hohe Free-Cashflow-Marge zeigt, dass ein Unternehmen nachhaltig liquide Mittel erwirtschaftet.
- Sie ist ein starkes Signal für finanzielle Stabilität und Ausschüttungspotenzial.
- Wichtig ist der langfristige Trend – sinkende Werte können auf steigende Investitionen oder rückläufige operative Effizienz hindeuten.
📘 Ergebnis je Aktie (EPS)
📈 Was ist das?
Das Ergebnis je Aktie (EPS) zeigt, wie viel Gewinn auf eine einzelne Aktie entfällt – und ist eine der wichtigsten Kennzahlen zur Bewertung von Unternehmen.
🧮 Wie wird es berechnet?
Die verwässerte Aktienanzahl berücksichtigt auch potenzielle neue Aktien, etwa durch Optionen, Wandelanleihen oder andere Umtauschrechte.
🏛️ Wofür ist es wichtig?
EPS bildet die Basis für viele Bewertungskennzahlen wie KGV, PEG oder Payout Ratio. Es macht den Gewinn für Aktionäre vergleichbar – unabhängig von der Unternehmensgröße.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- EPS hilft, die Profitabilität pro Aktie zu erfassen – und ist besonders wichtig im Zeitvergleich oder im Vergleich mit Analystenschätzungen.
- Steigendes EPS kann ein Zeichen für stabiles Wachstum oder Aktienrückkäufe sein.
- Wichtig: Verwende verwässertes EPS für realistische Bewertungen – besonders bei stark aktienbasierten Vergütungssystemen.
📘 Free Cashflow je Aktie (FCF je Aktie)
📈 Was ist das?
Der Free Cashflow je Aktie zeigt, wie viel freier Mittelzufluss einem Unternehmen pro Aktie zur Verfügung steht – nach Investitionen, aber vor Dividenden oder Schuldentilgung.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Der FCF je Aktie zeigt, wie viel liquide Mittel pro Aktie tatsächlich im Unternehmen verbleiben – wichtig für Dividenden, Aktienrückkäufe oder Schuldentilgung. Im Gegensatz zum Gewinn ist er schwerer manipulierbar und daher besonders aussagekräftig.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hoher Free Cashflow je Aktie ist ein Zeichen für hohe finanzielle Flexibilität.
- Er zeigt, wie viel Kapital ein Unternehmen effektiv einsetzen oder ausschütten kann.
- Besonders relevant für dividendenstarke Unternehmen oder solche mit starker Kapitalrendite.
📘 Short Interest
📈 Was ist das?
Short Interest zeigt, wie viele Aktien eines Unternehmens aktuell leerverkauft wurden – also von Investoren geliehen und verkauft, in der Erwartung fallender Kurse.
🧮 Wie wird es berechnet?
Der Wert zeigt den Anteil der Aktien, der aktuell auf fallende Kurse spekuliert wird.
🏛️ Wofür ist es wichtig?
Short Interest dient als Stimmungsindikator: Ein hoher Wert deutet auf Skepsis oder negative Erwartungen gegenüber dem Unternehmen hin – kann aber auch zu einem „Short Squeeze“ führen, wenn der Kurs plötzlich steigt.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein niedriger Short Interest deutet auf Vertrauen in das Unternehmen hin.
- Ein hoher Wert kann ein Warnsignal sein – oder eine Chance, wenn sich die Stimmung dreht.
- Besonders spannend in volatilen Märkten oder vor wichtigen Quartalszahlen.
📘 Employees
📈 Was ist das?
Die Mitarbeiteranzahl zeigt, wie viele Personen ein Unternehmen weltweit beschäftigt – ein Indikator für Größe, Struktur und Geschäftsmodell.
🧮 Wie wird es berechnet?
🏛️ Wofür ist es wichtig?
Sie hilft bei der Einschätzung von Skaleneffekten, Effizienz und Personalkosten. Zusammen mit Umsatz und Gewinn lassen sich Kennzahlen wie Produktivität je Mitarbeiter ableiten.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Viele Mitarbeiter bedeuten große operative Komplexität – aber auch hohes Umsatzpotenzial.
- Produktivität je Mitarbeiter ist ein wichtiger Indikator für Effizienz.
- Besonders spannend bei stark wachsenden Tech- oder Industrieunternehmen.
📘 Umsatz je Mitarbeiter
📈 Was ist das?
Der Umsatz je Mitarbeiter zeigt, wie viel Erlös ein Unternehmen durchschnittlich pro Beschäftigtem erwirtschaftet – eine Kennzahl für Effizienz und Produktivität.
🧮 Wie wird es berechnet?
Die Mitarbeiterzahl stammt in der Regel aus dem letzten verfügbaren Jahresbericht.
🏛️ Wofür ist es wichtig?
Diese Kennzahl hilft, Geschäftsmodelle zu vergleichen – insbesondere zwischen arbeitsintensiven und technologiegetriebenen Unternehmen. Ein hoher Wert deutet auf Automatisierung, Effizienz oder hohen Wertschöpfungsanteil hin.
🧮 Berechnung
🎯 Was bedeutet das für Anleger?
- Ein hoher Umsatz je Mitarbeiter spricht für ein skalierbares und margenstarkes Geschäftsmodell.
- Ein niedriger Wert kann auf arbeitsintensive Prozesse oder geringere Wertschöpfung hinweisen.
- Besonders hilfreich beim Vergleich von Tech- vs. Industrieunternehmen.
BioVie Inc Aktie Analyse
Analystenmeinungen
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Analystenmeinungen
8 Analysten haben eine BioVie Inc Prognose abgegeben:
BioVie Inc Events
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BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Hello. This is Craig Brelsford with RedChip Companies. Thank you for joining today's event with BioVie, which trades on the NASDAQ under the ticker BIVI. With us today is Cuong Do. President and CEO of BioVie. We will begin with a brief presentation in a moment and then we will answer your questions. Welcome to everyone joining us today on X, YouTube, LinkedIn and other social media platforms.
To submit your question, we invite you to join us on Zoom. Use the link provided. Once in Zoom, click the Q&A button at the bottom of your window and type your question into the text box.
Before we begin, please allow me to read the safe harbor statement. This call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results along with other statements about the future expectations, beliefs, goals, plans or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact should also be considered forward-looking statements. Of course, forward-looking statements involve risks and uncertainties.
Cuong, please go right ahead.
Good morning, everyone. Thank you for joining today. My name is Cuong Do. I'm the President and CEO of the company. Give me 1 minute to get my screen share up and running again.
Our company has 2 assets. Bezisterim is a novel modulator of inflammation, and BIV201, which potentially has the potential to become the first drug to treat ascites, which is a terrible end-stage liver condition, right? We've had a very exciting August and September. About 45 days ago, we reported out on our Parkinson's Phase II trial. And just within the last week or 2, we've reported out on our Long COVID trial. So we will spend today mostly on these 2 clinical trial results.
As we look forward to the remainder of this year, we have a lot more data to analyze from the trial. So we will provide additional clinical updates, and excitingly, we plan to meet with the FDA, end of Phase II meeting for Parkinson's and hopefully get -- and we expect to get formal feedback from the FDA on our Parkinson's Phase III designed by the end of the year, perhaps even by Thanksgiving time.
We believe that inflammation is a starting point for a lot of things that go wrong in the body, particularly starting with TNF alpha. And when you have the production of TNF alpha, which is considered to be the master regulator of inflammation, it leads to a lot of the bad things that you see on this page, right? And that's where our drug candidate bezisterim comes in. Bezisterim is a small molecule that's orally bioavailable. So patients take 2 capsules a day, 1 in the morning and 1 at night. It really crosses the blood-brain barrier, so it gets into the CNS. And it's believed to block the activation of ERK and NF-kappa B. And by blocking ERK and NF-kappa B, you block the production of TNF alpha, right? So essentially, when you have TNF alpha, it leads to a forward-feeding proinflammatory cycle, so it just creates more inflammation.
When you have TNF alpha, it activates something called IKK and JNK. IKK and JNK binds to the insulin receptor substrate 1 and 2 and blocks insulin's ability to bind to that same IRS 1/2, thereby causing insulin resistance. So by blocking ERK and NF-kappa B, we'd block the production of TNF alpha, we've reduce inflammation and we've reversed insulin resistance. That's how bezisterim is believed to work. And you can see the impact of that.
First, in Long COVID. This is a trial that we reported out on just the last couple of weeks. Long COVID affects a large population. There are up to 20 million adults in the U.S. that are affected with Long COVID. Nearly 4 million have it so badly that they are considered to be disabled. They can no longer keep up the physical demands of the jobs because they're suffering from brain fog, fatigue and post-exertional malaise. And research has tied these conditions to inflammation that works through exactly the same mechanisms where bezisterim affects. And that's the reason why we believe bezisterim has the potential to become the first therapy for Long COVID because there is absolutely nothing that's available now. All the trials in Long COVID has failed.
And first, it's important to recognize that long COVID is not the same as COVID. COVID is the infection caused by the virus. Many of us have had it. Many of us have been able to ward it off and life just resumes, life goes on. But unfortunately, for the 20 million Americans who have Long COVID, they continue to suffer from these different conditions, right? And it lingers for years after the initial infection. And we believe that bezisterim may have an impact on those conditions. So we conducted the ADDRESS-LC Phase II trial. It is a signal-finding trial that's designed to essentially identify which endpoints could show that bezisterim could work to measure how big of an impact bezisterim could afford patients that are on drug and to identify the patients that can benefit from this drug, right, all of this is needed so that we can design the Phase III trial.
The Phase III trial was really the critical trial where you have to have a single endpoint or a few endpoints and all you're looking for is statistical significance. Does it work or does it not work with statistical significant? That is a Phase III trial that we now will plan. This Phase II trial gives us the information to do so, the planning. And in this Phase II trial, we evaluated 22 different clinical outcomes. We collect blood samples, and we did all of this with the support -- with a $13 million grant from the Department of Defense now the Department of War.
We have always known that Long COVID is a very complicated heterogeneous situation -- condition. So at baseline, we had 203 patients enrolling in our trial. We have always known that patients that have high inflammation or high symptom burden are the ones that are most likely to respond most quickly and most profoundly. So we always prespecify certain subgroup populations with the FDA before we unblind the study, and we did exactly that here. And one of those ways of specifying subgroups is based upon disease severity, right? And which disease symptoms. So here, what you find is in the trial, we had 112 patients with high fatigue, we have 98 with brain fog and 70 with post-exertional malaise. And we always talk of -- break Long COVID in those -- essentially involving those 3 symptoms. But interestingly, you see that only 15% of the patients have all 3 symptoms with high severity, right? But yet another 35% have some combination of 2 symptoms and another 35% have some combination of just 1 symptom.
But the important thing to recognize here is that if you do not have a symptom, it's not possible for you to improve on it. So here, you see 112 patients with high fatigue. That means all of these patients that are red and in green do not have fatigue, and therefore, they have no room to improve. That's a critical thing to keep in mind as we go through the data. Because here, as you look at what happens on the results for all 203, you see that on 21 out of the 22 endpoints, treatment -- you see a favorable treatment effect, right? So it's to the right. The Cohen's d is positive, meaning that the treatment effect favors bezisterim. But interestingly, none of these endpoints reach statistical significance of p=0.05 or less. And that has to do, we believe, with what we talked about here.
Only if you have fatigue, right, so if you look at this fatigue, only this 112 patients can really improve on it, which means that the improvement for these patients, the red and green is 0. And so while you see some impact, some effect favoring bezisterim treatment, you do not get statistical significance you have because so many of those patients, those that do not have high fatigue do not show improvement. And that's the reason why we prespecify in our submissions to the FDA that we will look at different subgroups based upon the symptoms they have and severity. And by doing so, you see a remarkable outcome.
So if you look at patients who have high fatigue, the impact doubles, right? The Cohen's d doubles. And you see that patients have statistically significant improvement on fatigue and other measures, right? So you see the p values here are less than 0.5 for 5 measures, and you get trending improvements on an Method 3. So you need to have fatigue to be able to improve on fatigue with statistical significance.
Similarly, if you look at patients who have high post-exertional malaise at the start of the trial, those patients improve on post-exertion malaise, the DSQ-PEM, right, with statistical significance. But these patients also improve under cognitive impairment on their brain fog. And this actually is very easy to imagine and to see. Sometimes when I and I'm sure many of us on this call, if we wake up early in the morning, if it's too early or whatever it is, we could be foggy, and so forth. So we're just not thinking clearly, right? So fatigue directly affect our ability to think clearly. But for these patients who suffer from high post-exertional malaise, those patients are suffering from it all the time.
And so here, by bezisterim's ability to help patients improve under fatigue, not surprisingly, they're improving on brain fog as well. And like similarly, if you have high brain fog, you improve on brain fog. So this shows that bezisterim has the ability to help patients improve on all 3 of these symptoms of fatigue, malaise and brain fog, but you need to have the symptom to begin with in order to improve, right? And this is the reason why our experts and all of the experts in the field are so excited about this because there's been dozens of trials that have all failed to demonstrate impact on any endpoint, whereas here, bezisterim, this is the first trial that has been able to show to have an impact not on 1, but on all 3 often neuro symptoms of Long COVID. This is the reason why all 12 of our PIs and our advisers, all 12 out of 12 that we have spoken to so far poll strongly recommend that we move quickly to Phase III because this is the first thing that has worked for these patients in the community.
And remarkably, consistent -- bezisterim continues to show that it's a very safe drug. There is 0 serious events and so forth. And the safety profile is incredible because in this population, virtually everybody is taking 1 or more other kinds of medications. So the last thing you want is to add to this mix a drug that potentially could have a drug-drug interaction or lead to other problems. And we just did not see any of that in this trial. So this gives us great excitement that bezisterim could become the first therapy for Long COVID, and we're still waiting for some additional biomarker data to come back from the labs in the next 45 days or so. We will analyze that and give additional readouts at that time. And based upon we have the totality of the data, we will then ask for a meeting with the FDA, an end of Phase II meeting with the FDA later on this year to go and discuss our plans for Phase III for Long COVID.
So with that, let me -- excuse me, 1 second. With that, I'd like to move on to Parkinson's. Parkinson's is known as a progressive neurodegenerative disorder that leads to problems with both motor and nonmotor symptoms, right? And there are no disease-modifying therapy out there. So there's great unmet need for a new medication that can address motor and nonmotor symptoms and hopefully modify the progression of the disease. And when we talk about nonmotor symptoms, I'd ask you to focus on the green part at the bottom of this chart that we found in a great article. So we just shamelessly copied and pasted it here.
When we talk about nonmotor symptoms, we're talking about sleep disorders, depression, cognitive impairment, lots of GI problems and constipation. And these symptoms often show up for years before a patient is actually diagnosed with the motor symptoms. And when you have the motor symptoms diagnosed, sooner or later, you will go on to a drug called levodopa or a similar drug right, to help address the motor symptoms, to help you improve your muscle control, but those drugs eventually lead to other complications. And as you can see over time, the progression of the disease only heads in 1 direction, it's only headed up.
What we believe is there is the need and the potential to change that. To conceptually display what we mean is if you look at the dash line, this is directly what happens to a patient's symptoms if it goes untreated. It just continues to get worse over time. But as you go onto levodopa to address the motor symptoms, you're addressing the motor symptoms, so you're shifting that curve down. But since nothing has been done to address the underlying progression of the disease, it continues to progress at the same pace, but just at lower level. And what we believe is needed is for a therapy that bends that curve, that changes the slope of that curve. And we believe that bezisterim has the potential to do so. That's why we conducted the SUNRISE-PD trial, where we looked at 4 different things. We looked at inflammation, biomarkers inflammation because that's how we're convinced bezisterim works. We conducted a proteonomic study looking at different plasma proteins of inflammation and CNS disease. We looked a lot at the neuronal injury, and we looked a lot at clinical outcomes.
And in our entire trial of 57 patients, what we see is that patients treated with bezisterim do not worsen as quickly as patients that are treated with placebo. And the way to read this chart is that a positive -- a bigger positive number is greater progression. So you see that those that are treated with bezisterim, shown in blue, do not worsen as quickly as those that are treated with placebo. But we do not see statistical significance at this gross level of 57 patients in the whole population.
But again, we know that patients who have inflammation or have higher disease burdens are the ones that will respond most quickly and most profoundly. And that's why we prespecified with the FDA before we unblinded the data that we -- unblinded the data. So what we found is that patients that have high inflammation, as shown by high level -- baseline levels of platelets, you see that those treated with bezisterim not only slowed the progression, we saw a reversal, an improvement of something called the Unified Parkinson's Disease Rating Scale that comes in 3 parts. Part 1 is the nonmotor, Part 2 is the activities of daily living skills and Part 3 is motor symptoms. Part 3 is what -- is the endpoint that the FDA has used historically to approve Parkinson's drugs. They also use the total score, right? And so here, either Part 3 or total, you see that we win statistically, right? So we have statistical significance showing that bezisterim-treated patients improved on treatment.
But you also see that we win on Part 1, which is the nonmotor endpoint as well as the activities of daily living. So this is the reason why we believe that bezisterim has the potential to become the first drug to address both the motor and the nonmotor symptoms of the disease. And of course, you may note that the nonmotor symptoms are considered to be the biggest unmet medical need in the Parkinson's population at this point.
We also created something called the EPNIC-15, which puts together motor, nonmotor and all of those, the endpoints into a single measure, EPNIC-15. Let me focus your attention to the left chart. And here, onto EPNIC-15, an improvement is a smaller number, a negative number. And here you see, if you focus on the left side of this chart, you see that the majority of the patients that improved are those that are treated with bezisterim, as shown in blue, and those that are stable, that did not change are those majority are treated with bezisterim. The fact that you did not improve or did not worsen is a win in our part -- in our mind because we believe that we prevented these patients from worsening.
Conversely, if you look on the right-hand side, most of the patients that worsened were on placebo, right? So you see very, very big statistical significance here when you look at this metric.
Another way of looking at the data is you see that 25% of the patients treated with bezisterim meaningfully improved compared to just 4% of those who were treated with placebo. Now conversely, only 14% of those treated with bezisterim meaningfully worsened compared to 46%.
So when we look at the totality of this data, we conclude that bezisterim treatment help patients meaningfully and statistically significantly improved on both their motor and nonmotor symptoms, thereby positioning bezisterim to potentially become the first drug to address both motor and nonmotor symptoms in Parkinson's.
Let me move on to look at biomarkers. We looked at 380 different biomarkers in what's called a proteonomic study. In that battery, there were 7 Parkinson's-specific biomarkers and all 7 moved in a beneficial manner. So that's statistically significant. There were 36 biomarkers of neuronal injury. Over 90% of those biomarkers moved in a beneficial direction. So highly, highly statistically significant. There were over 150 different biomarkers of inflammation. Over 75% of those moved in a beneficial manner. So highly, highly statistically significant, right?
We also dived more deeply in neurodegeneration. I'm particularly interested in looking at something called neurofilament light, or NfL, and the other is GFAP. NfL and GFAP are well recognized biomarkers of neurodegeneration. So the longer do you have the neurodegenerative disease, the more that these biomarkers continue to get -- to increase, right? They get released into your blood higher and higher levels the longer you have a neurodegenerative disease. But in this trial, you can see that those patients that were treated with bezisterim saw a significant decline of their biomarkers, particularly NfL. And the reason I'm interested in NfL is that this is the biomarker that's been used as the primary endpoint to get 2 drugs approved, 1 for ALS and the other is for MS, right? And you see a statistically significant reduction in -- with bezisterim treatment.
In dark blue here, dark thick blue, we give the composite of all of these different biomarkers and we replotted on the right-hand side. On the right-hand side, we also give the placebo composite and you see that they just moved in a completely different direction. So highly statistically significant, right?
So we look at this and we can conclude that bezisterim has the potential when looked in a much longer study as we will -- as we plan to do in a Phase III study, bezisterim has the potential to become the first drug to slow the progression of the disease, right? And if you're slowing the progression and here's the signs of it, you're slowing the increase of these neurodegenerative biomarkers.
And so we have asked for a meeting with the FDA, an end of Phase II meeting with the FDA to discuss our Phase III trial design, right? We have good ideas of what needs to be done. We've worked on this with our advisers. And we don't believe that we will have any trouble with the FDA on this trial design. So we expect to get formal feedback from the FDA by the end of the year. And if so, with funding, we believe we can start the Phase III trial by the middle of next year or so.
All right. I see that -- in the interest of time, let me stop it there, and let's open it up for questions.
Thank you very much, Cuong. To submit your question, we invite you first to join us on Zoom, use the link provided. Once in Zoom, click the Q&A button at the bottom of your window and type your question into the text box. Because of the great number of participants today, we can take your written questions only, and we have many of them already.
With so much good news on the results, why is the stock getting crushed? When do you expect to put in for the Stage III trials -- well, let's work with that, the first one, please.
I wish I can give you a great answer, right? Unfortunately, what we are experiencing is what other biotech companies are experiencing as well, where people sell on the news. And unfortunately, the shorts really had it in for us, right? Some short sellers made a lot of money by essentially driving our shares down. And they were able to do that because everybody was expecting us to go and raise some capital on the back of the good news. And so basically, they sell. Some of this, we believe, were just naked shorts and so forth, which is highly illegal, but there's nothing we can do about it.
Just to kind of give you some context. We have about 8 million shares outstanding in the total float. On the day that we announced our Parkinson's result, which are really good results, over 120 million shares were traded that day, right? So it was just like every single share was traded 15x over, right, which is absolutely crazy. And that ultimately drove our share price down. And because of that, we decided not to do the raise, right? And when we decided not to do the raise, our shares spiked up because of the short coverage. There was a classic short squeeze because people had to cover their short positions, right? And it's been lingering in the 2-ish position for some time -- $2 per share for some time.
I believe that is woefully undervalues our company, right? And I think over time, as people truly understand the results and perhaps as we get formal feedback from the FDA, I believe our shares will rally, right? And I say that not only because I'm the CEO of the company, I'm also one of the largest shareholders in the company that's buying shares, right, in the company, right? For the first time in very long time, the lawyers cleared me to buy shares because I no longer have material nonpublic information. So when that became possible, I and other members of our management team bought more shares.
Thank you, Cuong. Where does BioVie stand on their liver drug at the present time? It was announced in January that ThinkEquity was going to bring it public under the symbol OPTN?
Right now, the liver drug, that's the BIV201 is on hold. We have agreement with the FDA and what's needed to proceed to Phase III to get the drug registered, but we need to raise about $25 million in funding for it. And that's why we were planning to take -- put all of the ascites assets into a new company called Option Therapeutics and go and raise funding for Option through an IPO. And market conditions just hasn't allowed us to do that, right? So we're just waiting for market conditions to improve and the first opportunity, we will proceed down that path.
Given BioVie's current cash position and the recently established ATM, does management believe it has sufficient liquidity to reach the next major regulatory or clinical value inflection points without issuing a substantial amount of equity at approximately today's valuation? And is management's objective to use the ATM primarily as a bridge rather than as the principal source of financing for the next stage of development?
That's a great question. Thank you for that. I've refused to do a raise given our current depressed share price. That would be too dilutive to existing shareholders. And I just don't think that is the appropriate way to go. And I'm not going to use the ATM as the primary mechanism to raise additional funding partly because we're in baby shelf land. So the -- we're somewhat limited on how much we can raise with the ATM anyway, right? So the ATM is there just good corporate practice. Whenever there's an opportunity to opportunistically raise some cash, we'll do so, right? But we are not doing it in a manner to drive -- that will lead to our share price being depressed.
We will have to raise capital at some point in time in the future to fund the trials. But -- and I've received the feedback from some potential investors to say, they would feel much more confident in the shares of the company if -- when they get formal feedback from the FDA about the Phase III design. And we -- and that's why we are so focused on the end of Phase II meeting with the FDA to get formal feedback on the Phase III design. And we believe that will create a catalyst for our shares to rally. And when the shares rally, under the right conditions, we would consider raising the capital then.
With these great results, how close are we to partnering with a large drug company?
We are just now preparing to restart those particular conversations. Pharma companies always want to have the data and frankly, they want somebody else to go and pay to do the next trial rather than do it themselves, right? So creating the right competitive environment so that a company is forced into taking action is ultimately how you get partnering conversations to really work in our favor. And so we're taking our time to do it the right way. So that takes months, not weeks, right? And so we're initiating the process to have the right conversations with the companies that we've had conversations in the past.
With regard to SUNRISE-PD, are you asking the FDA to consider that one successful, adequate and well-controlled Phase III trial, together with SUNRISE-PD Phase II data and the mechanistic biomarker clinical evidence already accumulated provide sufficient efficacy evidence for an NDA?
We do not believe that one Phase III trial will suffice. It will be nice to have that. And then of course, that was always going to be part of our conversation with the FDA, but we're planning on conducting 2 Phase III trials, right? And we have good ideas on what that Phase III trial would be. There will be about 160 patients each. We would conduct 1 in the U.S., another 1 ex U.S., so that we can get global registration for bezisterim in Parkinson's.
In presenting ADDRESS-LC data initially, you mentioned the possibility of breakthrough therapy, fast tracked or other expedited designations within the FDA. Is this still the case?
We are preparing the application for breakthrough right now, and we'll be submitting that within weeks in due course. And then we are still waiting for biomarker data to come back from the Long COVID trial. We collected a lot of blood samples. And what we'd like to do is to batch them all together at the very end of the study and go and have all of them analyzed at the same time so that we minimize what's called batch-to-batch variation, right? One would think that when you run a test, today, tomorrow, the day after you get the -- on the same sample, you get the exact same results. You do not, right? There's variability, right?
So what we'd like to do is to put everything together and run it all in 1 batch. And the labs usually take about a couple of months to run all the samples to get it back to us. So we're just waiting for the biomarker data to come back so that we can tie together the outcomes, the clinical outcomes data with the biomarker data to try to explain what's going on and build a much stronger case. And here in Long COVID, we are cautiously optimistic that we would only need 1 additional Phase III trial because we believe the trial that we have just completed was not only well controlled, but it was large enough to demonstrate the effect, right?
And so we are cautiously optimistic that we may only need to conduct 1 trial. Furthermore, we are in the process of applying for some grants that potentially could pay for the Phase III trial as well, right? So more to come in the coming months as we learn more on that.
When will additional ADDRESS-LC biomarker data be shared?
Like I just mentioned, we're still waiting for the data to come back. And I would guess that before the end of the year, we will share additional data on biomarkers. And frankly, we need to kind of move on that because there are some conferences, scientific conferences that we would like to present the data at early part of the year.
Thank you, Cuong. Please discuss the comment made that said the Long COVID trial misses statistical significance in full population.
See I do not consider that we missed statistical significance in the full population, right? We have to think about different trials as having different objectives, and therefore, have to be judged differently. In a Phase III confirmatory registrational trial, the objective is to demonstrate statistical significance on the primary endpoint. So you have a primary endpoint and the only objective of the trial is, did you get p-value of 0.05 or less. That's the only objective of a Phase III trial, right, to show that it works and that you have statistical significance.
A Phase II trial is different. A Phase II trial is exploratory. And very few Phase II trial has statistical significance at the entire population, right? Phase II trials are exploratory to help you identify which endpoints show that the drug works with which population, right? And you hope to get statistical significance on the endpoint and in that population. And that's where we believe we have demonstrated in spades that the drug works because in patients with fatigue, we had statistical significance on 5 different endpoints, 3 of which were fatigue endpoints.
With patients with malaise, we had statistical significance on the 1 metric that's malaise, so DSQ-PEM, but we had statistical significance on a number -- another half a dozen end points having to do with brain fog or cognitive impairments. And for patients who have brain fog, they improved on all 4 cognitive impairment endpoints with statistical significance. So we believe we absolutely hit on statistical significance with the relevant endpoints and the relevant population. So we consider this Phase II trial to be a major, major win.
And with that information, we now know how to design the Phase III trial. And our team has done this in the past, and we don't believe that we'll have any difficulties with the FDA around the way that we're thinking about the Phase III trial, and we look forward to discussing with them in due course.
When do you expect BioVie to pursue further Alzheimer's disease trials?
That program is proceeding far, far too slowly, right? And unfortunately, that is all funding related. And our priorities right now, unfortunately, has to be Long COVID and Parkinson's because these trials are relatively small and short. The Long COVID, it's only a 3-month long trial for Phase III, right? And the trial we believe is only going to be about 230 patients large, and it's 250 patients large or so. So these are relatively short and inexpensive trials to conduct, especially if we can get a grant to cover some of that cost.
In contrast, the Alzheimer's trial is going to be long. It's at least 6 months long, extendable for another 6 to 12 months. It's going to be large, right? It's going to be like 400 patients large, right? And so we just have to prioritize. And we will start that when the capital markets allow us to have the ability to raise the capital or when a partner materializes that wants to work with us on that.
Can you explain in simple terms what this drug, bezisterim, actually does in the body?
Sure. Let me take us back to a bit of high school biology. We all know that every cell in our body needs energy and energy comes in the form of ATP, okay? And in high school biology, we all learned that glucose goes through the Krebs cycle to produce ATP as the energy for the cell. And cells need to absorb glucose from the outside of the cell. And when it comes to glucose regulation, insulin is the key. Think of insulin as the key that has to fit into a lock on the surface of every cell in our body.
When everything is working well, insulin fits in the lock, opens up the door, glucose can be absorbed, everything goes fine. But when you have inflammation, so when you have TNF alpha present, it activates IKK and JNK, as I mentioned earlier. So think of inflammation as rust that builds up on the lock. And when you have a rusty lock, insulin, the key, cannot fit in, cannot open the door. So the cells cannot get the glucose that it needs. So those cells start to malfunction, and over time, they die. And of course, our neurons are the most energy starved cells in the body. And that is the basis for neuronal degeneration. That's why the term type 3 diabetes have been coined to describe the metabolic underpinnings of the neurologic diseases.
How bezisterim works, we believe, is that it blocks the production of TNF alpha. So think of it as it's getting rid at the rust that's on the lock. And by blocking TNF alpha, reversing insulin resistance, you're now allowing insulin to do its work. You're allowing cells to become -- to be healthy again, and you're keeping those cells alive versus if without the drug, cells would have gone on to die.
So hopefully, that explains it in a very simplistic manner, a bit long-winded, right? But the drug reverses insulin resistance, thereby, it restores cells ability to absorb glucose and function.
Thank you, Cuong. And someone said nice presentation. So thank you for that. Thank you for your attendance as well to that person and to everyone here.
Next question, Cuong. How do you plan to leverage or prove the great potential disease-modifying rather than just disease treatment potential of bezisterim, particularly with respect to Parkinson's disease given the biomarker data? Will this require additional or lengthier studies?
Well, we are -- the study that we have designed, we're trying to kill 2 birds with 1 stone. We are contemplating conducting a Phase III trial with 160 patients that has multiple endpoints. The primary endpoint will come after 6 months of treatment to get at symptomatic relief. So that's where we hope to show that we help patients improve on both their motor and nonmotor symptoms after 6 months of trial, right? And that's enough data for us to go and file to get registration approval. But as that 6 months come to an end, we will extend it for another 6, potentially 12 months so that we continue to monitor these patients to see if their progression is on the disease has changed from what is expected.
And all patients that were originally on placebo will -- at that point in time, will move on to drug as well. So if we see if there's progression that -- it's a very complicated design but -- to explain, but it's done all the time in clinical trials by essentially what's called a study extension. You monitor patients less intensively for a longer period of time to see what happens to them over time. And if you can show that their disease progression has changed, that's how you can get a disease modification claim.
We'll give a moment to consider any more questions they may have for Cuong Do, the President and CEO of BioVie.
Okay. Seeing no other questions, let me bring us to a close by summarizing that in bezisterim, we have a remarkable molecule that works to reverse insulin resistance and reduce inflammation. In our clinical trials that we have discussed today, bezisterim has been able to help Long COVID symptoms of patients who have the relevant symptoms at baseline improved on their fatigue, malaise and cognitive impairment also known as brain fog, right? The key to understand this drug is to recognize that you need to have the symptom to begin with to improve on that symptom, that bezisterim has shown that it's the first drug that we know of that's been shown in the clinical trial that it can help patients address not just 1, but all 3 of these symptoms with the critical proviso that you need to have the symptom to be able to improve upon it.
So we're very excited about this. And all of the key opinion leaders in the field, all the clinical trialists in the field that we have worked with and interacted with are excited about the results and they're urging us to move into Phase III as quickly as we can.
Also, bezisterim has demonstrated it can help patients improve their Parkinson's symptoms, both their motor and their nonmotor symptoms, and it has shown early signs that it can slow neuronal degeneration by essentially reversing the biomarkers of neural degenerative diseases, particularly NfL and GFAP.
We're very excited about this drug. We do not believe that our shares adequately represent and factor in the true value of the drug, right? And we're doing everything we can to get the word out to help people understand the true value of the drug. And we believe that the meeting with the FDA and the clear guidance from the FDA on our Parkinson's Phase III design will provide the next catalyst for our shares to rally. And I believe we are highly undervalued at this point. And I'd say that as one of the largest shareholders in the company, and I say that as a member of management who's been buying shares when allowed, right?
I thank you for your time, and please find out more about our company by going to bioviepharma.com. And we are also traded on the NASDAQ under the ticker symbol BIVI. Thank you very much for your time today. Have a great day.
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BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Good morning, and welcome to the BioVie conference call. [Operator Instruction] As a reminder, this call is being recorded, and a replay will be made available on the BioVie website following the conclusion of the event. I'd now like to turn the call over to Cuong Do, Chief Executive Officer of BioVie. Please go ahead, Cuong.
Good morning, everyone. Thank you, Tara. This is, we believe, is a great day for the Long COVID patient community with some of the results that we'll be discussing today. I'm very excited to be sharing this with you. I'm joined today by 2 experts, which will come on camera later on. Michael Peluso is one of our clinical investigators who's been part of many Long COVID trials as well as Ezra Spier, who is a Long COVID patient advocate, who's also been participating in many trials. Over the course of this presentation, we will make certain forward-looking statements. I will let you read this at your leisure. This presentation can also be found at our website.
We are here because of bezisterim. Bezisterim is a very novel first-in-class molecule that is orally bioavailable and it crosses readily into the blood-brain barrier. And it is believed to act the block ERK and NF-kappa B activation and thus, it blocks the production of TNF-alpha, which is considered to be the master regulator of inflammation. And we are studying bezisterim in long COVID because it's believed -- if you look at the graphic on the right, it's believed that the spike protein, the envelope protein on the COVID virus if you have the infection or if in long COVID, those fragments of those proteins are believed to activate something called the toll-like receptor 2 or the toll-like receptor 4, which essentially activates NF-kappa B and ERK to lead to TNF-alpha and the production of other inflammatory mediators. And of course, since bezisterim blocks the activation of NF-kappa B and ERK, it is believed that it can have all the downstream impact and thus hopefully help with long COVID symptoms. So we conducted this long COVID -- this ADDRESS-LC Phase II trial. It is designed as a Phase II signal finding proof-of-concept trial.
As you know, drug development typically goes through three different phases. Phase I first establishes that a drug is safe. Phase II is trying -- is a signal finding. So try to figure out does the drug have a signal? Does it have a chance of working in some patient population and how do you show that it works. And then once you get the information from Phase II that shows that, well, if we use this endpoint with this group of patients, you can move on to Phase III confirmatory trial, which basically says this is our endpoint, and we're trying to get a statistical significance. That's the purpose of Phase III is confirmatory. Phase II is purely signal finding. So what we're trying to do is to identify the efficacy signal. So namely what endpoints can we use to show that the drug has a favorable treatment benefit. We're trying to measure the magnitude of that impact, right? And we're trying to identify which patient populations can benefit from that. We need all this information so that we can design and size the Phase III trial.
In this trial, since it's the first time that we're looking in Long COVID, we evaluated 22 different clinical outcomes to try to figure out which measure works best. We also collected blood samples to assess a series of biomarkers, right? And we're conducting a proteomic study evaluating over 380 different proteins. And it should be noted that this work was supported by a grant from the Department of War. So let me first jump to give you the key findings before we go through the details. The first thing to recognize is that long COVID patients are not one homogenous group. And in fact, it is a very, very diverse and heterogeneous population. And that we tend to think of patients -- when we think of long COVID, we tend to think of fatigue, malaise and brain fog, and we often assume that a patient has all three of those. And in fact, they do not. Patients can have any combination of one, two or three of those symptoms.
And the key finding from our trial is that we believe bezisterim is very effective for these different patients who have severe symptoms when they start the trial. But the critical thing to recognize is that you need to have the symptom to begin with in order to improve, right? If you have high symptoms of fatigue, you improved statistically significantly versus placebo on five different fatigue endpoints, along with trending in sleep and post-exertional malaise. If you have high malaise levels, you improve on malaise as well as cognition. And if you have high cognitive impairment at baseline or brain fog, you improve on your brain fog. And lastly, the safety and tolerability profile of bezisterim was very similar to placebo. This is a very critical finding, especially in this patient population where virtually every patient is on multiple different medications.
So one thing to recognize is that long COVID is not COVID. It is not the same, and it shares an unfortunate part of the name. COVID, as you know, is the active infection that comes from the virus, right? Long COVID is a description of the aftermath of the infection. So these are symptoms that are quite diverse. It could be fatigue, it could be brain fog. It could be very heterogeneous and it could last for months and years after the actual COVID infection itself. It's a very large population out there, 17 million to 20 million U.S. adults are believed to have long COVID symptoms now and 3.8 million have it so severely that they're essentially considered to be disabled. They can no longer work or keep up with the physical demands of the job. And there are no therapies out there. So there is a huge great medical need for a therapy to address long COVID symptoms.
And the challenge of developing a drug for long COVID is actually not only to show that it works, but it's actually finding how, right? Because, one, you see it's a very heterogeneous population. There are no validated biomarkers. There is no biomarker that you can point to that says this person has long COVID. It is purely assessed symptomatically and has large background variability and everybody is on multiple medications. So it's actually very difficult to find out how it works, how a drug works, who can benefit from it. And that's exactly what we set out to do, and we believe we have achieved that.
I mentioned we measured 22 different endpoints. So this gives you a list of what those things -- those endpoints are. And a sense of how to read the numbers. When we're looking at clinical trial results, we're looking at two different things. One is just how large is the effect. So how much of a benefit does treatment provide compared to placebo. And statistics gives us a tool to do that, something called the Cohen's d, which measures the magnitude of the impact, the difference between two groups. The larger the number, the better.
The second gets to a question of how sure are we that this result is significant. And so we look at the p-value. The smaller the p-value, the better. And typically, something is considered to be statistically significant if the p is less than 0.05. At baseline, we looked at -- we compared the bezisterim treatment group versus the placebo group, and we see that virtually everything is very well balanced with one possible difference being slightly more women in the bezisterim treated group compared to those that are on placebo, but everything else is pretty much balanced across the board.
The other thing we find at baseline is there is huge variability in the symptoms, okay? The way to explain this chart is that if you look at the left blue circle, you see that there are 112 patients out of a total of 203 that have high levels of fatigue at the start of the trial. If you look at the green, there were 98 patients who have brain fog, high levels of brain fog, and at the red, there are 72 patients with high post-exertional malaise. And equally important, if you look at the yellow, there are 44 patients or 22% with low symptoms in all three. And as you can see, there is huge mixtures, right? So right in the middle, there's only 29 patients or 14% who have all 3 symptoms. And if you look around, there are about 35% of the patients who have some combination of 2, right? And likewise, there are about 35% of patients who have just one symptom.
This is really, really critical to understand, right, because this is where everything starts. If you look at the 112 patients who have high fatigue, that's about half -- 55% of the trial. But that also means that there are 45% of the patients in this population that does not have fatigue symptoms, right? And one thing that we have always known about bezisterim and frankly, many, many other drugs is that you need to have the symptom to begin with before you can improve on that symptom. So if you don't have the symptom and it -- you can't improve or if you have low levels of that symptom, you cannot improve, right? So understanding what symptom you actually have, what symptom a patient subgroup actually belongs in effect is critical to understanding the results. And you will see that on the next page because if you throw all 203 patients together and look at the 22 different endpoints, you will see something that's a very interesting pattern.
You will see that on all except one, all of the patients, all the Cohen's d favor bezisterim treatment. It basically giving us a signal that the drug is -- appears to be working for patients who are taking the drug. But yet, when you look at the p-value for any one of these endpoints, you see that they do not reach the 0.05 level for statistical significance, right? So if you were to just stop there, you would say, oh, the drug did not work because we did not have statistical significance. But drawing that conclusion would be a huge mistake because of the point I made here about the different subgroups. You have to have a symptom to improve upon that endpoint. So if you -- and this becomes very clear here.
So one thing that we do in clinical trials is we look at a patient at baseline based -- we divide them up into different levels of severities. We divide them into quartiles and tertiles, right? And you can very clearly see here that on baseline PROMIS fatigue, so this is an endpoint that looks at a patient's fatigue level at baseline, you see that those that have higher fatigue levels are the ones that are improving the most. And this is consistently what we see with bezisterim across the various trials that we have conducted. So all we really did is we said top half of patients. So patients who have greater than median fatigue are considered to have high fatigue. And this is what you find. This is a summary chart.
Let me just go into the next one to give a bit more detail. So this chart looks only at the patients who have high fatigue, so the 112 patients who have high fatigue level. What you see at the top in blue are five different endpoints that reaches statistical significance, the top three of which are all fatigue. The next one are the patient's impression of how they're feeling and the clinicians' impression and how the patient is doing. You notice that the Cohen's d is higher. It's double what you see before. And like I mentioned earlier, all five of these metrics reach statistical significance. So what this says to us is that if you look at patients that have fatigue symptoms, bezisterim treatment helps them improve statistically significantly on fatigue.
In addition, we get trending statistics on three additional endpoints being malaise, sleep and the patients also just -- they just feel better. It's less severe for them. So this makes intuitive sense, right, because all of these endpoints are fatigue related, right? And if you are less fatigued, frankly, sleep is -- goes along with it. If you're sleeping better, chances are you're going to be less fatigue. Now let me move on to the next group, which is the malaise group as measured by the endpoint, DSQ-PEM. Again, you see that the Cohen's d dramatically gets better.
The patient improved statistically significantly on malaise, the fifth -- I'm sorry, I don't have a laser pointer that could point out here. But the fifth item down, you see that on PEM, patients statistically significantly improve on malaise -- but they also improve on cognition or brain fog, which makes intuitive sense. I don't know about you, but I -- sometimes when I wake up in the morning and Ragi didn't have enough sleep, I just don't think as clearly, right? Unfortunately, this is a patient population that lives with that constantly. And by being able to help these patients with high post-exertional malaise, you're helping -- bezisterim appears to be helping not only in malaise, but you're helping them with their brain fog, right? And then four additional metrics get to trending statistics.
And lastly, let me show what happens to those with brain fog. Again, the Cohen's d gets dramatically higher and bezisterim treatment appears to help statistically significantly on four different brain cognition endpoints, and the patient also feels better on fatigue as well. So very consistent findings that if you have the symptom at the start of the trial, bezisterim treatment appears to give you statistically significant advantages over those on placebo. So it's helping you improve upon your symptoms. Safety tolerability is very comparable bezisterim treatment versus placebo. And that is actually very important when you look at what really goes on with these patients.
You see that 96% of the patients are currently taking one or more medication, right? And the medication classes that they're in, there are 13 different classes, right, representing 873 different medications here. So the last thing you want is to introduce onto this polypharmacy situation, a drug that could have safety concerns or drug-drug interactions or safety concerns, and that we did not see. As you can see from the safety information here, it was just very benign, very similar to placebo. So we look at this and our interpretation is that bezisterim appears to be helping long COVID patients with high baseline symptomatic burden, right? And baseline fatigue, post-exertion malaise and cognitive impairment really drives the patient's ability to improve on that endpoint.
So the key point is you have to have the symptom to improve. And if you are not affected by the symptom, there is little room for you to improve. Bezisterim's safety and tolerability is good. It allows for potential use with the patient population that's already taking many other medications. And this is a very compelling signal that warrants advancing it to confirmatory Phase III trial. This is what we interpret, right? But we also have the benefit of working with a lot of different leading experts in the field. So we've had the opportunity to share this information a couple of days ago with our investigators and our advisers, whom of those you can see in their names are listed here. And we asked, among other questions, we asked every one of them, what would you, do would you advance this to confirmatory Phase III trials? And all 12 out of 12 of our investigators and advisers strongly suggest that we move this on to Phase III because this is literally the first medication, we believe, that has been shown to show a benefit on these neurological symptoms of Long COVID in any patient population.
So looking ahead, we still are waiting for biomarker data to come back. We're hoping to get those in the next couple of months, and we will marry in the various biomarkers data with these clinical endpoints. We still have to plan for two different end of Phase II meetings with the FDA, of course, for our Parkinson's trial that we reported out about a month ago and now our Long COVID trial. And we have to plan for Phase III potentially pivotal registrational trials. We have lots of data to continue to analyze and there are a number of partnering conversations that we need to do in due course, right? So just to give you a pipeline overview, bezisterim is being developed for Parkinson's and Long COVID. We also use evaluating in Alzheimer's disease in due course as well. And we also have another asset, BIV201 for ascites, all of which -- any one of which of this indication are significant commercial opportunities as well.
So with that, I thank you for your time. What I would like to do is turn the floor over to Michael. So Tara, can you please stop the slides here? And let's bring Michael on screen. Michael Peluso is a clinical investigator from the University of California, San Francisco. He has been involved in a number of long COVID trials. So let me turn it over to Michael. Could you please introduce yourself real quickly and give us your take on the data as you've seen it here.
Yes. Hey, everybody. I'm really happy to be here. I think this is a really exciting day for long COVID patient community and long COVID researchers. I've been working in this field for 6.5 years now since the very beginning, and there's been a lot of disappointment. And these results, when I saw them, are shocking to me in the best possible way. This is really the most promising data that I have seen in this field, and I'm really excited to see where this goes.
Thank you, Michael. And can we bring Ezra on the camera as well? Ezra is -- has been working in this field for quite some time. He's a leading advocate for long COVID patients. So Ezra, can you introduce yourself a little bit and give us your take on this?
Sure. Thanks so much for having me. My name is Ezra Spier. I'm somebody living with long COVID. I've been sick for just about 4 years. And there are so many millions of patients like me who've been really struggling to get the care and support that we really need to live our lives and contribute to society in the ways that we want to. I'm very involved in long COVID clinical trials as both a participant in trials and also an adviser to different trials. And I think one of the things that's been very challenging is the heterogeneous population.
We know in the community that many of us try a very wide variety of repurposed medications that already exist and able to be used off-label and not everything works for every person. And when we look at the research that has come out, it's been very challenging. Even large trials have not shown consistent impact on patient populations. And even within subgroups, oftentimes, there's just not much of a signal. And I think what really stands out to me about the data that have been presented today is that it shows a very clear direction in the data.
And to me, that is almost more interesting than a particular -- meeting a particular threshold because what I know as a patient based on the community is that we need to figure out for whom is this going to work best and what dosage is going to work best and all these questions that come up, but we need to have a signal first. So I think this really gets us to -- gets me excited about the additional discoveries we'll hopefully be able to make. I think it gives the community some hope because we've been seeing trial fail after fail after fail. And so seeing something that provides demonstrated impact, I think, is really exciting.
Thank you, Ezra. Tara, can you please bring everybody on the panel on for -- let's move on to the Q&A. There are a number of questions, I'm sure that's been posed.
Great. Yes. Thank you, Cuong. [Operator Instructions] So our first question was ADDRESS-LC statistically powered to detect significance in the full ITT population? Or was it designed from the outset as an exploratory signal finding study without that expectation? And why was it designed without a single prespecified primary endpoint? And what does that mean for how the FDA will interpret the results?
Well, thank you for the question. The study was designed at the outset to be a Phase II signal finding study. This was a significant size. This is quite large. It's larger than a normal Phase II study because, frankly, we did not know what to look for nor did we know what to find. That's the reason why we look -- we had 22 different endpoints, right? And the beauty is that when you're in Phase II exploratory, you don't have to declare a single endpoint.
That's why we declared all 22 of these different endpoints as, frankly, we're on a fishing expedition to basically see which endpoints could show a difference for those that are on drug versus those that are on placebo. And we did not know what to expect. And the fact that we ended up finding a signal here that showed that it was effective for 78% of the patients in the study was astounding, right? This is a huge signal and it's for a and 78% is a much, much larger responder population than you would typically see in any kind of trial. I hope that answered your question.
Great. Thanks, Cuong. The next one, since this study is described as hypothesis generating, does it make more sense for management to conduct another Phase II trial in patients with high symptom burden rather than moving directly to a Phase III?
The purpose of the Phase II trial is to give us the information so that we can move forward to design and size the Phase III trial. So we think we have that information. But let me ask Michael and Ezra to comment. They've worked on more long COVID trials than I have, right? So Michael?
Yes. I mean a couple of points I would make from this discussion. There have been, at this point, a few dozen long COVID trials of other agents. And really none of those have had results that are as consistent and as compelling as what I'm seeing here. Often in these trials where you have lots of endpoints, you're kind of contorting yourself to do all of these exploratory analyses and maybe one thing sort of pops up as interesting or compelling. In this study, it's a very sort of logical, easy-to-understand partitioning stratification of the participants, those with the most severe symptoms and the most area to improve.
And the results are consistent across almost all of the outcomes. And so it's really a very kind of simple in a good way story to have results that just intuitively make sense. And so from my perspective, that gives this group a lot of direction on how to think about a Phase III study. So what I would say from my perspective as a clinical trialist in this field is that I think there's enough here to thoughtfully and carefully design a Phase III study that could be really a definitive trial.
Ezra, anything to add?
Yes. No, echoing what Michael just said, I definitely agree. I think that the -- I would have -- I would be concerned in a situation where a Phase II trial had less compelling results, to be honest. And I think that this is compelling enough to warrant a probably larger population that we're looking at. It gives us some areas to focus in on. There's still potentially opportunities for deeper dives into certain aspects or subgroups of the population. We'll leave that design up to the experts, of course. But I think the other thing that's really important to keep in mind here, and I'm not coming here as an investor, I'm coming here as a patient is my community is really struggling.
We really need assistance. And I think one of the aspects of the results that really stick out to me is the particular symptoms that seem to be improved with the drug. And so symptoms like post-exertional malaise, where a small amount of exertion can create a disproportionate amount of fatigue and other symptom exacerbation, really debilitating symptoms. And so to speak for folks in my community, we're really looking for things that can help us now. And so I wouldn't want to delay moving towards a potential FDA registration for a drug if there's not a really important reason to do that additional work. So I would, on behalf of my community, encourage expediency wherever possible.
Thank you, Ezra.
Great. Thank you for the responses. So our next question, can you describe the biological characteristics of the prespecified subgroup that demonstrated significant clinical benefit? And did treatment-related changes in inflammatory or neurodegenerative biomarkers correlate with the magnitude of clinical response in those patients?
Actually, Tara, can you please bring back the slides? That's -- I think that this question is best answered by looking at the Venn diagram and the what we call the forest chart. So the baseline characteristics are all defined by three metrics that gets at the symptom. One is fatigue as measured by PROMIS fatigue that's the endpoint, right? And here, we just took the median. If you have a higher than the median score of 65, you would consider it to be high fatigue. On malaise, post-exertion of malaise, we use the only tool that's available, something called the DSQ-PEM, right? And if you were in the top third, you consider to be high on post-exertion malaise. And on brain fog or cognitive impairment, we use something called the Cogstate batteries to measure objective cognition.
So we give a person an iPad and give them mental exercises. We give them exercises to do and various things are measured to see how long it takes you to do things and so forth. It all gets at brain fog, can you do it quickly, slowly and so forth. So that objectively measures your cognitive ability. It's as simple as that. And here, it's -- again, we use the median as the cut. And those three endpoints were used and those are the three most representative endpoints to define the cutoffs, if you will. And you can see on this chart, we call the forest chart. This is a summary of what I showed you earlier. If you have high fatigue when you started the trial, you saw statistically significant improvements in five different endpoints, three of which are all fatigue directly measuring fatigue, then two of which are just measuring how you feel and the clinician sense of how you're doing.
If you look at the high malaise group, you improve on malaise statistically, DSQ-PEM, the fifth line item down, but you also improve on brain fog as well because you're just able to think more clearly because you're just not tired and malaise. And lastly, if you have high brain fog or cognitive impairment, you improve on four different endpoints looking at brain fog with statistical significance. right? I hope that answered your question. So let's bring the panel back up, please, Tara.
Great. Thanks, Cuong. So the next question, what is the biggest piece of evidence from this trial that gives you confidence bezisterim can succeed in Phase III?
Actually, let me ask Michael to comment on that and then Joe.
I mean two things I would say. The first thing is that the effect sizes are pretty compelling. Often, we're reporting out these sort of miniscule effects. And here, across all of the domains that were mentioned, the people who responded seem to have sort of this moderate high effect, which I think is really promising and suggesting there's sort of a real tangible benefit that could be present here.
The second thing I would say is we're mostly focusing on the potential efficacy outcomes here. But the safety data are actually really important, right? I'm a physician, safety always comes first. The safety profile in the study seems really excellent. This is a patient population that can be very challenging to provide treatment to because people are often very sensitive to the effects of drugs. And as was mentioned, there are also concerns about interactions between different medications. And there is just no signal of any of that in this study, which makes me think that should the efficacy be proven, this is a treatment that could actually be implemented and scaled and given to people. And so I really think there's a really promising path forward here for those two reasons.
Thank you, Michael. Joe?
Yes. I think you need to look at safety, as Michael said, that's the most important thing because without that, really, you don't have an intervention. So safety first, of course. But I'm also looking at directionality. I'm also looking at confluence. And I'm also looking at the agreement between subjective, which is how sort of what the patient -- the individual tells us as well as what the computer tells us, in that case, the cognition. And we see this really nice confluence of effects that link fatigue, that link cognition, that link malaise they all go in the same direction. So as Michael said earlier, it's not just one thing that sort of sneaks across the finish line. It's the overall projection.
And if we were to look at that initial graph, that initial graphic where everything moves in one direction, but we don't quite get a statistically significant endpoint among those 21 or 22. That's because we're being incredibly diligent here. We're being incredibly conservative because if you look overall at that pattern, that pattern doesn't occur by chance. It just doesn't. So I like the fact that there -- again, you've got this mix of objective subject the patients feel better. The computer shows that they are thinking better, that they're reacting more quickly, memory improves. And then again, the drug is safe. So I think it's quite compelling. And I have to agree with everything that Michael said here.
Great. So the next question, given there are currently no FDA-approved treatments for Long COVID, does BioVie plan to pursue breakthrough therapy, fast track or other expedited designations bezisterim in this indication?
Pene, would you like to take that?
Yes, we would. We will pursue breakthrough therapy. We believe the results are very compelling. There's a coherent clinical signal here. And most surprisingly, to us, and we did not expect it is that the signal is clear in the most severe population. We did not believe, first of all, that the design of our trial, which required monthly visits would enroll such severe patients. And as we're blinded and watching the blinded data, we saw these severe patients move. Of course, we didn't know if they're in placebo or the drug arm.
And if we look at fatigue alone that the top half with the worst fatigue burden, you're essentially 3x more likely to have a dramatic improvement on your fatigue, almost 1.5 standard deviations than you are in the placebo arm. And we think the whole signal is just so coherent. If you enrich for cognitive impairment, you see movement of statistically significant improvement in the objective cognitive domains. We think it's fantastic data, and we are very excited, and we will pursue breakthrough.
Great. Thank you. So the next question, will the Phase III trial enroll only the high symptom burden subgroups identified here? And if so, how does that affect the addressable patient population, enrollment time line and trial size?
That's a hard question. I'll give that to Joe.
So the answer is we'll certainly bring in the more severe patients. That's clear. We'll also bring in some of the patients who don't fit that because when we go to FDA, FDA is going to want to see that what we saw in Phase II is replicated in Phase III because FDA is clearly interested in the overall population. They want the broadest possible label for the greatest number of individuals. So they're going to ask us to look at this again. And this is not unusual for a clinical trial to show effects in the most severe patients. We see that across neuroscience. We see this in other indications as well.
In part, it has to do with how well you are to begin with and the body's natural ability to heal as well as the basics of biostatistics. I think the hint may be in that initial visualization we show where everything begins to move to the right. That suggests the entire population is getting better. And that's just sort of the nature of biostatistics. So my hope is that FDA will be consistent with what it's done in the past that a signal that shows itself in the most affected patients is also shown to generalize in the full population and everyone ends up benefiting. So that's my hope and expectation.
Great. Thank you. So the ADDRESS-LC trial was fully funded by U.S. Department of War grant. Will any portion of the Phase III costs be covered by that award or other non-dilutive government funding? Or will the company need to fund the trial independently?
Pene, would you like to get started on that?
Yes, we're definitely going to pursue follow-up funding from the CDMRP. We will also look potentially to the NIH recover for funding. We believe this is very exciting. And in parallel, obviously, we will raise money to fund this trial. We -- ideally, this trial will have some element of decentralization where some cohort of patients, the most severe can be treated in their own homes. And ideally, we'd also like to include an open-label extension so that anybody in the placebo arm can roll over into and have an opportunity for treatment with bezisterim and the bezisterim arm. So not trivial. We will pursue. We think it's very exciting data and huge unmet need. We think this is the first really strong signal in this patient population, and we will pursue all avenues.
Great. Thank you, Pene. So the last question here. What are the next concrete milestones from here for the Phase III protocol financing or partnership?
Well, we just unblinded the data. So please give us a little bit of time. So in the coming weeks, we will be working with Michael and other experts we've been working with to design the Phase III approach, right? And this -- we have, of course, started thinking about that. But this is where we have to be extraordinarily thoughtful and double check, triple check everything because the Phase III is the trial where you say this is our endpoint and the only objective here is to get to p equals 0.05 or less. So we believe we can get there, right? But give us a little time to design that, and we will then go and have an end of Phase II meeting with the FDA to completely agree on that endpoint, then we will start. We've had some conversations with other pharma companies. So we -- and they've been waiting for the results. So now we can share the results, and we'll see where those conversations start.
And eventually, we'll go raise some money, but we have no imminent plans right now. We have sufficient funds to last for a while. So we're just going to really focus on right now, on the patient community, on the research community, sharing our data and getting the word out on what this thing could really mean for the patient community. We're really, really excited about this, if you haven't picked it up by now.
I'm particularly excited because this, we believe, is the first therapy that has been able to show a benefit on any of these long COVID symptoms, right? -- not just one, not two, but all three of these long COVID symptoms for a large, large portion of the population here, right? 78% of the patients have at least one severe symptom. And that is how large of a population we believe we can help. So we're very excited with that next step, right? And we're just going to keep our heads down and do the work that needs to be done. With that, let me thank everyone for joining. Let me thank especially Michael and Ezra for joining us and sharing us -- with us in the group on your experience and your comments, right? Thank you very much. And if you -- we wish you a great day.
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BioVie Inc — Special Call - BioVie Inc.
BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Hello. This is Craig Brelsford with RedChip Company. Thank you for joining today's event with BioVie, which trades on the NASDAQ under the ticker BIVI. With us today is Cuong Do, President and CEO of BioVie; Joe Palumbo, Chief Medical Officer of BioVie and Mark Stacy, a key opinion leader and the William E. Murray, Professor of Neurology, Movement Disorders Division at the Medical University of South Carolina. We will begin with a brief presentation in a moment and then we will answer your questions. Welcome to everyone joining us today on X, YouTube, LinkedIn and other social media platforms. [Operator Instructions]
Before we begin, please allow me to read the safe harbor statement. This call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results, along with other statements about the future expectations, beliefs, goals, plans or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact should also be considered forward-looking statements. Of course, forward-looking statements involve risks and uncertainties. Team BioVie, please go ahead.
Thank you, Craig, and thank you, everyone, for joining today. Before I start, I would like to thank everyone who's worked so hard for so many years to get us to where we are today. Our team has worked countless hours, sometimes pulling all-nighters and so forth, weekends, to get us here, right? And it's on their behalf that I'm speaking to you today regarding this. I would also like to acknowledge two people in particular, it's Clarence Ahlem and Chris Reading, who are senior members of our team who literally have spent decades of their life working on this to get us to where we are today, that they are -- we call them affectionately -- the -- we affectionately call them the fathers of bezisterim.
So Craig has -- I will let you read at -- by your leisure. But let's start with Bezisterim. What is bezisterim? Bezisterim is a small molecule that is orally bioavailable. So patients take it twice a day, a capsule in the morning, a capsule at night. It freely crosses the blood-brain barrier, and it's believed to block the activation of something they called ERK and NF-kappa B. When you block NF-kappa B and ERK, you block the production of TNF alpha. And therefore, what you're doing is you're breaking the continuous forward-feeding proinflammatory cycle that essentially is going on. So by blocking NF-kappa B and TNF alpha production, it is also believed that it's blocking the activation of something called IKK and JNK, which is essentially implicated in insulin resistance. IKK and JNK, when it is activated binds to something called the insulin receptor substrate 1 and 2. And when it binds to IRS 1 and 2, it prevents insulin from binding there. So that's the direct linkage between inflammation and insulin resistance. And that's the key to how bezisterim works.
We conducted the SUNRISE-PD Phase II trial to figure out what to do to get the data that's needed to design the Phase III trial. So the SUNRISE-PD was designed as a short 3-month long study. It is a classical signal finding study to essentially identify which endpoint can we get an efficacy signal, right? So which endpoints have the most favorable treatment benefits. Is the measure, the magnitude of this difference to identify the populations where the drug works best for and all of that allows us to design and size the Phase III trial. And that is the critical trial, right? So that's the trial where you basically say, what's the primary endpoint? And did you meet your statistical significance or not? That's the thing that really matters, right? It's in the Phase III trial.
The Phase II trial is signal finding. It's trying to get us the information so that we can do that in the Phase III. So in this Phase II trial, we evaluated a series of motor and non-motor endpoints. We created something called the Early Parkinson's Neuro-inflammation Composite 15, which is the EPNIC-15 and we will go in much greater detail -- much more detail about that as -- over the course of this presentation. We looked at the classical metrics of Parkinson's, something called the Motor Disease Society's Unified Parkinson's Disease Rating Scale. It has 3 parts. Part I deals with non-motor symptoms. Part II deals with activities of daily living, Part III is motor and then you add them up to get the total score.
We also looked at something called the Parkinson's Disease Sleep Survey, number two, at sleep. We looked specifically at sleep because in our prior trial, we saw a signal on sleep. We also looked at quality of life, using something called PDQ-39. In addition, we evaluated a series of biomarkers, and we conducted a -- what's called a proteomic study that evaluated 380 different proteins in our blood plasma. And as part of that battery, we looked at biomarkers of neurodegeneration, we'll come back much more to this, but I especially love NfL, which is neurofilament light and GFAP. We looked at neuroinflammation, and we also looked at peripheral inflammation. So that's what we try to do. That's what this study was designed to do.
And this is what we found. We found that patients treated with bezisterim appear to have experienced a statistically significant treatment advantage compared to those on placebo. We looked at the clinical outcomes. Where we saw significant impact on EPNIC-15. We saw that 283 out of the 380 proteins we looked at moved towards a direction that's beneficial. That's been shown by others to slow the progression of disease.
We looked at different metrics of neurodegeneration, and those moved in a very beneficial manner. And of course, we looked at various measures of inflammation. We also saw that while bezisterim appeared to have an effect across the trial population, we also found that our hypothesis that patients who have higher inflammation at the start of the study would be the ones that benefit the most and would respond the fastest, and that's exactly what we found. Those with higher inflammation at baseline that experienced statistically significant advantages over placebo across all clinical measures that we looked at including UPDRS Parts I, II and III and the total score as well as EPNIC-15. I'll share more of that as we go along.
The biomarker results also suggest that bezisterim may be slowing neurodegeneration over time. This is a big deal if we can show this. And certainly, this is something a hypothesis that we would be testing as we move into Phase III registrational trial. And lastly but not least, bezisterim continues to demonstrate a very safe profile. It was well tolerated. The safety profile was very similar to placebo.
Before I go into deeper, let me set the context for why these data are so important. First of all, we know that Parkinson's is an aggressive neurodegenerative disease. Once you have it, the brain cells will continue to die. Neurodegeneration will continue to happen. Right now, the community largely view Parkinson's as a motor disease, characterized by low dopamine levels in the brain, which then leads to muscle disorders and slowing motor symptoms. But unfortunately, it misses all the non-motor symptoms right now. And since that is such an important part of this, I'm going to dive deeper into non-motor and we will spend a lot of time on non-motor discussions.
We found this chart in a great journal article. So frankly, we just liberally borrowed it. We copied and pasted it here. The green at the bottom discusses non-motor symptoms. Non-motor symptoms include sleep disorders, depression, cognitive slowing, GI symptoms and a lot of constipation. And the important thing to note here is that many of these non-motor symptoms exist for years, many, many years before the actual diagnosis of the motor symptoms. And these non-motor symptoms continue to worsen, and some patients would say that's the non-motor symptoms that could be worse than the actual motor symptoms themselves. And when it's diagnosed somewhere along the way, patients will get put on to therapy and the current standard of care for Parkinson's treatment is levodopa, which is a great drug that's been around for over 5 decades. But treatment with levodopa and similar drugs also create their own treatment complications as well. And the thing to note about this chart is that it all heads upwards.
The disease continued to progress. Things always get worse. It never changes and never gets better. And that's where we believe is the big unmet need. And what we're trying to do with bezisterim and the unmet need that we're trying to address is conceptually depicted on this chart. So the dash line at the top, think of it as the top line on this chart. The disease, if untreated, will continue to progress. But then as you get put on to levodopa, what you're doing is that you are shifting that curve down because you are addressing the motor symptoms of the disease, right? So you're shifting this curve down. But since nothing has been done to address the underlying reason for the disease progression, disease continues to progress.
What we're trying to do is -- and we believe the big unmet need in the community is something that changes that trajectory, something that can slow the progression and essentially bend that curve, right? As I move on, let me ask that my -- others that are in the panel, Mark and [Terren], I'm not sure why you're on the panel, if you can just take yourself off camera, right, and mute yourself as well. Thank you, and Joe as well. Okay.
So we know that bezisterim works on inflammation. So the inflammation, the neuroinflammation hypothesis essentially shows that -- it goes something like this. It's peripheral inflammation that's activated by TNF alpha and NF-kappa B as I showed before, essentially leads to neuroinflammation and oxidative stress, ER stress and so forth in the CNS, which then leads to neuronal loss, which then leads to clinical progression. That's the hypothesis for what's really going on, right? And this hypothesis is partly driven by -- associated with term called Type 3 diabetes that was coined by [Dr. Suzanne de la Monte] from [Brown], who is also one of our scientific advisers, right? She believes that and we totally believe and many other people believe in her thesis that neurodegeneration has an underpinning metabolic reason that essentially starts with insulin resistance and insulin resistance is caused by inflammation.
So what we did in this trial is, again, it's a signal-finding study that is very short and very small, all aimed at getting us the data to design the Phase III trial. So in this trial, we looked at 4 different things. The first is inflammation. So this is really our primary endpoint is inflammation. Does bezisterim has an impact on inflammation? We also looked at what's happening in the -- with biomarkers associated with the disease. The third thing is we spent a lot of time looking at neuronal injury, things such as neurofilament light and GFAP. NfL and GFAP are known biomarkers of neurodegeneration. So they naturally increase in our blood samples as neurodegeneration progresses. And we're asking the question, can bezisterim have an impact on the progression of and the release of NfL and GFAP. And of course, we look a lot at clinical scales.
Before we dive into the numbers, let's spend a minute to understand what the numbers mean. We look at the numbers and we look at it for two things. One is just how big of a treatment effect is there. So how big of a difference is there between the treatment group and the placebo group. And statistics has a tool to allow us to do that, to understand that. And that's called the Cohen's d that measures the size of the difference, the size of the effect. Cohen's d of 0.5 is a really good, it's a moderate size impact difference, a 0.8 is a large impact, anything beyond 0.8 is just absolutely massive and huge. And the way to all the numbers are arranged is that a negative value means that's a treatment benefit, right? And as you will see, most of bezisterim's treatment effects are in the large range. So the first thing, treatment effect, how large is it?
The second is what's the likelihood that, that could be a fluke or coincidental. So that's why we looked at the p-value. The smaller the P the better, right? And statistical significance is defined as having a p-value equal or less than 0.05. So that's when we say something is statistically significant. It means that it had an impact, and it has a p-value equal or less than 0.05. So let me start by just showing the baseline characteristics.
We had 57 patients enrolled in this trial. Roughly half were in each of the arms. The bezisterim arm had 28 patients, the placebo arm had 29. And I'll let you read through this at your leisure. This is available at our website. But as you can see here, the treatment and the placebo arms appears to be balanced at baseline. They were very similar. And while our primary endpoint was inflammatory biomarkers, everybody wants to talk about the clinical outcomes because this is Parkinson's. So let's start there. So in the entire treatment population of 57 patients. We looked at EPNIC-15
and the MDS-UPDRS scores. And across the population, what you see is that patients treated with bezisterim are shown in blue, has a smaller change from baseline, right? Here a positive number means people are getting worse.
So what this shows is that patients treating -- treated with bezisterim are getting worse at a slower pace than patients that are treated with placebo, okay? And in the entire population, if you look at the p-values we did not get statistical significance on these different p-values. But this is not uncommon. This happens in the majority of Phase II trials. And remember, the second -- as I mentioned before, the purpose of the Phase II trial is to find the end points where the drug could work and with which populations that it could work.
Now we -- I mentioned before, we have a long-held -- have long had the hypothesis that patients with higher inflammation levels at baseline will be the ones that will respond the fastest and the ones that will respond with the greatest impact and that's exactly what we found. The hypothesis was true, turned out to be true. What we found is that patients with higher baseline inflammation as measured by platelet levels that are greater than 230 at baseline, 230 frankly, is we chose that because it's the median. The median platelet counts for the population was 233. So 230 was a convenient number. Half of the population study -- in the study had higher platelets. And what you will see in this chart is actually quite startling.
You see that patients to have higher inflammation not only had a slowing -- did not have a slowing or worsening. They improved, right? Patients treated with bezisterim improved over the course of 3 months of treatment. On the Part I score that's looking at non-motor symptoms, Part II score that's looking at activities of daily living and Part III score, which is the motor score. And historically, this is the metric that the FDA uses to approve a Parkinson's drug. And of course, when you add it all together, they improved on the total score as well. And if you look here, the statistical numbers are very high, very high, right? And of course, when you look at the EPNIC-15 and we will come back to EPNIC-15 later. The p-value here is highly, highly, highly statistically significant, right?
And before you may start thinking or concluding, well, maybe bezisterim doesn't work for patients who have low platelets or low inflammation, that is not the case at all, right? And you can see that on the next page. This page shows the entire population of 57 patients, arranged by their platelet count at baseline. And what you see on EPNIC-15, on Part I score, Part II score, that slope is very flat. And the vast majority of the patients have the numbers below 0. This is -- we took the bezisterim results, subtracted out the placebo results. And if it's below 0, it means it favors bezisterim. So what you can see is about 80% of the patients regardless of their inflammation level benefit from bezisterim treatment.
On Part II, that's for Part II -- so Part II, it's even higher than that. It's only when you get to Part III that you see a slight slope. And here, you see that more -- about half of the patients benefit from bezisterim treatment. So what does all of this mean? It means that we now have what we call an enrichment strategy for designing the Phase III trial because we want to go into the Phase III and be assured that we can win statistically on Part III score because that's most likely what the FDA will want us to demonstrate at a minimum, right.
So at a minimum, what that means is that when we go into designing the Phase III trial, we will enroll just more patients with higher inflammation with -- and perhaps, we will stay with platelets, but perhaps we will go with another metric of inflammation, but we know that higher inflammation patients are the ones that respond the most and the fastest. So we will enrich the population, a sample for the Phase III trial. That means we will over-enroll patients with higher inflammation so that at the worst case at a minimum, we will win statistically on Part III. And of course, we will have the conversation with the FDA about including Parts I, Parts II and perhaps even EPNIC-15 as part of our end points, right.
Because given this data that you see on this page, we are highly, highly, highly confident that we have a drug that we can go and discuss with the FDA with a trial design that would allow us to get approval at a minimum on motor symptoms by improving motor symptoms. But the data here would suggest that we would be able to have a win on non-motor symptoms as well. So what that means when you step back from all of the numbers is that we believe that bezisterim can become the first drug that could have an impact on both motor and non-motor symptoms of the disease, and that will be a drug that will uniquely meet the currently unmet need from -- in the community, right.
So takeaway number one, potentially the first drug to address both motor and non-motor symptoms of the disease. But we did not stop there. We have said that all prior drugs have been approved largely on motor symptoms, right? The Movement Disorder Society, the name itself says it all. So when you look at the last 10 drugs that were approved, 6 of them were based primarily on daily off time, which looks at how -- what portion of a day, how long the time during the day is your muscle in the off mode, so you can't move, they are stiff. Two of the last 10 approvals were based on Part II score, right? So all motor endpoints.
We know that bezisterim affects muscle function. We know that from our prior clinical trial, our first clinical trial in Parkinson's. We also know it from this trial, right? But if you use only motor endpoints, right, you're not getting at what we know to be a much broader clinical impact that bezisterim could have, and you would be undervalued what could be the first therapy for both motor and non-motor. And this is where we learned along the way.
We started this trial in April of 2025. And it was earlier this year that we learned about something that Biohaven did. Biohaven is a great company. It has a great terrific management team that I personally have a great amount of respect for. And they are known to be working in Parkinson's right now. They started this fairly recently, and they are also known to be looking at the mechanism of having to do something with inflammation. So Biohaven worked with Broadstreet and Pentara. Pentara is perhaps the leading biostatistician in the industry. We have been working with Pentara for many, many years. So they work together to create and publish on something that they called the Parkinson's Composite Scale or PARCOMS, right. Which is their effort, I believe, to see if with one metric, they can get a measure of both the impact on motor and non-motor. So frankly, we just borrowed a page in their playbook. We frankly just copied what they did.
We worked with Pentara to create the EPNIC-15 to include these 15 different domains of Parkinson's. These were prespecified. We hypothesized that these are the ones that bezisterim can most impact based upon our understanding of how the drug works. Therefore, that's where the EPNIC-15 comes from. We did it because we wanted a measure, a single measure that can assess both motor and non-motor impact of the drug. And what we found was startling.
Let me first focus your attention to the left-hand side of this chart. This chart plots what happens to each individual patient. So each patient is a bar. It plots the change from baseline to see the patient improve as shown by a negative number. That they stay stable, stay the same, shown by a 0, or that they worsen as shown by a positive number.
Now let me focus your attention to the left half of this chart. What you immediately see is the majority of the patients that improved or remained stable are the ones that are on drug treatment. They're on blue lines. And remember, this is a neurodegenerative disease. So if we can help the patients slow the progression, prevent them from worsening, prevent them to be -- allow them to be stable with a 0 score that is a win in our book.
Conversely, if you look at the right-hand side of this chart, you see that most patients that worsened are the ones that have -- that were on placebo. So as a result, patients that are on bezisterim collectively as a group led to an EPNIC score of a negative 0.04. The placebo group had a positive 0.18 and look at the Cohen's d, look at the treatment effect. It is huge, right? And as a result, the statistics here is highly, highly, highly statistically significant, right? Showing that patients treated with bezisterim had an advantage.
Another way of looking at this data is to look at the right-hand side. Here, you see that 25% of the patients treated with bezisterim meaningfully improved over the course of 3 months compared to only 6% of patients on placebo having meaningfully improved. Conversely, if you look at the other end, you see that only 14% of the bezisterim treatment group worsened meaningfully over the course of 3 months, whereas 46% of those on placebo meaningfully worsened. When we look at this data, we interpret it to mean that patients treated with bezisterim experienced a statistically significant and clinically relevant improvement or advantage over the dose on placebo on both motor and non-motor outcomes, right?
So let me now move on to the biomarkers. We conducted a study that looked at 380 different proteins found in our blood plasma. Out of that battery, there were 7 that were specific to Parkinson's disease. And we found that all 7 of them move in a beneficial manner, right, that helped that contributed to the slowing of the disease. So statistically significant. We looked at 36 different biomarkers having to do with neuronal injury and over 90% of those biomarkers moved in a beneficial manner, highly statistically significant. There were over 150 different biomarkers of inflammation. And over 75% moved in a beneficial manner but also highly statistically significant. And we named some of those on the right-hand side here.
But let me call your attention to the last 2 rows. Here, we did not get 0.05 statistical significance, but we did get what in statistics is known as trending, which is also very good, right? We saw advantages on a beta amyloid -- beta 42 and pTau-127 (sic) [pTau-217], which are known biomarkers of Alzheimer's. Some of you may know that we have conducted an earlier Alzheimer's study that was longer in duration was 6 months long and saw a signal that bezisterim was having an impact on beta and pTau there as well. And we are very excited to pick up that same signal here in just -- after just 3 months of treatment.
We, of course, want to dive deeper into neurodegeneration, right? What we found is that in the group treated with -- the bezisterim-treated group, we look at particularly NfL, I especially like NfL, because this is a known biomarker that the FDA has used as the primary endpoint to approve 2 drugs already. One for ALS and one for MS and other is GFAP. And what we found in this trial is that initially that these biomarkers do go up, right? And frankly, we don't know how to explain that yet. No one really knows how to explain that yet. But this is consistent with what has been reported by other studies that test these kinds of drugs. They go up. And in other studies, they slow, they increase, they may be stable. But what we see in our trial, especially with NfL is we see a very rapid statistically significant decline, right. Suggesting that we are slowing the release of NfL, namely we are slowing neurodegeneration.
So we also see the same trend for a-beta and pTau, where we are slowing the increase. And so when you put it all together, you see the composite line in blue. We replot that composite line for the bezisterim treatment on the right-hand side and we add to it a composite for all the placebo patients, and you see a very stark difference. The bezisterim treated patients saw a decline from baseline on these neurodegenerative biomarkers, whilst the placebo patients saw an increase. The effect size is good, a statistically significant finding.
We also found the information that basically show us mechanistically how that -- how the drug could be working. But in the interest of time, let me move on, skip this. I would just close with the safety information. What we found was the drug continues to be very safe. There were no serious adverse events in the trial, no severe events and certainly 0 deaths. There were only 2 treatment-related AEs, one in the treatment arm and one in the placebo arm, right? And in the treatment arm, that AE was very mild and the patient continued on to the study.
So let's put it all together and see what does this really mean. First of all, the drug has an excellent safety profile. In this trial, it is consistent with everything we found to date. Bezisterim demonstrated clear target engagement, has an impact on inflammation, has an impact in the proteomic study, the 380 panel and it has an impact on neurodegenerative biomarkers. There is apparent clinical impact on both motor and non-motor symptoms, has a favorable biomarker for disease modification, right? And it gives us clear rationale for the design for the Phase III.
The way that we're thinking it right now is that we would have 2 phases to the Phase III. The first phase would be a 6-month long study that gets at symptomatic relief. We would use most likely Part III score as the primary endpoint and if we use the enrichment strategy that I talked about before, that's how we ensure ourselves a win, a win on statistical significance. But we will also put in there the non-motor symptoms as well. So 6 months the primary endpoint of symptomatic relief. And with the data at that 6-month point, we can go to the FDA and seek approval for registration. But at the same time, with that same patient, we would extend for another 12 months to see if we can get an impact on disease progression. So can we show that we're modifying the progression of the disease.
Lastly, we are not all about Parkinson's. We are very excited at Parkinson's. Of course, the results here are phenomenal is beyond what typically would be expected to get from a Phase II trial. But we also have another trial underway that's finishing, if that's our long COVID trial. And if everything goes according to plan, we should be able to report out the data of that in the next 30 to 60 days.
We now need to start planning for an end of Phase II trial with the FDA and whereby we would discuss with them the Phase III trial design. So we need to plan, work with our experts to plan the Phase III, start planning for the trials and so forth. And we are -- right now, we have looked maybe a the tip of the iceberg and how much data do we have. We need to continue analyzing the data, prepare for presentations and conferences and continue to submit these to various scientific journal, right? And we will provide additional feedback and readouts over the course of the coming weeks and months as we learn more. And of course, everybody wants to know what's happening with partnering conversations. We will reactivate those in due course now that we have data, right, which is what these -- the larger pharma companies have been waiting for.
So in summary, let me just recap that today, we have discussed the top line results for Parkinson's. And we believe bezisterim could become the first treatment in Parkinson's that could address both the motor and non-motor symptoms of the disease. We believe bezisterim can become the first treatment to slow the progression of the disease and may become the first -- a new approach to treating Parkinson's since levodopa was introduced over 5 decades ago. And if we're able to demonstrate that into Phase III, we believe this will be easily a $3 billion to $5 billion annual sales product in the U.S. alone.
We did not talk long COVID. The long COVID is coming to an end. We believe bezisterim may become the first treatment for long COVID and given that it affects so many more people that could become a $10 billion annual sales product in the U.S. alone. So with that, I would like to stop my screen share. I would like to ask Dr. Mark Stacy to come on screen as well as Joe Palumbo, to come on screen. And Mark, let me turn it over to you based upon what you've heard me describe here, what is your takeaway? What's -- what are your thoughts about what you've heard?
Well, thanks, Cuong. It's always exciting to think about these data and the more chance I get to look at them the more excited I get. From a more historical perspective, I remember starting my fellowship with Dr. Joe Jankovic, a very famous movement disorders person. And he had just published a paper looking at tremor-dominant versus postural instability and gait disturbance Parkinson's disease. And the scientific world had erupted with disagreement. There cannot be 2 kinds of Parkinson's disease.
Now we fast forward for another 40 years, and we're talking about Parkinson's disease as synucleinopathies as alpha-synuclein degenerative disease. And there are statements that occur that we will find hundreds of different causes of Parkinson's disease. And this study underscores that. You've looked at more than 400 biological markers. And in theory, all of them could play a role in a particular type of Parkinson's disease. What you've really found is this platelet marker as a way to identify and enrich this population. And those data are striking.
They're striking from a potential neuroprotective standpoint and from a potential motor control standpoint. So I am all in on this. I think this is a very interesting molecule that's going to generate hundreds of new thoughts about how we treat Parkinson's disease, and you're ahead of the curve.
Well, thank you, Mark, for those comments. Now with that, Craig, let me turn it over to you, and let's start the Q&A. I'm sure there must be a lot of questions from the audience.
Yes, there sure are, Cuong, and thank you for that excellent presentation. [Operator Instructions] We have many of those, Cuong. Here's the first one. You mentioned that the results allow you to have an enrichment strategy. What does that mean when designing clinical trials?
Thanks for that question. And that, of course, is a key part of what we will be focusing on now as we design the Phase III trial. Enrichment simply means we over-enroll a population that we think the drug will work for, right. So let me give you an example, and I'm just going to use round numbers. This is purely, purely hypothetical. So please do not take it for anything more than it is. So if we were to enroll 100 patients in a trial, the data that we saw here would suggest that about half of them would have the higher inflammation and about half of them will be lower inflammation. And since we know that bezisterim works faster and more -- with bigger impact for those that are in higher inflammation. And since we want to have an insurance policy to make sure that we win on Part III scores on the statistics, we would over enroll.
We would, just let's say, enroll another 25 patients. with higher inflammation. And that's all it really means. We will over enroll the patients that meet the characteristics that we have here in this particular case, inflammation. I hope that answered your question.
What does the Phase III trial design look like? And will the FDA agree to that?
Sure. I somewhat alluded to that before. Let me -- Joe, let me start and then you can chime in to see and build it up. We are thinking that the agents -- the regulatory agencies, of course, are very conservative. They'd like to do the things that they have done before, right? And all Parkinson's drugs have been approved based upon motor symptoms, right? And that essentially comes down to Part III scores, sometimes Part II and III, but majority of the time, it's Part III score. So we don't want to stress or put anything at risk. So chances are we will have our primary endpoint as Part III score.
But the FDA also knows that there is huge unmet need around non-motor symptoms, right? And that's been well known. In fact, the Parkinson's Foundation conducts an annual survey of their community. And every single year, non-motor symptoms comes up to be the #1 or the #2 unmet need in Parkinson's. So of course, we will go and make the argument that Part I score, Part II score, the sleep score and perhaps even EPNIC-15 should be part of our endpoints as well, right? And so those are early symptomatic endpoint for the first 6 months of the study.
Notice we are extending the study from what we just did here from 3 months to 6 months. Again, that is an insurance policy to make sure that we're able to see bigger separation between those that are on placebo, and those that are on drug. Remember, it's neurodegenerative. So the longer you are on placebo the more worsening you are going to experience. And the longer you're on drug chances are the more benefit you're going to get. So even widening benefit between the groups. That's the 6-month symptomatic input that we can use to go to get approval, to seek approval. As we end the study at 6 months, that doesn't mean we stop.
We will extend with those very same patients for another year, perhaps, right? And we can design it so that we can stop whenever we get the results that we won. And that the results there, of course, is to show that there is for having an impact on slowing the progression of the disease. And there are a number of different ways that, that could be shown, and that's we are working with our experts in that. So clinical trial, primary endpoint around symptoms at 6 months, extend that for another 12 months to get disease modification. And we just do not believe there would be any reasons why the FDA would disagree with that because that is a very classical design. Give them what they seek at a minimum, Part III score, give them beyond that and they would love that, and they are there to help the patient community as well. So hopefully, that answered that question. Joe, anything to add?
I think you've covered it. The wonderful thing here is you have 2 activities. You have what we've seen to be improving in symptoms. And we also have the ability to slow the disease, at least we hope so, right? That's what the biomarkers suggest. That's what this data suggests and this is what we'd have to prove in the next study.
Thank you, gentlemen. The neurodegeneration data look really interesting. What needs to be done in the Phase III to get a disease modification claim?
Yes. I think I touched on that a little bit. It's -- we would need to extend the study for 12 months or so beyond the first symptomatic relief endpoints. And the longer we extended out, we believe that there will be even greater and greater separation. We will be able to show that patients that are on treatment, one, hopefully, we'll see a benefit on the biomarkers of neurodegeneration of GFL, NfL and GFAP, right? But the FDA loves biomarkers, right? So we will absolutely track those. Then the other, of course, is what's happening to them on MDS-UPDRS parts I, II and III and EPNIC and anything else that we can show. And if there is a tight correlation between biomarkers and disease progression, that is a home run. If we could just show a change in disease modification to the trajectory over time, that is a big deal as well, right? Mark, Joe, anything to add on that?
I think in the second phase of the study the most important thing is to see the change in slope in the active study group. And so in that slide you showed where you were just treating a patient like levodopa did, you didn't see a change in slope. Patients continue to progress, but they had an improvement in symptoms. With bezisterim, you should see a change in the slope to identify disease modification.
Terrific. Thank you, Mark. Right, let's go to the next question. Craig. Craig, can you give us the next question?
Excuse me, I was muted. How can one contact the doctor doing a physician-initiated study of bezisterim?
Great question. Thank you for that. Let me first explain what -- an IIT first investigator-initiated trial. The FDA has many different kinds of clinical trials that they would consider and approve. This is -- what we just saw today is your classical what's called sponsored trial where a company would conduct a trial, right? But there are trials where a researcher can initiate himself or herself, right? So if Mark wanted to go and do a clinical trial, he can go and initiate a trial himself. And we believe that bezisterim has a much broader impact than what we, as a small company can study by ourselves.
So we have put in place a program where we would support investigator-initiated trials, where if an investigator comes to us with a plausible hypothesis for how this patient could benefit from our drug, or if a patient is -- has a condition that we are studying, but doesn't qualify for a clinical trial. We could support that investigator and they would need to submit a protocol to the FDA and get authorization from the FDA.
They would need to get approval or by what's called an IRB, right, that looks after safety of patients in trials. So they have to do a number of other things. But if they cross all those hurdles, we would support the trial by giving them drug, free access to drug to see if the drug works for patients. And we have done this for a number of different indications.
Frankly, the first study that we did in Alzheimer's was an IIT, we have supported a trial in traumatic brain injury. Some of the early data that we have in long COVID come from IIT. With that said, there is not a simple place that you can go and look "Oh, these are the clinicians that are doing IIT." So that just doesn't happen. I can just -- at this point in time, I can just tell you that there are IITs underway are being considered in the New York Metropolitan Area. And then there's one in the San Francisco Metropolitan Area. And I believe there's one in Miami area as well. And a few are in that area -- those areas. And if you have something perhaps in Parkinson's and so forth, reach out to us, and then we will endeavor to -- we will ask those clinicians if they are open to having a conversation and if they are, we would endeavor to connect you. But that's the only way to do it. Or if you have your own -- if you have a clinician that is interested in initiating a trial, we would entertain and have that conversation as well.
If I can add something, one of the great byproducts of this Phase II trial is the company learned how to recruit patients. And you work closely with websites. You work closely with the Davis Phinney Foundation and the Parkinson's Foundation and you figured out how to recruit patients rapidly and in the right phase. You also figured out and demonstrated that patients could be seen in their homes versus traveling to long distances to be seen in an office and that's one of the more exciting pieces of data that we will look at, because I think you will prove that it is cheaper to see patients in their home, and we already figured out how to collect data. So if the questioner is worried about Parkinson's disease enrollment, you have figured that out, and it will be easy for patients to seek enrollment.
Yes. Next question. I was in the trial IIb study. What's ahead for me?
Oh, Joe, that's a hard question. I'll let Joe answer that one.
So number one, talk to the clinician, the trialist with whom you were treated. I can't know your name, all right? That's all blinded to me. Our hope is that we'll be able to provide drug to people who've been in this study. That requires a discussion with FDA, but it starts with you talking to the individuals who treated you and making yourself known to them when they would let us know. So yes, that's what we would have to do.
Is it reasonable to assume that you may have an answer for long COVID before Parkinson's?
That's a very hard question. And the short answer is a maybe, is maybe. The reason I say that is the following. That trial, hopefully, will read out in the next 30 to 60 days. And if we get -- and we will go into the FDA for an end of Phase II meeting in the fall or later on this year. If the result is like phenomenal right? Phenomenal. I don't know what that means, but if it's phenomenal, right? It could be the first therapy for long COVID, which is a condition that affects 17 million to 20 million of U.S. adults, 4 million of whom have it so badly that they've had to quit or change their job simply because they cannot keep up the physical demand of the job. They are essentially on disability. So huge unmet need. So if we get phenomenal results, knowing the big unmet need, we may go and ask the FDA to consider an accelerated approval and perhaps an emergency use authorization.
The chances of emergency use authorization is very small, right? Let me be very clear. That is a small chance, but we have to ask, right? And if we get any of that, the other -- the beauty about long COVID is that we know how to recruit that very, very rapidly. And it's likely to be a short trial. Is it 3 months to primary endpoint instead of 6 months for Parkinson's. And since we are the only company that's testing a new drug in long COVID, we benefited from the support of many of the leading academic medical centers out there. So places -- medical centers that have long COVID sites. So places like Yale, Mount Sinai, Northwestern, Mayo Clinics, Stanford, UCSF, they all worked very closely with us, and we were able to very rapidly enroll 200 patients in that Phase II trial. And so I think we would -- could be counting on their support again when we move to Phase III.
So when you take all of that together, there is a possibility that long COVID could get to registration before Parkinson's. But time will tell. That's purely speculation on my part, the answer I just gave.
Next question. Given that bezisterim targets many of the same neuroinflammatory pathways involved in traumatic brain injury, do you envision bezisterim being tested for the treatment or prevention of the destructive neurochemical sequelae of TBI?
That one is yours. Joe?
So yes, we -- we're impressed by it as well. So that is a good observation and good science. We'd like to get there. There are a number of other targets we'd like to get to as a very small company right now. We have to stay focused on what we're focused on. but that's something we've already looked at briefly in an investigator-initiated study. So the answer is yes. You're spot on.
The person who had been in the trial has followed up. How do I get in touch with the clinician to continue? I can't emphasize how important this is to me. I have a feeling I got the drug and want to continue with its progress.
Okay, very quickly. I'm assuming then that you were seen at home because that would be how you were managed. The best way is to contact the organization that had done the study with you. So that would likely be the people who came to your home and have them follow up. If it was from a clinical trial site, you would need to contact them. And if all else fails, talk to your neurologist and have them contact us on your behalf.
We're not trying to pass the buck. No, not at all. I -- just not allowed -- we're just not allowed. We are just not allowed to know who the people are. right? So that's why it always has to work through your clinician or the organization to help with the clinical trial.
So we care very much. We really do, but I just can't. Your identity can't be known to me. That's the only reason why.
On Slide 14, the MDS-UPDRS Part III panel shows bezisterim roughly 4 points worse than placebo below about 250 platelets. Will low platelet patients be excluded from Phase III? And how are you treating that as a safety consideration?
Short answer is low platelet patients would not be excluded. We will enroll everybody, the entire spectrum because we believe over a 6-month study, we will win on that. And we don't believe that there is a safety concern looking at platelets. So I think that's -- we will take everybody, but we will enrich the Phase III sample by enrolling more -- higher inflammation patients to the group as well.
And we have time for a final question, here at the 1-hour mark. My understanding is that bezisterim statistically reduced biological markers of neuroinflammation across all groups and platelet counts in this study so that even subjects who did not show improvement on observable physical measures, nevertheless, showed reduced statistically significant neuroinflammation. Is this correct? And if so, can bezisterim potentially slow down the progression of disease even in these patients, as would be demonstrated by a longer study?
Joe?
That's exactly the right question to ask. Your observations are our observations. We have biological engagement and processes that mean a lot. We also showed improvement in certain patients more, maybe on the Part II and the Part I at lower platelet counts. But putting that aside, the findings with NfL, with neurofilament light are extremely encouraging. And your question is our question. We think probably so. But that's -- we'd have to test. That is exactly what we have to test.
All right. So with that, since we are out of time. Let me just bring the call to a close. Let me thank everyone for joining today. And let me just recap by saying that we are extraordinarily excited about the results that were -- that we -- that came out of this trial. The results suggest that bezisterim is having a statistically significant impact for -- on both motor and non-motor symptoms of Parkinson's. It is showing an impact on neurodegeneration, right? Thereby, we have a hope of being able to show that we can slow the progression of the disease, and it's having an impact on all these different inflammation biomarkers.
We have gotten far more out of this Phase II trial that one would expect to be able to get from a small and short trial. And we certainly have data that we need now to go and design the Phase III trial, right? So our next step is to design that Phase III trial, request an end of Phase II meeting with the FDA sometime very soon, and we'll go in and have that conversation, and we will give further updates over the course of the coming months on additional clinical -- any additional insights that we find from analyzing the data furthermore. And of course, any feedback from the FDA, we run the Phase III trial design. We will make available when the time comes. Thank you very much, everyone. Have a great day.
Yes. Thank you very much, Cuong. And for more information about BioVie, reach us at 1-800-REDCHIP or e-mail us at [email protected]. Please visit RedChip's Investor Information page for BioVie. It's bivinfo.com. There, you can view and download the investor presentation and fact sheet and sign up for news alerts on BioVie. Finally, join RedChip's next webinar with Comprehensive Healthcare Systems on Tuesday, August 18, at 11:00 a.m. U.S. Eastern. Register for all RedChip webinars at redchip.com/events.
Thanks again to the many participants today, and thank you, Cuong, Joe, and Mark.
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BioVie Inc — Special Call - BioVie Inc.
BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Hello. This is Craig Brelsford with Red Chip Company. Thank you for joining today's event with BioVie, which trades on the NASDAQ under the ticker BIVI. With us today is Cuong Do, President and CEO of BioVie. We will begin with a brief presentation in a moment, and then we will answer your questions. Welcome to everyone joining us today on X, YouTube, LinkedIn and other social media platforms. [Operator Instructions] Before we begin, please allow me to read the safe harbor statement. This call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results, along with other statements about the future expectations, beliefs, goals, plans or prospects expressed by management constitute forward-looking statements. Any statements that are not historical facts should also be considered forward-looking statements. Of course, forward-looking statements involve risks and uncertainties. Cuong, please go right ahead.
Thank you, Craig, and thank you, everyone, for joining this afternoon. My name is Cuong Do. I'm the President and CEO of BioVie. At BioVie, we have 2 assets. Our lead asset is a drug called bezisterim, which modulates the production of TNF alpha. So in clinical trials, bezisterim has shown that it can reduce inflammation and reverse the associated insulin resistance. Parkinson's patients have been seeing improved muscle control, Alzheimer's patients have seen a 68% slowing of cognitive decline after just 6 months of treatment, and everyone have seen lower levels of DNA methylation, which essentially is a modulation of the biological aging process. Our second asset is a drug candidate named BIV201, which has the potential to be the first therapy for ascites, which is a terrible end-stage liver disease condition that has greater than 50% mortality within 12 months. We have 2 very near-term catalysts. The top line readout for our Parkinson's trial is now expected in the summer of this year, right, so in the next month or 2. And then our long COVID trial should be -- is expected to have top line readout in late summer and perhaps early fall. And as our company, we are all about inflammation and insulin resistance, right? And how that starts is really in Parkinson's. We believe that the current perspective on Parkinson's is too limiting, too limited. The medical community currently view Parkinson's as a disease that's characterized by loss of muscle control and driven by insufficient levels of dopamine in the brain. That is required, of course, but we believe that is too narrowly defined and misses on 2 critical dimensions. The first is that it ignores the non-motor symptoms that Parkinson's patients have. So for years before the actual diagnosis of Parkinson's, these patients start to experience non-motor symptoms that has to do with sleep disorders, anxiety, depression and cognitive slowing, such that by the time the motor diagnosis is made, over 90% of patients already have experienced these non-motor symptoms for years. And unfortunately, these non-motor symptoms are currently just ignored because very people pay attention to it or believe nothing could be done, right? And of course, the current Parkinson's therapies are not able to address these non-motor symptoms. The second reason is too narrowly considered is that it disregards the underlying metabolic drivers for the disease that essentially starts with inflammation and insulin resistance. And we believe that future treatments should address both motor and non-motor symptoms, and we believe it also needs to address the underlying metabolic drivers, right? The metabolic driver starts with insulin resistance because it's been known for decades now that 2 conditions need to be present at the same time for someone to develop Parkinson's symptoms. The first, of course, is low dopamine levels, but the second is insulin resistance. But if you're able somehow to reverse the insulin resistance, the body adjusts and has been able to make use of the dopamine that's there. But unfortunately, over the decades, nothing has been done to be able to reverse the neuroinflammation and the associated insulin resistance in the brain. As such, all treatment approaches up until now has been to increase the level of dopamine so that you can restore muscle control through the use of a drug called levodopa. Now levodopa is a terrific drug, and that's why it remains the standard of care 5 decades after it was first introduced. It's a terrific drug in helping Parkinson's patients restore muscle control. But it does have limitations, one of which is that it has a very short half-life. And that means patients end up having to take it 2, 3, 5 times a day, and that could become problematic overnight as you sleep because as you sleep, the drug wears off. And when you get to the morning, Parkinson's patients can't move. They can't get out of bed. And that's the reason why Parkinson's patients do not like to schedule anything first thing in the morning because when they wake up, they take their morning medication, wait an hour and bed for the medication to kick in before they can actually start to move and get out of bed, right? So that's one very significant limitation. But perhaps the most dire limitation is that Parkinson's fundamentally is a neurodegenerative disease. That means the longer you have the disease, more and more of your neurons essentially have died away. So the way to address that is you actually have to end up taking higher and higher doses of levodopa. But as you get into higher doses, it leads to something called levodopa-induced dyskinesia, which is the uncontrollable jerky motion that you see Parkinson's patients have. And when you get to dyskinesia, all you can do is reduce your levodopa dose. And as you cut back on the dose, you lose muscle control, right? So it's a terrible situation that Parkinson's patients have, but it's a situation that we believe bezisterim can help address because in preclinical studies in rodents and in nonhuman primates in monkeys, we found that bezisterim alone was equally effective as levodopa in restoring muscle control. Now that's a huge statement in and of itself because no other agent has been able to match levodopa in its ability to restore muscle control over the decades. But what we also found is when you use bezisterim in combination with levodopa, you get a synergistic effect. You saw maximum muscle control. This is being a disease score, a lower score is better. So this is better muscle control. But what got us and our experts most excited is that at the end of the study, when we sacrificed the monkeys and looked at their brains, we found that those who were treated with bezisterim and levodopa retained twice as many neurons as those who were treated with levodopa alone, suggesting that there is a neuroprotective property in the drug. And that to us is not at all surprising because we know bezisterim reverses insulin resistance. That means there's greater glucose available in the brain for neurons. We also know that bezisterim enhances blood flow to the brain, and that means there's greater oxygen available in the brain. So when you have greater oxygen and glucose availability, that only bodes well for better neuronal health. So based on this information, we went into the clinic and enrolled 40 moderate to severe Parkinson's patients and essentially replicated in humans the study that we did with the monkeys. We enrolled half the patients in an arm that gave them a placebo and levodopa, so essentially levodopa alone as shown in red. The other half were given the combination of bezisterim and levodopa as shown in blue. And what we found essentially was the same as what we saw in the monkeys. Essentially, those that were given the combination had significantly greater muscle control than those that were treated with just bezisterim alone. In addition, we found that roughly 1/3 of those that were given the combination had their muscle in what's called the on state first thing in the morning, meaning that they had control of their muscles. They can get out of bed first thing in the morning before they take the morning medication, whereas none of those that were given just the levodopa alone had the ability to move first thing in the morning. The other thing that we found was interesting is that we started to see a signal that bezisterim has some effect for non-motor symptoms, namely these 40 patients, a number of them told us that they actually had better sleep. They had actually better anxiety and depression scores and so forth, right? So here, with this study, we have established human proof of concept that you can use bezisterim in combination with levodopa to help moderate to severe Parkinson's patients improve their muscle control. And this establishes the first half of what we want to do in Parkinson's. And we are now concluding a study to establish the second half of what we want to do, which is to use bezisterim alone with Parkinson's patients at a much earlier stage in their disease. We enrolled 60 patients who are needing to go on to therapy for the first time to address their symptoms. Half of those patients were given a placebo and the other half were given bezisterim. And the trial aims to show that bezisterim can help these patients address both their motor and non motor symptoms, right? This trial has completed the patient-facing portion. The last patient has already come in for his or her last visit. And we are now in the process of essentially cleaning the data, waiting for biomarker results to come back in the labs, which is frankly the thing that's taking the longest time. And once we have all the data, we'll lock the database and start the analysis of the data. So if everything goes according to plan, we hope to announce the results of this data within the next month or 2. Let's call it sometime in late July, early August is when we hope to have results from this Parkinson's trial. And once fully developed, we believe that bezisterim could become the first therapy -- first new therapy in Parkinson's in over 5 decades. It has the potential to be the first therapy to address both motor and non-motor symptoms of the disease. And it has the potential to be the first therapy to potentially modify the progression of the disease, namely slow the progression of the disease, right? And when fully developed, this, we believe can become the $3 billion to $5 billion annual sales opportunity in the U.S. alone. So let me now move on to Long COVID, right? And you may ask -- be asking yourself, why are we talking about long COVID and what does this have to do with Alzheimer’s and Parkinson's. And the honest truth is we had no idea ourselves when we started on this journey. It was our experts who helped us understand the situation. I've had long COVID -- I had COVID now 3 times. And I kept myself among the lucky majority of people who have COVID, namely when we had the infection, our immune system kicked in, got rid of the virus, the body returned to normal, life went -- goes on as usual. And that's why the whole world essentially thinks that COVID is long behind us, right, despite the fact that there are 30,000 people who are still getting COVID each day right now. But there are 17 million Americans who do not have it so lucky. So these 17 million continue to suffer the lingering after effects of the infection that shows up as brain fog, fatigue and severe post-exertional malaise. 3 million have it so badly that they've had to quit or change their jobs simply because they can no longer keep up with the physical demands of their jobs. Many of them essentially are on disabilities. And within the last couple of years, researchers have tied these long COVID symptoms to inflammation that works through something called the TLR 4, the toll-like receptor 4, which leads to the reduction of TNF alpha, which, of course, is exactly where our drug [indiscernible] affects. And what's really going on with these patients, what's believed to be going on with these patients is that after their immune system got rid of the virus, they still have fragments of the spike protein and the envelope protein continuing to circulate in their bodies. So while there's no active infection, their bodies believe that there is still -- their immune system still believe that there is one. So the immune system is constantly mounting a response and that, that goes through that immune response is producing a lot of TNF alpha, which is the inflammation, which is then associated with these long COVID symptoms, right? That's what our experts helped us to understand. These experts also told us about a grant program that we applied to where we received a $13 million grant. We're the only organization, right, company nonprofit organization of any kind that received a grant to test the therapeutic to see if we can address these CNS symptoms of long COVID of brain fog malaise fatigue. Using that grant, we have enrolled 200 patients in a trial with the help of some of the best leading long COVID centers out there, places like Yale, Mount Sinai, Mayo Clinic, UCSF, Stanford and so forth. That helped us enroll 200 patients in a trial that randomized half to placebo and the other half to bezisterim. And we are exploring a number of endpoints having to do with cognition malaise, fatigue and a number of biomarkers as well. This is an exploratory study that's looking at about 20 of these different endpoints. This trial is now fully enrolled. Yale enrolled the last patient a couple of weeks ago, which would put it on track to have the last patient come in with the last visit sometime in early August, early to mid-August, which means that we should be in a position to have top line data readout at the end of August to early September as well, okay? And when -- and the data really looks promising. And if we get the promising results upon unblinding that we hope to see, -- this would allow us to go to the FDA in the fall to have a conversation about an accelerated approval or an emergency use authorization. And we believe we can get a breakthrough designation for this as well. And that, we believe, would make us a strong takeover target or partnering target for big pharma companies out there. And we believe Long COVID could represent a $10-plus billion annual sales opportunity in the U.S. alone. Just recognizing the time here, let me stop it here, and let me recap where we stand. We believe that BioVie represents a terrific opportunity for investors. We have a -- we have 2 imminent data readouts in the coming months. We have sufficient cash to last towards the tail end of the year. And despite that, we are still lumped in with other biotech microcaps, whereby our current market cap is $10 million, which is a fraction of our current cash balance. And that's the reason why I believe we represent a terrific investment opportunities for investors who have a 12-month planning horizon. And I say that not only because I'm the CEO of the company. I also am one of the largest shareholders of the company as well, right? And I have significant amounts tied up in the company over the years. With that, let me stop it here and open up for any questions you may have. Thank you.
Thank you very much Cuong, [Operator Instructions]. Cuong we've already received several great questions here. Here's the first one. BioVie has 2 clinical top line data readouts expected in the coming months. Can you give us any more details about those?
Well, I cannot really give much more because right now, our team is going through the process of conducting the trial. What I can tell you is that we look at the data on a regular basis as they come in on a daily basis, primarily to monitor for safety just to make sure that the drug is safe, right? And as of now, bezisterim continues to essentially replicate what we know from historical trials, namely, it's been very, very safe. There are no drug-related severe adverse events, very few adverse events at all. right? We also see from the blinded data, a promising pattern of separation, whereby some people get better, some people get worse and some people stay the same, right? And if the ones who got better are the ones that are on drug upon unblinding that these 3 studies would represent a terrific move steps forward for the patient community. So all we can really tell right now is in Parkinson's. We are cleaning the data and getting ready for database lock as we wait for the biomarker data to come back from the vendors. And on long COVID, the patients are going through the trial, the monitoring process. They're taking their drugs, they're coming in for visits periodically, and we should have the last patient -- last visit in the early to mid-August time frame. Hopefully, that answers your question.
As you made clear in your presentation, you think you are different from most of the world that thinks that Parkinson's disease is caused by low dopamine levels leading to loss of motor control. You point to inflammation. Can you tell us more, please?
Well, let me answer that by starting with a very basic biology review, right? Every cell in our body needs energy to function. And that energy comes in the form of glucose. You may remember from your high school biology, how glucose remember the crep cycle and how that produces ATP and so forth. So it all starts with glucose. And when it comes to glucose regulation, insulin is the key factor. So think of insulin as a key that has to fit into a lock a receptor on the surface of every cell in the body. That receptor is called the insulin receptor substrate 1 and 2. So insulin has to fit -- it's a key that has to fit into the lock so that it can open it cell, the cell could then absorb and take in glucose. But when you have inflammation, that's TNF alpha, TNF alpha also triggers and drives something called JNK n IKK that also binds to the IRS 1 and 2. So think of inflammation as rust. It's the rust that builds upon your lock. And when you have a rusting lock, the insulin key cannot fit in and therefore, it cannot open the door for glucose to come in. So as a result, cell starts to malfunction and over time, cells die. And recall that neurons are among the most power-hungry glucose hungry cells in your body, okay? And when you have inflammation that leads to insulin resistance, it affects the entire brain. So if you have a problem that starts with substantial nigra, that leads to Parkinson's because those cells that use and make dopamine starts to die. If the problem comes in other parts of the brain, you then can have problems with Alzheimer's, ALS and so forth, right? So inherently, it starts with inflammation leading to insulin resistance that provides the metabolic driver of many of these neurodegenerative diseases, starting with Parkinson's going on to Alzheimer's and many others. I hope that answers your question.
Yes, it does. Thank you very much, Cuong. Thank you, everyone, for all your great questions. We've got many coming in here. Cuong, is there a pronounced placebo effect for Parkinson's brain wise? The emotion of hope causes the release of dopamine, which may influence results? And how do you control for that effect?
That is a great question. Thank you so much for that. And you are absolutely right in saying that there is a potential for a high placebo effect in conducting Parkinson's trial. Just the fact that you're thinking about it or doing something about it could lead to a higher release of dopamine. So we have done several things that are quite innovative in this trial. First, to avoid any placebo effect that people may have just by the mere fact that they're leaving their house and coming into a clinical site. We actually set nurses out to their homes, to conduct the assessments, right, to reduce the variability of what's going on. And so we basically try to keep people in their same environments just not to trigger any anxiety or anything that could create a placebo effect. But nurses would go to the patient's home. All the activities there are recorded by video and all the videos across all of the patient's home, across all of the clinical sites are then scored by a central radar so that you, again, do not have variability. You have a single person reviewing all the videos from all of the patients, right? so that you then have consistency across all of that. And that helps reduce the placebo effect by having patients in their home. It helps reduce variability by having a single reader. The second thing that we did to try to avoid the placebo effect is to -- in our protocol, we designed in what's called a placebo run-in, which is patients would come in with -- or the nurses would go out with the first visit, call it, visit 1, and we would assess their activities and so forth on the various scales that we use. And we wait 30 days and then we repeat that again, right? And if there's been a change in the various assessments, but that's most likely a placebo effect because nothing has been done in that 30 days. No drug has been given or anything like that. So if there was a significant change, then that patient then became ineligible to participate. On if those 2 metrics, those 2 visits were roughly dissimilar where you then randomized into a placebo arm or the drug arm, right? So those are the things that we did to try to minimize and avoid the placebo effect that is common in many Parkinson's trials. Thank you so much for that very perceptive question.
Thanks, Cuong. How do you compare your Alzheimer's disease solution to LEQEMBI and Kisluna?
Well, we are very, very different from the 2 approved drugs. Perhaps the easiest way to answer that is for me to bring up a chart. Give me 1 minute to share my screen. When I first started working in Alzheimer's in the late '80s, everyone, myself included, we focused on the amyloid plaques and the amyloid buildup. So here on this chart, everything that's in blue is a part of the amyloid pathway. Everything in red is a drug that is trying to modify the pathway in some manner, right? And over the years, we started looking at tau and tau tangles as well. But the reality is over those 5 decades, for 5 decades that I've been working at it in Alzheimer's, there's never been a drug that can reduce plaques and tangles and arrest or reverse the cognitive decline in Alzheimer's. And within the last decade or so, more and more researchers are coming around to the point of view that plaques and tangles themselves are not the toxic agent that's causing neurodegeneration, but they're inflammatory in nature. They cause inflammation that works through the TLR4, the toll-like receptor 4 receptor here that we talked about in long COVID or RAGE and other receptors, right? But as you can see, many other things cause inflammation as well. But all of these things share one thing in common. they all activate ERK and NF-kappa B to produce TNF alpha, right? And of course, when you have CNF alpha, you have more cytokines and more APP. APP then leads to more amyloid buildup, right? TNF alpha actually drives the kinase that drives that turns tau into phospho-tau. And we are very different than all the other drugs in that we block the production of TNF alpha right before the production of TNF alpha, which is considered to be the master regulator of inflammation. The drugs that were mentioned, LEQEMBI and lecanemab and donanemab, they actually block -- try to reverse the production of amyloid and block the production of the plaques. And they have -- and the reason I believe that those drugs have only had a limited impact on the disease progression is that the only thing that they're doing is they're reducing the inflammation that's created and are driven by the plaques, but hasn't done anything about the inflammation that's driven by the other factors, right? And that's why they've only seen a very modest impact on cognitive decline, whereas we believe we will have significantly greater impact because we block everything. And as you may know, we had a Phase III trial in Alzheimer's. Using the same endpoint that was used here for LEQEMBI, for example, CDRS-B, we had a 68% slowing of cognitive decline versus placebo after just 6 months of treatment. And to put that into context, the anti-amyloid drugs had as a class, roughly a 30% slowing of cognitive decline versus placebo after 18 months of treatment. And we have had a very mild side effect profile. The #1 reported side effect was a mild headache reported by about 8% of patients, whereas, as you know, the other drugs have had significant concerns about brain swelling, brain bleeding such that they need to be followed by -- monitored by PET. And so we look forward today when we can essentially proceed further and go to the next Phase III trial to show statistical significance of the results that we're seeing here in this trial that has fewer patients. I hope that answers your question.
Thanks, Cuong. Status of the ascites drug, is there a spin-off anticipated?
A great question on that as well. We have, as of now, have received all the feedback that we need from the FDA to proceed to the next step, to essentially conduct the one registrational trial that's required because as you know, we already have orphan and fast track designation. We have filed an S-1 to take a company called Option Therapeutics public. Our intention is when market conditions are right, we will put the ascites drug into Option Therapeutics. We would endeavor to raise $25 million, $30 million or so for that company, and that would allow us to conduct a trial that's needed, right? And we believe that trial will probably take about 2, 2.5 years to conduct and to get it registered, right? So right now, we're waiting for market conditions to be right for us to be able to go out and try to raise about $25 million for the ascites program by floating by taking Ox Therapeutics.
Thank you Cuong.
2. Question Answer
Can you show the potential revenue chart for Parkinson's, Alzheimer's, Long COVID and ascites Thanks, Cuong, Great job.
I wish I had the most updated chart on that to show that in one place the numbers, I do not. But let me give you the numbers, right? We believe that ascites could be a $1.5 billion to $2 billion annual sales drug in the U.S. alone. We believe that Parkinson's could be a $3 billion to $5 billion annual sales drug in the U.S. alone. Long COVID could represent a $10 billion annual sales in the U.S. And of course, Alzheimer's is going to be the blockbusters that could be a $30-plus billion annual sales product in the U.S. alone, right? And thank you for asking that question. That just reminds me and I could go and put that chart into the next version of the deck.
Thank you very much Cuong.
Let's give everyone a moment to consider any more questions they may have for Cuong Do, the President and CEO of BioVie. [Operator Instructions]
This person wants to know more about collaborations. Could you go into more depth with those collaborations that you mentioned with large pharma for the co-development or out-licensing of bezisterim in specific regions or indications?
We've had a number of conversations with larger pharma companies. And the feedback from them has been pretty consistent, which is they are very intrigued with this mechanism of action and the drug, and they want us to essentially come back to them when we have the clinical results, which I get it. I've been on their side of the table as well, right? What I've learned in big pharma is that no one ever gets fired in big pharma by playing it safe, right? And so pharma -- and as you know, the world out there is littered with promising Alzheimer's and Parkinson's drug that has failed in Phase III, Phase II. And that's why big pharma companies now are quite shy and quite reticent of doing collaborations and deals before you actually get data that actually is much further along. So those conversations have been had. They're on hold. And I think the -- we will reengage when we have additional data, right? And I think COVID could also be a very interesting conversation for us to have in the very near future as well.
Thank you, Cuong.
How much would an Alzheimer's trial cost total start to finish?
That's a very tough question to answer, but let me give you a ballpark. Based upon what we know now and what we have modeled out, we believe that Alzheimer's trial could take $25 million to $30 million to conduct. That's one trial, and we would need to conduct 2 of them, right? So the total cost to bring bezisterim to market for Alzheimer's would be $50 million to $60 -- and that's part of the reason why the program is currently on hold until such time as market conditions would allow us to go and raise the funds, right? That's why we have focused everything so far on Parkinson's and Long COVID because those trials tend to be smaller, easier to do and shorter to conduct.
Will there be any analysis of blinded data on Parkinson's as you did with Alzheimer's?
We are conducting dose analysis right now, and we have been conducting for Parkinson's as we go along. But we purposefully have chosen not to share or release any of them for the reason that was alluded to by -- in an earlier question, which is we want to avoid doing anything that could lead to a placebo effect. If we announce, for example, that, oh, we're seeing great separation with patients, that could lead anybody in those patients in the clinical trials or even the clinicians in the clinical trials to actually start to see things that may not be there. And that's the reason why we have chosen not to say anything or announce anything about the blinded data as we've gone along.
Thanks a lot, Cuong.
Can you recommend where to go to learn more about the mechanism of action and other pharmacological details of bezisterim?
All of the information is currently available at our website. So please to go to our website, which is www.bioviepharma.com. There's a section there that talks about our science where you could go deeper into the science. Under the Investors section as well, you can see links to the KOL events that we have held within the last few years on these indications, right? Just a few weeks back, we had a KOL event on Parkinson's. And about a year ago, we had a KOL event on Long COVID, right? And we are considering doing another KOL event on long COVID in the next couple of months, just to kind of reappoint everyone in the market to what we're doing on long COVID, the causes of long COVID and why we're optimistic bezisterim could help address long COVID, doing all of this to essentially build awareness in advance of our data unveiling late summer, early fall.
It seems that so much of what BioVie is doing is based on its approach to inflammation and aging. Tell us again how your approach to inflammation and aging differs from your biopharma peers.
Frankly, you can count on 1 possibly 2 hands the number of companies that are working in inflammation and CNS diseases out there. The whole world has really focused on in Alzheimer's, for example, on amyloid and tau. And we believe that is just a dead end, right? We have tried for years to basically sound the alarm and say that there is -- that is a dead end, but we're a tiny company. And so as a result, we don't get a lot of attention, and we actually just kind of have to go out there and demonstrate it. So we count ourselves among the 2 handful of companies that are working as working with inflammation as the key driver of disease pathology. What also makes us very different is also our approach on insulin resistance. Because bezisterim blocks TNF alpha, right before it activates JNK and IKK, it actually reverses insulin resistance. Please remember that bezisterim was originally brought into the clinic as a diabetes drug. And in that first trial, it showed exactly what you would want to see in a diabetes drug. It reversed insulin resistance and it brought all these different biological systems back into normal ranges. So glucose, HbA1c, cardiovascular and so forth. But that was before the team truly understood the mechanism of action and how it blocks TNF alpha. And once we understood that, we recognize that bezisterim role is potentially much bigger in the world and in addressing the biggest unmet medical needs right now, essentially Alzheimer's, Parkinson's and a number of other CNS diseases. And I believe that's what makes us very, very unique in the biopharma world out there. We, I believe, have the most unique molecule and leads us to have the most interesting portfolio in the biotech world.
Thanks, Cuong. And we probably have time for a few more questions. We've been getting so many great questions. Thank you so much to all of our participants.
This is medical related here.
So if someone has type 2 diabetes and inflammation and insulin inefficiency, are they more likely to get Alzheimer's?
The answer to that is, unfortunately, yes, absolutely. If you have type 2 diabetes and insulin resistance, statistics shows that you are at a significantly greater risk of developing Alzheimer's, Parkinson's and other CNS diseases. Let me give the statistics the other way around. If you look at Alzheimer's patients, the Mayo Clinic and NHS out of the U.K. have shown that about 80% of Alzheimer's patients have or are developing insulin resistance. The comparable statistics in Parkinson's is about 50% of Parkinson's patients have are developing insulin resistance, right? And so that's the part of the reason why we so strongly believe that many of these diseases have a metabolic underlying underpinning driver to it. And it's not just about just low dopamine or amyloid and so forth. It really starts with TNF alpha, which is the master regulator of inflammation. And when you have TNF alpha, you're going to create insulin resistance and insulin resistance starts to kill off cells. And that's why neurodegeneration is a real problem. That's a link between inflammation, insulin resistance and neurodegenerations in our belief.
All right. We are just at the top of the hour. We'll wrap up here. And if you do have any questions for Cuong Do, no matter whether they were answered today or not, you can write us at [email protected], and we will make sure that Cuong sees your question. For more information about BioVie , again, you can write us at [email protected] or you call us at 1800redchip. Please visit Redship's Investor Information page for BioVie. It's bivinfo.com There, you can view and download the investor presentation and fact sheet and sign up for news alerts on BioVie. Please watch Small Stocks Big Money, Redship's program featuring exciting small-cap companies every Saturday night at 7:00 p.m. Eastern on Bloomberg USA. And finally, please join our next webinar with Foremost Clean Energy tomorrow at 4:15 p.m. U.S. Eastern. Register for all redchip webinars at redchhip.com/events. Thanks again to our many participants today. And as always, thank you, Cuong.
Thank you, everyone, for joining. Have a great day.
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BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Good afternoon, and welcome to the BioVie Virtual KOL event. [Operator Instructions] As a reminder, this call is being recorded, and a replay will be made available on the BioVie website following the conclusion of the event. Please refer to this slide about forward-looking statements, which describe the disclaimers and risk factors related to such statements and consult Bovie's public filings made with the Securities and Exchange Commission that can be found at www.sec.gov. With that, I'd like to now turn the call over to Cuong Do, Chief Executive Officer of BioVie. Please go ahead, Cuong.
Thank you, Tara. Thank you, everyone, for joining today. My name is Cuong Do, I'm the President and CEO of BioVie. I'd just like to take a few minutes to give everyone an update on where we stand on our 2 clinical trials. Our first trial, of course, is in Parkinson's. That trial is nearly complete. The last patient has come in for his last visit. So our team is working through the study closeout process right now to basically go through all the data to clean up the data and so forth. But most importantly, we need to wait for the biomarker data to come back in.
We are hoping to have everything in and analyze by the end of this quarter, but it may slip into next quarter, depending on how long it takes for us to get the biomarker back in. In addition, we are enrolling a long COVID trial. And that trial should complete its last patient visit next week. And if everything goes according to plan, we should be in a position to have top line data readout before the end of the summer.
And so today's cable of that is really aimed at helping lay the groundwork to understand our Parkinson's data readout. Everyone thinks of Parkinson's as a motor disease that's essentially driven by lack of dopamine in the brain causing motor dysfunction. That's part of the answer, but that's not the only lease total answer. And as you will hear today from Dr. Dela Monti and our team, there is a big insulin resistance and metabolic component to what really underlies Parkinson's. So with that, I'd like to turn it over to Joe Palumbo, our Executive Vice President of Research and Development and Chief Medical Officer, to get us started. All right, Joe, over to you.
Thank you very much, Cuong. I think we're going to be able to show you quite a bit of science today that really supports the theories we have about our molecule. And looking at neuro inflammation, metabolic and inflammatory effects, I think there's no one better to comment than Dr. Suzanne de la Monte, who is a professor long-standing at Brown University, Professor in Pathology, laboratory medicine, neurology and neurosurgery. She is Chief of the Pathology and Laboratory Medicine Department at the Providence and VA Medical Center. And she is exceptionally well informed, and we are delighted to make her available to present on these topics. Dr. De la Monte, if you'd like to begin, please.
Good afternoon. Today, I'm going to present the evidence behind insulin resistance and metabolic regulation. -- not only is a feature of brain aging, which is the necessary component of neurodegeneration but also as a feature of the neurodegenerative processes that take place in most circumstances.
I hope to be able to show you that there is an important consideration for benign versus malignant aging. Malignant aging is the thing that sets off the boat for us to undergo neurodegeneration, whereas benign aging is something that we have to do because we just get older.
Alzheimer's disease has been Type 3 diabetes because of the roles of insulin deficiency and insulin resistance, but Parkinson's is not far behind in terms of having those abnormalities. We do have challenges, however, that relate to the accurate detection and monitoring of the disease. This is a big problem.
Cofactor and lifestyle contributions, which are muddying up the the features of the disease, but also increasing the rates and then the potential for therapeutic interventions. So first of all, as I mentioned, aging is the most important risk factor for neurodegeneration. Alzheimer's, which we claim accounts for 70% of -- or 80% of dementia is a chronic progressive disease. It keeps going downhill. It's not something that stays steady, but there are challenges because these diseases, Alzheimer's in particular, is often mixed. It's not so clean it often is vascular disease, overlaps with Parkinson's.
And the second major challenge is the cofactors, obesity, diabetes, hypertension, stroke, environmental factors, these are all adding to the rates and also the picture of what's going on with AD. So it's looking different today than it did when always Alzheimer's saw the first patient.
The market for this is huge. We are probably going to have at least 115 million people worldwide with Alzheimer's or close to it by 2050. And the process begins with mild-cognitive impairment, which is added to this and the prequal basically to AD, the biggest problem are the people who have the asymptomatic, I like to call them sneaky period disease because we don't know who they are. And the patients are offering us in denial or not willing to admit anything.
The disappointing therapeutic outcomes largely relate to the challenges of face with respect to diagnostics and understanding the disease pathogenesis. As mentioned, Parkinson's we often regard as a motor system disease. And that old idea is very old. And instead, we know now that at least 80% of them go on to developed debenture within 10 years.
So they're being present with motor symptoms, but they often progress to varying forms of neurodegeneration that impair cognition and behavior. This is an important thing because the treatments are not available for that part of the disease. So when we're thinking about Alzheimer's, we still have this old idea about the plaques, which is the AB, the plaques of the center here versus the neurofibulators angle and the last 40, 50 years have been spent on finding these abnormalities in the brain.
And as a result, PET imaging has evolved to very accurately detect their presence in accumulation in the brain. The problem is that these aren't the only things that are abnormal. And by targeting this alone, we haven't had any success in treatment. There are no treatments that actually cure or actually stall the disease based upon those 2 molecules alone.
The other group, the Parkinson's is we always think of it as a motor system disease. This is a substation again at the upper left should be block and pigmented where it's on the right in Parkinson's, those neurons die. Those are pigmented neurons. They have -- they're parting for dopamine and they get called Lewy bodies. Well, the Lewy body are just like neuro fiber tangles and as if their accumulation of stuff that should be in the trash barrel and they cause stress and dysfunction. So they start out with motor symptoms, but actually, these eventually hit the cerebrum and the behavioral centers of the brain.
But Alzheimer's from -- I don't know how many years ago, several decades ago, it was known that there's a metabolic disregulation. Glucose utilization, glucose is the main fuel in the brain. Glucose utilization, as shown here in the PET imaging, the lower part, the less red and green you have is corresponds with impaired glucose utilization and effectively a brain servation compared with the top, which is a control.
But if you study these are you really so happy about it, you study Parkinson's and frontal temporal event, they also have a problem with brain metabolism and glucose utilization. So now we have a mirror thing, and this usually happens particularly after the patient undergoes cognitive impairment. So we're dealing with a problem that has to do with metabolic dysregulation in the brain. Just to remind you, the whole process of glucose utilization is dependent upon the function of insulin.
Insulin is the master hormone. It does have cousins that are out there that function. But insulin is the big 1 that helps with glucose uptake utilization and eventually metabolism. The outcome of having good insulin function is we have plasticity. We have learning. We have memory, we have cell survival, and we have mitochondrial function. And without these pathways, we basically have cells dying and not talking to when disconnection happens, that's when we have cognitive dysfunction.
It's important to realize that insulin doesn't work by itself, there are other pathways, and not enough time to go into it, but they're known as not wind epidermal growth factor and importantly, recently are the incretin pathways, which we'll touch on in a bit. but the signaling pathways are basically the same. So what's wrong with our concept of Alzheimer's and Parkinson's is that we're only considering neurons only considering neurons in certain parts of the brain.
In fact, when you look at the brain, every single cell type, whether all of the dendrocytes which make the Mylan, the astrocytes, which are important for the blood brain barrier, the structure, the vasculature and the microglia, which regulates inflammatory responses. So all of these are impacted by neurodegeneration. And it makes no difference if you're looking at Alzheimer's and Parkinson's, they all are effective. We don't address these points at all. I mean, we deal with the neurons and specific neurons, but we need a bigger picture for what -- how they'll address the problems that are going on.
In essence, with aging, because the cells aging and neurodegeneration, accelerated aging, we end up with increased sensitivity of the neurons to oxidative stress, meaning that if you have anesthesia, you have hypoxia or anything like that, those neurons are vulnerable so they get hit easily. And that's one of the reasons why people were 60 years old or older, and they have anesthesia, they can wake up to various, they can actually end up with early cognitive impairment and the more trials you have, the worse it is. And that's a big deal. So we need to know who those people are.
Cell survivals impaired and oxygenates. Neuroinflammation, which will be covered by a Clarence leader, that's a big deal as well. Every degenerative disease has the microentheastrocite it going on and inflammation could be coming from the periphery into the brain. In essence, it's causing trouble because of the damage that takes place and then the vascular dysfunction, the blurb barrier is very important for regulating what gets into the brain, but there's also a vascular part that gets trashed out of the brain.
So it's backward and forward flow system is impaired because of the vascular dysfunction. So if you think about these diseases, the way I'd like to think about it, we need to remove our narrow focus on the few signature abnormalities in these diseases and look at the bigger picture, which is basically a metabolic dysregulation that is championed by impaired glucose utilization and oxygen metabolise -- oxygen utilization and having effectively a brain starvation.
And it seems more complicated like how do we get this to get all these things that are wrong. But if you look at metabolism as a driver for all of these -- it makes it a lot easier actually because now you're dealing with every cell type and all of the things that are wrong with them when you go neurodegeneration, we could do a comparison for metabolic diseases that take place in other parts of the body. And believe it or not, they are virtually identical. I work in the lab where we study liver I always have with the cancer lab.
And when we start looking at the metabolic deficiencies and involve carbohydrate and lipid metabolism, those that did on by the kind dysfunction is always a problem basically right out of good mitochondria, so they can't have full oxidative metabolism. The vasculature is messed up, the stress response is also activated and you have increased cell death of inflammation.
So these things are make us think about how do we borrow the treatments and the approaches across disease processes. So how do we know it's insulin? I mean how do we know that's the problem? Well, when you look at brains with Alzheimer's disease and you look at severity of Alzheimer's, you can find that there is a decline in the amount of insulin trophic factor insulin-like growth factor, Ig1, there's an impairment in the binding and the receptor function.
So this is basically graph showing brock stage, brock stages like a golf score, the higher the number the worse you are. And basically, the -- with the severity of Alzheimer's going from 0 to 6 we find that the insulin, the insulin-like growth factor, the receptors and the ability of the truck effects, all declined with increasing severity of disease, not necessarily age per se.
In fact, when you go to earlier stages of disease, we looked at cerebral fluid, and we find that the abnormalities began much earlier than patients would report in terms of their symptoms. So that's automatically a kind of biomarker for dysregulated metabolism.
And when you look at what's going on, what are the roles of insulin IgF, they basically take care of the neuroinflammatory response, synaptic plasticity, in learning the memory, all the things we talked about that were important in the pathogenesis of disease.
Now the other thing to be aware of, and I take a keen interest in it is the role of the incretins and ensure these are very popular GLP-1 receptor agonist, I think talks about. But it's important to know there are a bunch of them. It's not just GLP-1, GIP is AMOLED, who knows how many are coming out. These all hit. You can see that white diagram around the media that this area here is really pretty cool because this is the major region of brain damage in most of neurodegenerative diseases, other hits into the brain stem, which is Parkinson's. Cerebellum in is into the frontal lobes and the temporal lobes. And so these are the structures that are impacted by impaired insulin signaling, but incretins also participate in this thing.
So we have to be aware that these incretins signaling, which regulates a lot of the insulin pathways or cooperates with them is important in these factors. And in fact, when you measure the level of incretin and reactivity in the brains and look at all kinds of biomarkers that those are bad. So we're dealing with a bunch of things, all of which regulate metabolism in the brain.
And then the last group of the neurosteroids and won't go into to HF, but notice where those signals are, they seem to overlap exactly what the figure I just showed you. So we're dealing with the same structures that are abnormal in the brain for the new steroids. And so that gives us hope about the direction.
So in terms of where can we go with this? The oldest treatment has been to try insulin sensitizers, which have had limited permeability to the brain and effectiveness and curtains are coming along as being trial, but I know one by one is not going to work, but there is some promise. And then the other group, the third group are the neurosteroids, which have added, which seem to be really strong for providing both anti-inflammatory, antioxidant and insulin sensitizer function.
So I think the promising view is to include at this additional component into the cocktail, if you will, to enhance brain metabolic function that's deteriorating, both in Alzheimer's and Parkinson's. And I'll stop there.
Okay. Thank you very much, Dr. De La Monte. Clarence, Clarence Ahlem, who is our SVP in our group looking right now at Parkinson's disease and he'll give us our introduction to bezisterim.
Hello, everyone. I'm Clarence Ahlem. I'm the Senior Vice President of Operations at BioVie and I've been working on the development of bezisterim for about 20 years now. We can get the next slide. Here we go introduction to a bezisterim. Now you go back -- that's good. I'll start with a quick review of bezisterim characteristics.
Here's the molecular structure. Bezisterim is a novel anti-inflammatory agent. It's a sterile, but it's not bound by and does not target steroid binding nuclear hormone receptors. bezisterim is not active in the synapse, it does not interact with neurotransmitter receptors. Next slide, please.
Bezisterim is orally bioavailable and briefly permeates the blood-brain barrier. It targets extracellular-signal-regulated kinase in pathology specific signaling pathways, but it doesn't inhibit ERC in signal pathways are involved with homeostasis. Bezisterim is promotor and neuroprotective and MPTP Parkinson's disease models, it improved motor and nonmotor symptoms in our previous Phase II study Parkinson's, and Bezisterim improved cognition and decreased DNA methylation-based biological age acceleration in Alzheimer's patients.
Next, Bezisterim's mechanism of action is unique in BunstoRCand this large protein complex that also contains a signaling protein called MAP3K8, along with the NF-Kappa-B complex, and mitogen and ERK kinase, that's MEK. This pathway is stimulated by inflammatory mediators interacting with their cognate receptors, which activate ERC and incapabin the scaffold to stimulate inflammatory cytokine production and phosphorylation of a tumor necrosis factor receptor 1, which is responsible for chronic inflammation.
It's important to note that ERC is also essential as an essential component of a different scaffold that promotes insulin signaling that's shown here on the lower left. Bezisterim does not interfere with ERC this scaffold and in fact, the drug was originally developed to improve insulin signaling in type 2 diabetes. We believe that the selectivity of Bezisterim for inflammatory signaling is essential to its attractive safety profile.
Next slide, please. As we heard from Dr. De La Monte, neuroinflammation, insulin resistance, mitochondrial dysfunction and oxinative stress are causally related in forward feeding. Chronic inflammation drives epigenetic changes that are important to biological age acceleration in Parkinson's disease progression.
Next slide, please. So epigenetic age acceleration and unfavorable epigenetic changes in genes associated with Parkinson's pathophysiology of promote disease progression. The information theory of Beijing is based on the notion that epigenetic changes and loss of epigenetic information drive aging. However, and this is very important to development of Bezisterim, these epigenetic markers that have been lost can be restored under appropriate conditions.
So changes in DNA methylation can change the expression of genes to promote Parkinson's disease symptoms and progression. The expression of harmful genes can be increased and the expression of protective genes can be decreased. The process is controlling methylation to specific gene or its regulatory elements like the promoters and enhancers are complex, and they're not necessarily intuitive.
And the effect on expression is free when we sell, but ultimately, inflammation has a deleterious effect. And the cumulative effect of these small changes in cascades can be busy logically significant. Obviously, age is the risk factor for disease of aging with biological age, however, and not chronological age being the most important factor Until recently, the concept of lowering biological age has been more theoretical than practical. However, we have found that Bezisterim alters DNA methylation to lower person's biological age.
When we talk about clinical study results, we usually refer to age acceleration, which is the difference between an individual's chronological age and biological age with a positive number indicating that the biological age is greater than chronological age, which is not good for that person. And a negative number indicating biological age is less than chronological age is, of course, what we would all like to have.
Bezisterim by interrupting the self-reinforcing loop of inflammation, resistance, oxidative stress and epigenetic drift lowers age accelerations measured by assortment of epigenetic clocks. The results of 3 DNA clocks imputed from the published results from our Alzheimer's study are shown here on the lower left, you can see the negative changes in Bezisterim treated subjects in the green column and the positive change in subjects in orange with placebo.
Again, for age acceleration, negative is good. The Pheno a clock on the far left is the subject of our next slide. Next slide, please. Okay. So there was a U.K. biobank study analyzed the time for 569 subjects with Parkinson's to progress to death from the time of enrollment. The findings of the study showed that epigenetic age acceleration predicted mortality.
On the left, you see that Pheno age acceleration greater than 0, that is faster aging, progressed to death faster than acceleration less than 0 and it is slower aging. And on the right, you see that the greater the aging, that is the greater age acceleration, the faster the progression to debt. These results and the results of other studies showing a correlation between DNA acceleration and disease progression create a new and exciting possibility of using DNA methylation as a biomarker to predict Parkinson's disease progression.
We're hopeful that the DNA methylation results from Sunrise PD will be similar to what we observed in Alzheimer's, but of course, the data will have to speak for itself. And additionally, I have to say we have not met with the FDA to discuss DNA methylation as a biomarker, but nevertheless, this line of thinking appears to have great potential.
Next slide, please. The important message here is that there are many interacting inflammatory pathways that NF-kappa-B, is the master regulator of inflammatory cytokine production. And as a reminder, NF-kappaB has many important homeostatic activities, which makes Bezisterim apparent selectivity for inflammation signaling critical to its potential usefulness in chronic diseases.
Next slide, please. So inflammation disrupts homeostatic mechanisms in the brain. Glial cells that is microglia and astrocytes have critical functions in support of neuronal activity. Inflammatory cytokines controlled by the Napa influence the function of cells and the molecular messages that are released. Bezisterim acting in the MAP3K8 in FCoRC signaling pathway can reduce the production of inflammatory cytokines that disrupt the homeostatic cell functions.
Peripheral inflammation and CNS infiltrating inflammatory cells disrupt the blood-brain barrier and contribute to the inflammatory mailman Parkinson's disease. Bezisterim by acting systemically as well as in the CNS can reduce inflammation quote.
Next slide, please. So neuroinflammation is a major factor, IV motor and nonmotor symptoms in Parkinson's. Neuroinflammatory viral infections such as influenza and COVID-19 can induce transient Parkinsonian behavior, including non-motor like symptoms without apparent extensive neurodegeneration. The inflammatory cytokine storm associated with these viral infections is believed to be responsible to symptoms.
And in Parkinson's patients, intranasal insulin and GLP-1 receptor agonist can improve movement even though these medicines do not impact dopamine bioavailability. In animal models of PD, many of which are created with inflammatory challenges various anti-inflammatory treatments improve all aspects of disease.
We believe that symptomatic treatment with Bezisterim will have much less potential for motor applications and neuropsychiatric side effects.
Last slide, please. So to summarize, the Bezisterim appears to act in an inflammation specific pathway. Bezisterim adverse effects have been similar to placebo in clinical studies. Neuroinflammation in Parkinson's disease drives motor symptoms and disease progression. Bezisterim has had positive effects on DNA methylation in Alzheimer's subjects, and we're hopeful that we observe similar effects in our SUNRISE-PD study. Neuroinflammation drives Parkinson's pathology through dysregulation of energy homeostasis, alpha-synuclein folding and cellular interactions. Bezisterim may reduce inflammation in Parkinson's to improve both motor and nonmotor symptoms and slow disease progression. Dr. Palumbo, the floor is yours.
Well, thank you very much, Clarence. So we're just going to change slide sets right now. But what I'm going to be talking to you about is why we hypothesize that bezcterin is going to be a good match for the progression of Parkinson's across the lifespan of Parkinson's. So as we bring those slides up, the journey in Parkinson's is quite difficult. And it begins well before motor symptoms typically emerge.
So I'm going to talk to you about the sequence of symptoms, how Bezisterim will likely interact with that if our hypotheses are correct. The medical need for a drug that works on multiple stages of the disorder and the path to future therapies and our embracing of precision medicine. So I'm going to go to the next slide here and see if I've got control, and I do. So I said we would talk about disease evolution.
And really well before the onset of classical Parkinson's motor symptoms, the tremor, et cetera, there's a progressive nonmotor disease that emerges. And when I say non-motor, I mean, it doesn't necessarily relate to movement, but you can see these in various body systems and brain and behavior. So up to 10 years before the diagnosis, patients may present with constipation and anosmia, which is not being able to smell, REM sleep disorder, that means acting out your dreams and potentially hurting someone.
Three to five years before there's a depression, anxiety, fatigue, and 1 to 2 years before perhaps cognitive flowing and some apathy. But by the time someone reaches a motor diagnosis there's already tremendous inflammation going on. There is a neuronal loss, and these non-motor symptoms have already presented in 90% or greater patients.
So you've got this path of progressive disability that begins before agility, tremor and slow movements begin. So I'm going to present 2 of the kinds of patients I might have seen. Now these are composites. These are not real people, but I present them in this way to kind of show what happens.
So the first person is, make believe. This is James, who's 52 and engineering director. And I mentioned this REM sleep disorder. So 7 years of REM sleep disorder, an inability to smell before slowing of his body starts to be noticed at work. And someone said, I'm going to start you want a dopamine agonist.
And we know that these dopamine agonists for some people result in impulsive behaviors like gambling, et cetera. So here he is 7 years later, he's now on disability leave, managing his depression and really looking at cognitive impairment and side effects for medications. It's not the motor symptoms that impaired him, it is really the nonmotor that contributed to his disruption of career.
This is another, make believe, person Maria, 48 an attorney. And again, a typical progression 5 years of fatigue, constipation, restless sleep. And then after those 5 years, he developed a right hand tremor that leads to diagnosis. So she started on levodopa and for some people, that drug begins to wear off. And if you look at the drugs that have recently been developed and approved, they tend to be various versions of levodopa that act a little bit longer, are continuously infused, but it's kind of the same thing.
So for this individual, she's less able to work. She's removed herself from the work that she does. And it's just perhaps a little depressed more socially isolated. So what this -- the take home from this is while levodopa can control tremor, it doesn't do much or anything really for fatigue, cognition, mood or the other non-motor symptoms that really impaired her ability to do her work.
So let me take you to the next slide. So we can really what actually drives the quality of life. And this is based on a study that was funded by the Michael J. Fox Foundation to look at what impairs you. And listed from top to bottom are depression and anxiety, fatigue, sleep disruption, cognitive impairment and autonomic dysfunction, none of which typically gets identified as a symptom of Parkinson's, but are part of Parkinson's.
So these things in order, depression, fatigue, sleep, cognition, autonomic dysfunction, like orthostasis, constipation. These things compound disability and control for 65% of the impairment and quality of life, we don't have a treatment for them.
So why do the current treatments fall short? Because you can look at that slide and say, "Well, no, we've got treatments for depression and anxiety. Well, for nonmotor symptoms, there's nothing actually approved specifically in the area. And we use antidepressants and anxiolytics off-label. We may try cholinesterase inhibitors for cognitive decline. We try to symptomatically manage sleep, fatigue and pain, but there really isn't any therapy that manages the underlying neuro inflammation.
It's not a small thing. This is an $82 billion per year burden in the United States. And the majority of that is actually lost function, right? So less than half of that relates to medical cost, right? The rest of this is, I can't work. People have to look out for me. So this is really a tremendous loss.
Now for motor therapies. And next slide, part of the slide, yes, we have to levodopa. It is a gold standard, but it's after a period of time, there's some wearing off decreased efficacy, dyskinesia, which are unusual motions. And that happens to about half of patients within about 5 years. We've mentioned dopamine agonist and that other patients in yet. And that happens in -- with some dyscontrol in post and maybe 1 in 6 patients.
MAO-B inhibitors provides some symptomatic benefit, but no disease modifying efficacy. And then once you get to deep brain stimulation, which requires a neurosurgical intervention, the implantation of electrodes, they can be effective. That's a procedure that can be effective for advanced disease, but it doesn't slow progression.
So let me take you to the next slide. So what's our testable hypothesis why are we developing Bezisterim? Because our core hypothesis is that if you can get in early and you can target neuroinflammation, you can intercept and improve non-motor symptoms. We can affect quality of life in the short term and engage those systems that drive motor progression over time, right? And try to slow down -- slow that down.
So testable hypothesis. Number one, remember that Parkinson's is a multisystem disorder. You heard that earlier for Dr. De La Monte, inflammation-driven cellular dysfunction for see a company's neuronal loss. And we think an anti-inflammatory strategy makes sense there.
Number two, and again, why is evident space? Do are inflammation clearly contributes to nonmotor symptoms. And as I've mentioned before, this is the strongest in determined -- independent determinant of quality of life. And now three, it can be measured, right? The standard measure, the MDS UPDRS part 2, which looks at activities of daily living reflects combined nonmotor domains, including mood, cognition and fatigue, autonomic function, we talked about, as well as motor functional impact. This is a functional scale.
This is not looking and seeing how much you're shaking or how little you're shaking, this is really looking at the part 1, along with function to see if a person can benefit. And this is really where we're invested. And again, just to review Part 1 is a nonmotor experience as a daily living, Part 2 or the motor experiences of daily living and Part 3 is motor examination. With Part 2 really being about function. And this is what FDA likes.
So let me remind you of where we've been. This has been a 20-year story, but more recently, with funding from the Michael J. Fox Foundation as Clarence has told you, we did a primate study, we looked at lesion marmoset, gave them this strong and they did well. So if you go to that Northeast primate key findings. We improved mobility statistically, lower immobility, reduction in dyskinesia and neurons for viable.
And so you'll recall a couple of years ago, we presented data on our first translational study in unit in which we sought to replicate these primate findings. And we were able to do that. We were able to show an improvement in the UPDRS 3, which is motor. And in this part of the disease, yes, motor is really important. We showed greater on-time, and our adverse event profile was really quite similar to placebo, in fact, equal to it.
So why are we looking at early Parkinson's disease because this is a brain-derived molecule. We have made it oral. We have made a blood-brain barrier permeable, the measure -- the mechanism of action is what Clarence had described. And you heard a little bit about increnins, the GLP-1s being an example. We know that those are being looked at. we don't have some of those burdens. And I'll talk a little bit about that.
And again, our prior Phase IIa study that I had just talked about was an advanced Parkinson's. These are people who are on levodopa who are having that loss of efficacy that we had talked about earlier. We presented that study to a group of international experts and they said, "Fine. Let's move on. Let's go early, and that's what we've decided to do. So that's that early study. Now looking at those folks, we decided on a precision Phase IIa study in early Parkinson's, Cuong told you earlier that the last subject had enrolled and had really had moved along very, very nicely.
So our study design is very efficient. We're looking at about 50 to 60 early patients who are drug-naive who have been approved by an executive committee to make sure that they meet the diagnosis, they're on 20 milligrams twice a day versus placebo for 12 weeks, followed by a 4-week follow-up period, right?
And the idea here is to show target engagement to characterize this drug. This drug is first in class, which you can't expect it to an MALB inhibitor. We can't expect it to look like any of the other medications that improves. We have our hypothesis and we need to, therefore, look at how does the drug interact with the experience of having early Parkinson's and the endpoints are what I imagined earlier along with measures of quality of life, safety, tolerability and as Cuong mentioned, very much earlier, a number of biomarkers that are related to the mechanism of action of the drug, DNA methylation, which is really telling your DNA, how to function.
So let's take a look at the next slide. And this is not an easy thing. You have to match your drug design to the patient. So we know that when you use the UPDRS, you tend not to look at signals early really, really well because there's a lot going on. On the other hand, we're very much aware that there are targeted measures. The PAR comms is one where there are awaited composites. We're aware of that. We've incorporated some of that.
But again, it's to finding the drug's differential profile. And small studies really do that well, you get a lot of biological signal per patient as long as you concentrate on mechanism. You can get a rapid proof of mechanism before committing pivotal scale resources. We think that's a wise use of funds. You're basically fingerprinting across motor and nonmotor domains. And you can do it. It's been done, right? So this is another 1 of the incident. They showed some efficacy using this kind of model, but about 50% of the patients really had adverse events. And for us, with the oral route and what we've seen so far in our previous studies, we think we're going to be very well tolerated.
So I've told you we're doing things in a very precise way. You can certainly read that. But for us, what does success look like -- it means we've got target engagement. We can map a biological effect and then map that clearly to a clinical effect that we have a signal, we have a path and we have prospective endpoints really identified with precision.
We've looked at a spectrum of disease elements, and we're going to be working on them. So we're looking at a unique signature and a new class of medication. We're looking at the effects of an anti-inflammatory and nonimmunosuppressive drug, and that's really important. This is not a steroid, right? This is not immunosuppressive. This is anti-inflammatory, and that makes us unique.
We will understand our time course, the signal of the drug, and we think we'll be able to give investors and regulators clarity but how this drug works. We will have a coherent data package that will justify additional investment. And with that, I'm going to turn it back to our CEO, Cuong Do to hear his viewpoint on this and whether or not we've got any questions because we've probably got good answers.
Thank you, Joe. Thank you, Dr. De La Monte and Clarence for walking us through this great presentation. Let me see if I can get started on the Q&A.
My first question is actually for Dr. De la Monte. You had mentioned that metabolic dysregulation is really the key driver for a lot of disease conditions, like a lot of things that start to go wrong in the body. And a lot of that really affects insulin resistance, expires with insulin resistance. So if you had an agent that is able to reverse or modulate the insulin resistance that's going on in the body, would you expect that to be able to address not only the disease symptoms, but also perhaps modify the progression of the disease.
I would hope that, that would be 1 of the strategies. I wanted to clarify that the abnormal to take place in the brain actually reflect neurodegeneration. So the brain neurons, all those cell types are metabolically disregulated. However, their function is also heavily impacted by systemic insulin resistance. And if you add that to an ongoing problem in brain, that's what's driving these increased cases of Alzheimer's, Parkinson's, et cetera, because I didn't show you a graph of it, but there -- if you look at a person who is 70 years old in 1980 versus somebody today, the rates of Parkinson's and Alzheimer's have skyrocket it. And that doesn't make sense if it's only genetic, but what we do know is they parallel with changes in obesity, diabetes, et cetera, meaning that the Parkinson's is there, but it's made much worse and much more common because of the systemic disease.
Will it take away the Parkinson's if you cure the diabetes? No, but it will certainly reduce the onset, severity and really probably pretty much delay the whole process by taking care of those systemic problems. Once the brain cascade gets going, we need to address it specifically with medications that attract the brain.
And you mentioned earlier that what you saw in the brand, kind of the mechanism, the pathway of what affects the brain affects the rest of the body as well, right, in other diseases. Wouldn't that be fair to say that the big that if you could affect -- if you can reverse the metabolic dysregulation you could basically affect multiple diseases at once, not just Parkinson's or Alzheimer's in particular.
No, absolutely. I think that our mistake, whether you're dealing with the brain, the liver, the kidney, whatever his metabolic is disregulated, we're going to have to cross talk and borrow from one another because the mechanism seems to be quite related.
The big deal will be, can you get those drugs across the blood-brain barrier? Can you make the more specifics of the brain is targeted more directly than say, skeletal muscle? If we think about -- I'd like to think about it as atherosclerosis, we don't really think of athero in the heart, the kidneys, the aorta as being different. It's all the same thing. It's just a real estate problem. And so the consequences are different because of the organs.
In the brain, we really do have to address what's going on in the brain specifically, but we also need to address the systemic problems because they're making the brain worse.
Right. Thank you for that. I'd like to next go on to Clarence. You heard De la Monte said it's all about metabolic dysregulation, insulin resistance and so forth, but you focused a lot on inflammation and neuro inflammation. Can you help really draw the link between inflammation and insulin resistance for metabolic dysfunction.
Certainly, I mean, they are closely interrelated, and they are mutually inducted. And so wherever you have inflammation in the brain, you will have insulin resistance, even if you do not measure it in the periphery. And I think that's where some people frequently think of the insulin resistance problem in terms of a systemic and in insulin or in model treating Type 1 diabetes, most people mostly think of that. The type 2 diabetes where you have the systemic insulin resistance.
It's a lot -- there's a lot of inflammation that drive type 2 diabetes. And in fact, as term was made for that. And again, when you have oxidative stress, you will have inflammation when you have inflammation, you have insulin resistance. We think with Bezisterim, we have one drug treating by treating inflammation, we treat both sides of that. [indiscernible] information.
Another question for you, Clarence, is your presentation was -- I'm not sure how clearly everybody got the point about DNA methylation and how that is actually accelerating or driving disaster rating diseases or particularly age-related diseases. So can you help -- you talked a lot about age deceleration. That's great. We all would like to be younger, healthier, right? But how does that directly affect individual diseases that we tend to think about?
Well, the role of epigenetics in disease is become increasingly well understood and that -- in the past, people thought of genetics itself in the DNA sequence has been a thing people are more prone to a disease or not, but actually, the dominant factor is the way that DNA gets methylated controls gene expression and the identical twins can have different expressions based on different exposures and environmental exposures. It's DNA inflation is for most of us in the -- typically, things go downhill, okay?
As you get old, you have all this damage, things which alter the methylation, the epigenetic signature for pattern your DNA, and it just gets worse. Inflammation is what drives that. systemic inflammation. TNF is a big driver of that. And the theory of aging or the mechanisms of aging are complex. I mean people are always proposing theories of how they all relate in what comes first, which is the chicken and which is the egg, and how do we move forward. But we know it's a lot of different factors.
They contribute to both -- there are both mutations, real point mutations, which can impact, for instance, TNF expression. And then we have modifications of the expression of these inflammatory factors, which are driven by think of most of the environment. What we experienced either when we find it even stressed like all the people who are in -- grow up in economically disadvantaged environments have more stress and they alter their epigenetic profiles as well.
These things are all related to inflammation and the reversibility of it is the key here. And that once at least there are elements of it that are reversible. And those elements appear from our Alzheimer's results appear to be specifically associated with the disease. And that -- or with inflammatory type diseases. And we know that Bezisterim doesn't just increase or decrease methylation nonspecifically, it doesn't in a way that's extraordinarily specific.
And we, of course, can't understand how it does so in each instance because of if you want something complicated to -- if you want to be confused, start studying gene regulation, the involvement of epigenetics and things, it's not very intuitive, but it is for people spend their lives trying to understand how a gene or a system of genes are regulated.
We happen to have an activity though because the inflammation is at the top of these changes by controlling that, we can control in what seems to be specific ways these changes in a beneficial way only, and that is we have yet defined, things which are moved in the wrong direction.
I assume that you are capturing or collecting a lot of DNA methylation data in your trial. And with that, how are you trying to link DNA methylation to the physical progression of Alzheimer's -- I'm sorry, Parkinson's disease.
There are a couple of ways that we're doing things. One, firstly, this linked to the clocks, like the the age clock, where where others have shown that this site progression is linked to time of debt. We also are looking at specific genes which are associated with Parkinson's pathophysiology and looking, are we going to increase or decrease the expression of those. Those are 2 different types of questions. And 1 is that type where we're looking at specific genes in their function or influence on Parkinson's is more the traditional way of looking at, hey, what could launch progression.
However, the studies that have done, for instance, by Horvath as well and the U.K. biobanks study that where they're looking at here population studies looking at the influence of DNA methylation on time-to-death, progression of disease is a new topic, a new way. We'll be looking at both of those together to try to understand how we are influencing these progression pathways.
And again, progression is the most important thing for Parkinson's. If we can get the drug available to people and using even itself with its a nontraditional therapy for nonmotor symptoms, anything that will allow people to use it. They will have the potential then of significantly arresting the disease, and that's our objective because that's the thing that everyone with Parkinson's needs. And the sooner they get it in after diagnosis or even before official diagnosis, the better off they're going to be.
All right. And now moving on to Joe. Joe, you spoke a lot about the non-motor symptoms of Parkinson's and the fact that it could show up, let's say, early as 10 years before the official diagnosis with the motor symptoms. Why is it not discussed more often? And why does everybody still believe that Parkinson's is fundamentally a motor disease?
In part because we're not educating people particularly well. People know the warning signs of heart attack. People know the warning signs potentially of dementia. We've not done a great job of educating the public or even in the medical community, frankly, on what to look for. Now I'll give the example of Alan Alda who had done some research for a television program he was doing. And in that program, they discussed REM disorder, REM sleep disorder, he noticed that he had it. He was able to engage in treatment much earlier because he was educated as to these kinds of REM disorders can lead the Parkinson's and you should be evaluated.
Had he not participated in that particular educational work, he would not have been able to be diagnosed that early. We don't do a good job looking at these kinds of symptoms. So it's going to require education. I think we can do it. But there also has been a treatment, right? And physicians like to treat things for which they have a therapy.
If we're incredibly fortunate, right? If this all goes well, things will change. Medicine will change. We'll have an agent in which we can treat nonmotor symptoms. If you think about, say, Prozac, we were very reluctant to use, drugs to treat depression because those drugs you can actually suicide on. When a good solution came out, then primary care physicians started diagnosing and now you can't go to a doctor's office without someone screening you for anxiety and depression. And I would imagine that medicine will evolve in that way once someone is able to engage with the early portion of the disease and make folks understand, yes, there is something you can do about it. So it's worth asking the questions.
All right. Thank you, Joe. Tara tells me that there are a number of questions that have been submitted online. So let me turn it over to her for those questions.
Great. Thank you, Cuong. So yes, please hold for a brief moment while we poll for questions from the audience. So our first question, what are your benchmarks for success in the PD trial? And how will you define that success?
Why don't we go back to my very last slide or we don't pull it up, I think too long. Success is identifying a mechanism of action that gives us target engagement that shows us a clear relationship to clinical symptoms that we are able to change and modulate. That gives us a clear path to design of our next study. It's really about taking this very efficient study with the biomarkers, understanding what's happening to genes and gene function, which is really what methylation is about genes create things.
Once we understand all of that in its complexity, then it tells us where to go next, how we can be different. Now we expect to be differentiated. We do not want to leave value on the table. So we're going to interrogate everything. We have access to really great computing capacity. We can look at every single element of data and related to every other element of data. We're looking at over 350,000 gene products or actually genes. We're going to crunch through it all and be able to tell you what the personality of this molecule is in early Parkinson's. And it may be different from what we saw in late Parkinson's and put together a program that takes advantage of what we've learned. That would be a win.
Great. Thanks, Joe. Our next question here, what are the key biomarker results that we should be focusing on?
I'll start, then to Clarence and perhaps Dr. De la Monte. We're looking at metabolic outcomes, and those are standardly evaluated. We're looking at inflammatory outcomes, which can be as simple as looking at your composition of white cells. We're certainly going to be looking at those -- systems biology that underlies at all.
We'll be able to look at what happens to nerves and genes out of that with epigenetic work, and we may have a few other secrets.
Thing I'd like to add, Dr. Palumbo, that the Demaethylation results are really for the program overall. And Parkinson's patients is key because the data, I believe, are compelling that progression is linked to biological age, DNA methylation age. And that is the unmet medical need. I mean obviously, the nonmotor symptoms or something which have not been adequately addressed because the tools -- the pharmaceutical tools are not there. They haven't been there and because they're very difficult to develop without adverse side effects or undesirable side of it.
But for us, DNA methylation, if we have this lowering of biological age as we've seen in Alzheimer's before, we have the first step and a big step to developing a drug to that every Parkinson's patient needs. We use that in conjunction with anything that emerges for a symptom, whether it's motor or nonmotor that is developable by the -- in cooperation with the FDA, we have then a treatment that everyone will need or want.
And Dr. De la Monte, what would you look for a few you are giving me advice. So what advice would you give relative to this particular question. What should we be looking at?
So one of the things we always want to do is to lay back to what we know already and what people are looking at. So we would probably make sure that we obtain the standard biomarkers of the people looking for cynically neuropathies and the like for Parkinson's disease because you always want to take your new findings and relate them to the old findings that people can connect them. I think that's an important component.
The second thing would be in terms of biomarkers of -- it wasn't clear what the oxide is stressing, but there's a lot of lipid peroxidation and oxygen indices that are out there. These are either biochemical or they can be immunomarkers of it. Their panels, I think the goal would be to instead of working one by one model because you have small panels that would kind of collectively tell you that you have an inflammatory, an oxidative stress index that's abnormal, and that with your treatment, they've actually gotten better.
You don't want a delta look at the change over time and how fast it changes and whether people continue to clinically improve a subjective report of how you're doing as well as objective findings in terms of cognition, the motor is easier to look at because those are standardized, but the cognitive piece, behavior, sleep, all the things that are disturbing patients with Parkinson's should be assessed.
Thank you.
Great. Thank you. So a few more questions here before we wrap up. What can be done with patients that are 5 to 8 years down the line?
I mean are ideal for the treatment because basically, that's the window where their cognitive impairment is starting to show up. So I would think it would be ideal candidates for intervention because thus far, we don't have anything.
Yes. And we can reflect on our earlier study and folks who are maybe a little further along in 5 to 8 years, but who were sort of wearing out of their medicine, we were able to show some interesting effects there and motor as well as nonmotor. Obviously, we want to replicate that. But I think those are good signals.
At any point, no matter how far down the road the patient is, slowing progression that Parkinson's is good. It's better than having the disease move forward because Parkinson's symptoms into late stage are especially horrific. And improving cognition to the degree we can and all the non-motor symptoms that will -- that are apparent all good, there is no downside in making a patient better or slowing the progression of the disease, no matter how far along they go.
Tara. Do you have another couple of questions.
Yes. So that's actually all the time that we have. So I'll turn it back to Cuong for quick closing remarks.
Thank you, Tara. First of all, thank you, Dr. De la Monte, Clarence, Joe, for walking us through our discussion today. It's been very, very helpful. Thank you, everyone, who joined us. I hope you put something away from this conversation. I certainly did.
Let me just share with you my take away from the last hour, I took away following points. First, from Dr. De la Monte, is that disease is really has its roots in metabolic disregulation. Many diseases start there and we displayed through insulin resistance and so forth that metabolic dysregulation is the underpinning of many human diseases has been one.
The second, we heard from Dr. De la Monte and in clearance is that inflammation is that often at the root of this, and information often goes hand-in-hand with insulin resistance, right? It's practically impossible to have inflammation without having insulin resistance and vice versa. And that's why it inflammation and its own resistance becomes a treatment target for many diseases. And if you have insulin resistance, net of by disregulation, you're not going to have just one disease, you're going to have multiple manifestations of diseases out there, right?
The third thing I took away, unfortunately, is that we are under informed, we are not sufficiently well informed about the nonmotor symptoms and the needs to treat the nonmotor symptoms of Parkinson's, Joe told us about how those symptoms could show up as early as 10 years before the motor symptoms. But unfortunately, the community just has not been very well informed to look for them, partly because there hasn't been a drug available to treat those nonmotor symptoms, right?
And the last point I took away from Joe's slides is that in Bezisterim, there is the potential for the first time to go and address both the motor and the nonmotor components of it, right? And the trial, the last patient has come in for his last visit. The team now is going through the data cleanup process. And as Joe mentioned, there are hundreds of thousands of genes and so forth that the team needs to kind of compute the study, we need to wait for the biomarker information to come back from the various vendors and labs and so forth.
And as such, we will -- we hope to have top line data readout. We hope to have the results from the trial and now it's by the end of second quarter, although that may slip into third quarter a bit. Time will tell, all depends on what we -- how long it takes to get things back in the lab and how long it takes to analyze the mountain of data that we have. So with that, I thank you, everybody, for joining, and you have a great day.
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BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Hello. This is Craig Brelsford with RedChip Companies. Thank you for joining today's event with BioVie, which trades on the NASDAQ under the ticker BIVI. With us today, we have Cuong Do, President and CEO of BioVie. We will begin with a brief presentation in a moment and then we will answer your questions. Submit your question at any time by using the Q&A tool at the bottom of the Zoom window.
Before we begin, please allow me to read the safe harbor statement. This call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results along with other statements about the future expectations, beliefs, goals, plans or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact should also be considered forward-looking statements. Of course, forward-looking statements involve risks and uncertainties.
Cuong when you're ready, please go ahead.
Thank you, everyone. Thank you for joining today. My name is Cuong Do, I am the CEO of BioVie. At BioVie we have 2 assets that are currently in development right now. Our lead asset is a drug candidate named bezisterim, that's formerly known as NE3107. It's known as a modulator of inflammation. So it modulates the production of TNF alpha. In various clinical trials, many patients treated with bezisterim have experienced reduced inflammation and the associated insulin resistance.
In our Parkinson's trial, Parkinson's patients have seen improved muscle control. in our Alzheimer's trial, Alzheimer's patients have been seen improved cognition and function. And all these patients have also experienced lower levels of DNA methylation, which is essentially in modulation of the biological aging process.
Our second drug candidate where the heritage at the beginning of the company is a drug called BIV201, which is a novel formulation of a drug called [indiscernible], which has the potential to become the first therapy for ascites, which is a horrible end-stage liver disease commission that has greater than 50% mortality rate within a year.
Before we go into the detail, let me give 2 near-term updates on expectations. We have 2 near-term catalysts. As you know, our Parkinson's trial has been fully enrolled since the end of 2025. And those patients are continuing on through the trial. And as such, the last patient is expected to come in for his or her last visit at the end of April. This basically puts us in line to have top line data readouts from this trial in the second quarter of 2026. So that's coming up very quickly, right? So I'm hoping that, that will come by June, hopefully by May. So second quarter of 2026 is when we expect to have top line readout from our Parkinson's trial.
The other guidance -- new guidance that we're giving, is that our Long COVID trial is expected to have top line data readout towards the end of the summer of 2026. This trial has been enrolling very rapidly. Now that all of our large academic medical center partners are online, the large Long COVID sites at Mayo Clinic Yale, Stanford, [ UCSF ], Mount Sinai are all online. They are actively recruiting and enrolling patients right now in that trial. And as such, at the pace that we're going, we expect to have the last patient enrolled by the end of March and perhaps early April at the latest. And that would mean that we will have top line data readout, let's say, in the August time frame of this year. So exciting few months coming up for the company with 2 top line data readout of our Parkinson's and our Long COVID trials.
And as you may recall, we are all about inflammation. Bezisterim blocks the production of TNF alpha and TNF alpha is considered to be the master regulator of inflammation when you have TNF alpha produced, it goes on to create more inflammation creates a forward-feeding pro inflammatory cycle where it produces more [ chemokines ], cytokines and so forth that leads to more inflammation all of which leads then to more TNF alpha. And of course, this has been associated with a large number of diseases. TNF alpha has also been known as a key driver of insulin resistance which, of course, leads to diabetes and various metabolic disorders. TNF alpha has been implicated directly in the driving the serine kinase that phosphorylates [indiscernible] to become [indiscernible] in Alzheimer's disease. And TNF alpha is the key driver, a key driver of something called DNA methyltransferase 3A and B, which leads to the hypermethylation of our DNA which has been associated with a large number of diseases, particularly age-related diseases.
And bezisterim works to block the production of TNF alpha and it works through something called ERK with the extra cellular regulated kinase. Now it's been known for decades that ERK placed 2 very different roles in virtually every cell in our body. The first having to do with insulin signaling where insulin works through ERK for all of its cellular growth repair and regeneration activities. That's known as homeostatic ERK. But ERK also plays an important role in inflammation, whereby various extra cellular signals or agents such as cytokines and [ chemokines ] will enter the cell activate ERK, which in turn activates NF-kappa B and NF-kappa B triggers the production of TNF alpha. So when you have TNF alpha, it then just goes on and create, release more inflammatory factors and create this forward-feeding pro-inflammatory cycle.
So it's obvious what you want to do. What you want is to block inflammatory ERK but not do anything with homeostatic ERK. But unfortunately, that's much easier second done because dozens of teams over decades trying to do it. But no one was able to succeed. Everyone failed because they were not able to create a selective enough inhibitor of just inflammatory ERK. And frankly, that's where NE3107 comes in or bezisterim. And honestly, this is where we got lucky. We did not set out to do this. we just had our eyes open when we recognize what it does. It turns out that bezisterim blocks the activation of ERK in NF-kappa B. As a result when you do not have NF-kappa B activation, you are not going to get TNF alpha, right?
And so when we thought this, it became very clear that bezisterim has a critical role in a number of different disease conditions. Right now, we are focusing on Parkinson's. It has been known for some time for decades, that 2 conditions need to be present at the same time for a person to develop Parkinson's symptoms. The first is you need low dopamine levels in the brain and the second is to need inflammation and the associated insulin resistance. If you're able to reverse the insulin resistance in the brain, the body is then able to make use of whatever low level of dopamine that is currently there, right? And people have tried for a long time, including doing some really crazy things, far out things like, in this case, using inhaled insulin, just trying to get more insulin nose, get it up into the brain to see if you can refer to insulin resistance.
And it does work in this clinical trial, as you can see here, those that received insulin inhaled insulin we're able to improve their muscle control significantly, but this is a disease score. So a lower score is better. So those that were that were treated with insulin saw an improvement of what's called third unified Parkinson's disease rating scale Part III with the muscle or the motor control. But unfortunately, that is just not a very practical solution, right? You can't easily get inhaled insulin out to everybody and treat it and it's just not as effective as you want it to be. It's not very practical.
So to this day, the only practical solution for Parkinson's patients is to use a drug called levodopa that was introduced over 5 decades ago. Levodopa is a terrific drug. What it does is it enhances the dopamine level that's in the brain and therefore, it enhances muscle control and it does a terrific job at that. But it has several problems. One is that it has a short half-life, which means that patients have to take it multiple times a day. which is very inconvenient. But the real problem comes in is overnight when people don't normally wake up in the middle of the night to take their overnight medication. So that means that by the time they wake up in the morning, levodopa has already worn off. It's kind of out of the patient system. So that means they do not have muscle control, they're rigid, they can't get out of bed.
This is part of the reason why Parkinson's patients don't like the schedule in the first thing in the morning is because they can't get on a bed. So they have to stay in bed, take their medication wait an hour or so for the levodopa kick in before they can actually start to move and get out of bed. So that's one significant problem with levodopa.
The second is that the effect of levodopa wears off over time. So that the longer you're on the drug, you end up having to take higher and higher doses or amounts of the drug. And as you get into the higher doses of levodopa, it leads to something called levodopa-induced dyskinesia, which is essentially the trembling that you see many Parkinson's specials have. And when you have the dyskinesia, all you can do is reduce the dose of above, right, and wait for the tremors to pass. But as you cut back on the dose, you lose muscle control. So these patients are in a terrible situation with a terrible trade-off that they have to make, right? We believe bezisterim could be a solution for that. Because in our preclinical studies in both [ ROTAs ] and nonhuman primates, we saw that bezisterim was equally effective as levodopa in restoring muscle control.
Now that's a huge statement in and of itself because over the decades, no drug has been not equal levodopa, right? And with the support of Michael J. Fox Foundation, we -- the original study showed that bezisterim alone was equally affected as levodopa. But we also found that when you give it in combination, when you give bezisterim in combination with levodopa, you saw a synergistic effect. You saw the greatest muscle control. You also saw a reduction in the dyskinesia, but most importantly, we saw that at the end of the study, when we sacrifice the monkeys and look at their brains. Those that were treated with bezisterim retain twice as many neurons as those were not treated with bezisterim suggesting that bezisterim has a neuroprotective property to the drug, which, to us, is not at all surprising because we know that bezisterim reverses insulin resistance, which means there's greater glucose availability in the brain.
We also note that bezisterim enhances blood flow to the brain, which means -- and when you have creator oxygen available in the brain because of greater blood flow and you have more glucose that's only going to bode well for better neuronal health. So based upon that, we went into the clinic and enrolled 40 patients, whereby frankly, we replicated in Parkinson's patients. the study that we did with but nonhuman primates. We gave half the patients of placebo and levodopa as shown in red here, so essentially just levodopa. And then we get the other half bezisterim and levodopa as shown in blue. And after 28 days of treatment, you see that those that were treated with the combination, saw about a 3-plus point advantage on the most -- on the motor score, the Part III score of the Unified Parkinson's disease rating scale. Now that's a clinically meaningful improvement. And that is a huge deal because this is on top of what levodopa alone, let's say, with the produced, right?
And I've said before, levodopa is a great drug. And with the combination with bezisterim, we make it even that much better. But we also found is that those who are younger than 70 years old, presumably whose disease progression has not done so far. That difference is over 4 points. In fact you don't see that you don't see that very often. So this shows that in humans that you can use bezisterim in combination of levodopa to help moderate and severe Parkinson's patients improve under muscle control. We also found that it has a dramatic impact on the overnight effect, right? So as I mentioned before, we saw in our trial has seen every single day by hundreds of thousands of Parkinson's patients. None of the patients that were given a placebo and levodopa or just levodopa, none of them had control of their muscles first thing in the morning, what's called [indiscernible] muscle state, none at muscle control or could get out of bed, where roughly 1/3 of bezisterim patients, has muscle control and could get out of bed.
So this establishes the first half of what we want to do in Parkinson's. Mainly, we have shown that we have now human proof of concept that we can use bezisterim in combination with levodopa to help moderate in severe Parkinson's patients, improve the muscle control. We completed this trial last year, and that's why we are now on to our second trial and that's the trial that's currently underway. In this second trial, we are enrolling 60 patients. who are earlier in their disease state. These are diagnosed Parkinson's patients who need to go on to therapy for the first time. We are giving half of them just a placebo and then the other half, we're giving bezisterim. And what we're hoping -- what we're trying to show here is that those that are treated with bezisterim has sees a delay of their disease progression.
This trial alone would not show that, right? The next trial, the Phase III trial that we will conduct will be for a longer period of time and explicitly what we're trying to show in these combination of trials is thzt bezisterim could become the first drug to modify the progression of the disease to delay the progression of the disease. And that's really the most important thing we're trying to demonstrate for bezisterim in Parkinson's.
But this second trial establishes the second part of what we want to do in Parkinson's. This second trial is fully enrolled now, and we expect to have top line data readout for this trial in the second quarter, hopefully in the May or June time frame of this year. In the interest of time, let me move on we'll come back and answer any of the questions you may have here.
Then the second trial that we have underway right now is in Long COVID. And you may ask why are we looking at Long COVID and what does this have to do with Parkinson's and Alzheimer's where we have really focused for years. And the short answer is we had no idea that Long COVID was tied to Parkinson's and Alzheimer's. But first, to set the stage Long COVID symptoms are defined as persistent symptoms that last long after the COVID infection has been [indiscernible]. So many of us on this call may have been effective with COVID, after a couple of weeks or maybe longer, the body was able to mount an immune response to essentially get rid of the COVID infection. And for the vast majority of us, our lives continue, right, the infection is behind us.
But unfortunately, right now, there are at least 17 million Americans who continue to suffer the lingering effect of COVID infection, right? And it shows itself in terms of brain fog, extreme fatigue and malaise. Many of these patients cannot stay out of bed for more than a couple of hours at a time. And that's why there's more than 3 million Americans in fact, to quit their jobs or change their jobs simply because they cannot keep up with the physical demand that of the job, right? And there are no approved treatments for this. There are no treatments for Long COVID symptoms. And that's why there are these huge Long COVID centers all around the country, whereby these academic centers are just trying to provide palative or symptomatic relief for these patients because nothing works. And the situation is only getting worse. We estimate that there are tens of thousands of new patients that are getting diagnosed with Long COVID each day, right?
And how does bezisterim comes in? It comes in because it's been shown over the last couple of years that Long COVID symptoms result because there are fragments of the spike proteins and fragments of the envelope proteins continue to circulate around in the person's [indiscernible]. So while it is not infectious, the virus has been killed. So this is just a fragment of the virus that continues to circulate. But the body believes that there's still an infection. So the body is constantly mounting an immune response to the circulating protein fragments. And because of that, it generates a lot of inflammation. Inflammation works through exactly the same mechanisms where bezisterim effects. So namely ERK and NF-kappa B activation.
And because bezisterim blocks ERK and NF-kappa B activation, we believe it may become a mechanism to essentially break the cycle of information, creating more inflammation and more Long COVID symptoms. And we have been granted a $13 million grant to fully fund the Phase II trial that's currently underway. This grant allows us to enroll 200 patients half of whom will be given a placebo and then the other half will be given bezisterim. And we will follow these patients for roughly 4 months from beginning to the end. And the last patient, last visit from our Long COVID trial is expected in the back half of July which means that this trial would be in a position to read out in the August time frame of this year.
For the interest of time, let me just skip ahead -- well, let me just skip spend a few minutes ascites. Our other drug candidate is BIV201, which essentially targets and treats ascites. Ascites is end-stage liver disease. And when you get to this stage, the liver has been so scared and it creates all kinds of portal hypertension. And as a result, fluid cannot flow through the liver the way it normally does and the kidney is implicated, and therefore, the kidney cannot get rid of fluid as well. So all that fluid accumulates in your abdomen, right? So you can see that this patient has a huge amount of fluid buildup, ascites fluid build up. There is no approved treatment for ascites.
And the only thing that you can do right now is stick a large needle into the abdomen and physically remove 5 to 10 liters of that fluid. Like to get it -- procedure [indiscernible]. [ Impersonthesis ] does work. It does provide the patients with some initial belief, symptomatic relief. But since nothing has been done to address why the fluid accumulates to begin with. If fluid can reaccumulate so these patients are back in the hospital every week or 2 to get another 5 to 10 liters of fluid removed. So you can just imagine the stress and strain that this puts on to the body. And frankly, the only treatment objective at this point is to try to keep the patient alive long enough so that he or she may qualify for a liver transplant.
And since there are not enough livers to go around, [indiscernible] greater than 50% mortality rate within a year, right? And this is -- this has a huge unmet medical need. So our solution is a novel formulation of a drug called [ thromopressin ], something that's been available in Europe outside the U.S. for quite some time. It's a drug that acts as a very potent vasal constructor right? So everything your body constructs. But because of that, it has a horrible side effect profile. Our solution essentially solves that problem. So we take [ pelabresib ] and we put it into a saline bag we stick it into -- connected to a portable infusion pump that patients can wear under belt. And we slowly drip the drug in over the course of 24 hours.
So in our first Phase II trial, we showed that we were able to maintain a constant therapeutic dose of the drug by continuously dripping it in slowly over the course of the day. And by doing so, we avoid the problem of creating these massive peaks that are associated with the bolus injections, which is this the way that [ bilipessen ] is administered now, right? So in the Phase IIa, we saw no drug-related SAE or Severe Adverse Events, right? So by avoiding the peaks, we avoided the safety concerns.
Then we moved on to a Phase IIb trial where we were targeting enrolling 30 patients to try to measure how much ascites fluid buildup we can reduce. And we decided to stop the trial early halfway through after 15 patients have been enrolled because by then, we already had data showing that those that completed treatment saw a 50-plus percent reduction in [ duracites ] fluid buildup, whereas those or standard of care solve really no change.
So since this is a condition that has such high mortality, we concluded that the best thing to do, the most ethical thing to do is to stop the trial early and engage the FDA in a conversation on what it would take for us to get -- to move the drug to Phase III and registration. And since we already have fast track and order designation for this drug, all we need is one Phase III trial to register the drug. The Phase III trial for about 200 patients large. And as of now, we have received all the feedback we made from the FDA to initiate the trial, and we're exploring some things to get the funding to initiate [indiscernible] strong.
So in just a time, let me stop here by saying, in BioVie I believe we have a very, very exciting portfolio. We have 2 drug candidates in bezisterim and BIV201. So we have multiple, multiple shots on goal. Bezisterim is currently in the clinic for Parkinson's and Long COVID. The Parkinson's trial is expected to have top line data readout in the second quarter of this year. And the Long COVID trial is expected to have top line data readout by the end of the summer.
So with that, let me stop here, and let's open it up for questions.
Thanks a lot Cuong. Yes, if you wish to reach Cuong, please click on the Q&A button at the bottom of your zoom window, a text box will appear and then you will be able to type in your question.
We already have some questions already submitted. Can't COVID live for a long time in the gut and then pass spike into the system.
Frankly, we know that Long COVID last a long time. It does circulate in the blood from blood, they can go to a lot of different places. And so that's part of the reason why there has been no treatment. And we believe that we may be able to provide symptomatic relief, but there's nothing that bezisterim does that will get rid of the circulating protein fragment. So that will have to come from a different solution. But hopefully, in the meantime, we will provide a symptomatic relief, a solution for symptomatic relief for these patients, right?
And just to give you a sense of how important that is, there is absolutely nothing available for these patients out there. So if we get significant results at the end of the summer, our plan is to go to the FDA in the fall and have a conversation about what it would take to get accelerated approval or even perhaps emergency use authorization for the drug because the mercancy-use authorization is there for situations where there is a big unmet medical need, and there's nothing else that works, right? So depending on the data that we get, we will go and talk to the FDA about that.
And just to kind of give you a magnitude here. We believe that Parkinson's when fully developed could be a $3 billion to $5 billion annual sales opportunity in the U.S. alone loan. And Long COVID could be even bigger than that because of the numbers that are involved, there are 1 million Parkinson's patients in the U.S. There are 17 million Long COVID in the U.S., 3 million of whom have it very severely such that they've had to quit or change their job. So this leads to a very, very large market.
Thanks, Cuong. As a long-term investor, I have been hearing about liver ascites for quite some time. Previously, I was told BioVie was seeking a partner, what progress has been made? And when do you think you can bring this solution to the market?
That's a great question. You share my frustration with the delay in developing BIV201. We have been looking for talking with partners. Unfortunately, the partners we've been talking to has the same problem that we have, which is they are also small cap companies. So we've had significant challenges raising the $25 million that's needed to go and initiate to conduct the trial and get this drug registered. And so we are continuing to do that. But if you are a keen observer of BioVie, you may have noticed that we have also -- we are also considering taking the ascites drug public through a separate company as well. And so we're working it through with our banks and advisers and so forth. And we believe market conditions in the coming months may make it possible for us to put the ascites drug into a separate company and take that a portion of that company public to raise the funds that are needed to go and conduct that trial. So we will hope to have this drug in the clinic this year.
Thanks, Cuong. When the reverse split was initiated last year, the stock quickly fell to previous levels. Do you feel the reverse split was a good decision?
The reverse split is a terrible, a terrible thing to have to do. But unfortunately, it was the only solution that we have. We do not want to do a reverse split nor would we ever voluntarily want to go through something like that. We just had -- we faced a regulatory requirement which was our shares need to be trading about $1 in order to be maintained on the NASDAQ. And as it drifted below a dollar, the only way to get it above $1 is to do to reverse split. And our company like many, many other biotech small cap companies also have seen the exact same problem over the course of the last year, 2 years or so, which is frankly, the lack of interest. So our volume, our trading volume has also dried up as well.
So I am very frustrated with our volume, very frustrated with our share price. I do everything possible to try to get the word out about BioVie but frankly, the entire small cap biotech market is just severely depressed. And I think that's the reason why our share price has been stagnant. And believe it or not, we are in a better state than some of the other companies that we track, right? Which is not great relief, right? What we are obviously hoping for or waiting for is positive results from our 2 clinical trials and hopefully, that will give people to be more aware of what we're doing, more excited with what we're doing and they come into our stock as well.
Another question here about partnerships. How do you envision the structure for any potential partnerships? And again, have you been in any discussions with potential partners?
Over the years, we've been in discussions with a good number of partners and let me answer the question in 2 different parts. First is the question of structure, everything is on the table. Over the years, we've discussed things such as a potential partner licensing our products anywhere to potentially buying out the whole company, right? So everything is on the table regarding this got potential, right? And so we will entertain anything that makes sense for the company. And of course, there's always a question of why has it a big company [indiscernible] deal with us and so forth?
And the short answer to that comes in of the fact that in big pharma, no one has ever been fired for playing it safe. I get it, I've been on that side of the table as well. The world out there is littered with promising Phase III assets in Alzheimer's that ultimately fail and big pharma companies are just waiting for us to complete the trial to show with positive statistical significance then they will slip in, right? We know that there are big pharma companies that are monitoring us. We are continuing to have those conversations to keep the conversations warm. And what they constantly call us is going to do the trial and come back on the data. When you have positive clinical data, that's statistically significant. They want us. They want a completely derisked deal, right? And unfortunately, that just means it's -- the onus is on us to go and do the trials to show that this drug works.
Thanks, Cuong. Why wouldn't a large drug or biotech company take a chance on investing in your company?
Well, the answer is partly what I've just mentioned, which is these big pharma companies are very risk-averse, right? And no one wants to invest in a company in a field that has repeatedly failed right Alzheimer's and Parkinson have had many failures in the past. So people want to play it safe. They want now to wait for a drug that has completed Phase III clinical trials before sweeping in. And frankly, they know they will have to pay a premium. And because by then, once we have a proven drug, we then can run an auction for the drug, to run an auction for the company, and it goes to the highest bidder, right? So everyone knows that they will have to overpay worth a drug in an auction but they're willing to do that to avoid the downside risk of investing in something that fails later on.
Cuong anything happening with legal action against the testing centers in Florida whose [indiscernible] resulted in results, which did not achieve statistical significance.
That's a great question. It has been 2 years since we've had this problem. And over the course of those 2 years, we have been cooperating with the FDA and the Department of Justice and we've been working behind the scenes to urge them to move more quickly. What we know is that the FDA has inspectors out to, I believe, 2 handful of the 15 sites that we reported to them. They have set inspectors out. And there have been a number of what I call 483 site patients, I think that's the number, whereby essentially saying that these sites did not adhere to the protocol did not do things correctly. And as well, there's been a few other kinds of reports and filings that the FDA has put in for many of the sites that they have visited.
What we have been waiting for is for them to initiate a criminal prosecution. We believe a criminal investigation is underway. We will give it a little bit more time, right? And if the FDA of the DOJ does not initiate the next set of litigation we may initiate a litigation ourselves because part of the reason why we wanted to wait and cooperate with the FDA is that they have far more investigative powers than we do, right? And they -- what they do is, frankly, free to us, we do not have to pay money for various investigators and people who go out to the sites and so forth.
And more importantly, they have [indiscernible] power. And we know that the reports at the FDA have sent back to the sites. We get a copy of those through our contracts with the site base if we make it very clear that the sites that received those reports did not follow what they were supposed to, i.e., they're at fault. So we will give it a little bit more time because if the FDA initiate litigation, it will make it easier for us just to jump on their [indiscernible]. But if -- they do not do something within the coming months, we may choose to initiate the litigation ourselves. Hopefully, that explains the question. Thank you for the question.
Cuong this may be a good time to remind everyone that you have prior experience at Merck and Samsung and this person wants to know, given that prior experience, what lessons, including partnerships would you be applying to BioVie's overall strategy?
That's a very broad and big question, right? What I know in my 3 decades of developing drugs. Is that a company is valued based on its track. If you have a drug, especially if you have a drug that's in Phase III or a completed Phase III, that's when the big value inflection comes in. It's point -- it's lesson number one. Lesson number two is data from these trials becomes the catalyst whereby different companies get noticed, not only by investors by potential licensors or acquirers. And if we're able to get more than 2, more than 1, right? So if we get 2 companies that are potentially interested in our asset or our company you have a bidding situation. And when you have a bidding situation for a drug where there are a few alternatives, it very quickly leads to a bidding frenzy I've seen that over and over and over again.
And obviously, that's what we are working to create here at BioVie so that we can get the drug into the hands of the best and the most logical owner because they want have more resources, they can develop a drug and get it off with the patients. And by doing so, going through that process, that would generate significant returns for our shareholders who have been backing us well all this time.
This person writes pardon my ignorance is the top line data for Parkinson's and Long COVID later this year, Phase II results?
I believe the answer to that is, yes. Both our Parkinson's and Long COVID trials are Phase II trials whereby we are trying to demonstrate the magnitude of the impact of the drug. And to a certain extent, safety, although we've already demonstrated safety, we've never seen a safety concern. So while I do not make light of any safety observations, we believe that we have shown bezisterim to be safe. What we need to do now is to show the magnitude of the therapeutic impact that bezisterim has in Parkinson's and in Long COVID.
What that means is we want to measure how much improvement can bezisterim treated patients, experience compared to those who were treated with placebo. And by understanding that different. We then can go and use a standard statistical model to estimate and to determine how many patients do we need to have in the Phase III trial so that we can get the positive results and statistical significance that will be required for us to go and register the drug with the FDA. I hope that's clear.
I certainly think so. So thank you very much Cuong. We've got a few here to choose from. Let's get one about bezisterim here. As you said, you are addressing with bezisterim, Alzheimer's, Parkinson's and Long COVID and longevity. And what is the priority there? What's the hierarchy there looking like strategically?
Number one priority is Parkinson's. Number two priority is Long COVID with [ 1 proviso ]. If we get very strong results and the FDA is willing to give us clear guidance on accelerated approval or emergency use for Long COVID, that becomes the #1 priority. So that -- those 2, the data will tell and frankly, the FDA's reaction will tell. Long COVID is the big wild card because it's the first drug of its kind and we just don't know how the FDA is going to react. Whereas Parkinson's, is the first new drug in Parkinson's in over 5 decades and the registration path, the clinical trials and so forth is fairly clear.
And let me give some guidance. I believe there's a question about how long it will take. So we make you some timing guidance as well. But when we get the positive Phase II data from Parkinson's, it doesn't mean that we can turn right around and start the Phase III trial right away. From Phase II data until such time as we can start the trial, it could take at least 6 to 9 months time for us to plan the Phase III trials to get the clinical sites up and running to raise the funding for the trial and so forth. So it could be 6 to 9 months between second quarter of this year, when we get results until such time as we can start the Phase III.
Once we start the Phase III, we expect that we can have the top line data for the -- for the easy indication, just symptomatic relief, we can have the top line data within 1 year, right, for symptomatic relief. But we will continue that trial probably for another year beyond that so that we can demonstrate that we can help the patients modify the progression of their disease right? So the ultimate trial will last longer, but that's when we get the final ultimate trial, that's where the true value comes in because if we're able to demonstrate, but the first time that a drug can modify the progression of disease. That's the holy grail. I hope that answers your question.
Okay. Well, we have reached -- we're even beyond the 5:00 hour here at Eastern Time. Cuong wrap up here, and we appreciate you taking the time to do today's webinar. For more information...
Thank you, Craig. Thank you, everyone, for joining today. Let me just reiterate and recap that at BioVie, we have 2 drug candidates in bezisterim and BIV201. I believe we have a very exciting portfolio in these 2 drugs that are being developed for multiple different indications. Of course, bezisterim is being developed for Parkinson's, Long COVID and Alzheimer's. Our Parkinson's trial is currently underway, top line data is expected second quarter of this year and when fully developed, Parkinson's could become a $3 billion to $5 billion annual sales opportunity in the U.S. alone.
The Long COVID trial is expected to read out at the end of the summer. And if it could become the first therapy for Long COVID and when fully developed, that could cross the Parkinson's market size opportunity, just given the need -- the significant need that's there.
And in [indiscernible] or BIV201, we've received all the feedback that we need from the FDA to initiate or to move into the next stage, us getting this thing into the Phase III clinical trial. And when developed, this could become the first therapy for ascites, which is a condition that has greater than 50% mortality rate within 12 months. At this, we believe, can become a $2 billion annual cell drug in U.S. alone.
So I thank you very much for your time. And if you're interested in more information about us, please go to bioviepharma.com and that's spelled bioviepharma.com for more information about the company and our clinical trials.
Thank you so much for your time. You have a great day.
Thank you Cuong. And just a bit more information. There's another page you can go to. Certainly, you can go to the page that Cuong just gave you. RedChip has a Page too biviinfo.com that can also lead to BioVie corporate page. But when you're on biviiinfo.com, you can also sign up for news alerts on BioVie. You can read the investor presentation. as, of course, you can also do on the corporate website.
We also have a fact sheet that we create exclusively for BioVie on that web page, you can download it very easily. You can also reach us if you want more information on BioVie at [email protected]. Of course, you can always call us at 1800 RedChip. Please watch small stocks big money, RedChip's program featuring exciting small cap companies, including, of course, occasionally BioVie every Saturday night at 7:00 p.m. Eastern on Bloomberg USA.
And finally, please join our next webinar with Jackpot Digital that will be tomorrow at 4:15 p.m. U.S. Eastern. Just like today, register for all RedChip webinars at redchip.com/events. Thanks again to our many participants today. And as always, thank you so much, Cuong.
Thank you, everyone. Have a good day.
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BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Hello, everyone, and welcome. This is Paul Kuntz with RedChip Companies. I want to thank everyone for joining today's event with BioVie. BioVie trades on the NASDAQ under the ticker BIVI. With us today, we have Cuong Do, President and CEO of BioVie.
We will begin with a brief presentation in a moment and then we open up the event for your questions. You can submit your questions at any time by using the Q&A tool at the bottom of the Zoom window.
Before we begin, please allow me to read the safe harbor statement. This call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements pertaining to future financial and/or operating results, along with other statements about the future expectations, beliefs, goals, plans or prospects expressed by management constitute forward-looking statements. Any statements that are not historical facts should also be considered forward-looking and, of course, forward-looking statements involve risks and uncertainties.
With that, I will now turn the webinar over to BioVie's President and CEO, Cuong Do. Please go ahead.
Thank you, Paul. Thank you, everyone, for joining today and good afternoon. I'd like to give us an update and a bit of a recap of where we stand over the course of the year. Just a reminder, we have 2 assets in the company. Our lead asset is a drug called bezisterim, formerly known as NE3107. It is a novel inhibitor of TNF alpha and in clinical trials that's been shown to be able to reduce inflammation and the associated insulin resistance. Our Parkinson's patients have seen improved muscle control in our earlier Phase II trial. Alzheimer's patients have experienced a 68% slowing of cognitive decline after just 6 months of treatment, and everyone has experienced lower levels of DNA methylation, essentially a modulation of the biological aging process. We can go into some of this later as part of the Q&A, if you like.
Our second drug candidate that we don't spend enough time discussing is BIV201. This is essentially the origins to company. This drug is being developed for ascites, which is late-stage end-stage liver disease. It's a condition that have over 50% mortality rate within 12 months, right? So it's a horrible condition, then we'll spend a little bit of time discussing today.
Before we go into each one of these, I'd like to give a bit of a status update, so a timing update and outlook for what to expect over the course of next year. First is our Parkinson's trial. We are currently conducting a Phase IIb in Parkinson's, as you know, and we'll go into greater detail on that program in a minute. But the update is that program is now almost fully enrolled. We are almost complete with enrollment and we expect a full enrollment by the end of this year. And this is a 3-month treatment duration trial, which puts us in a position to expect to have top line data readout in May 2026.
So Parkinson's is nearly fully enrolled, we expect full enrollment by the end of the year, top line data readout in 2026. In addition, we are currently conducting a Long COVID trial. Enrollment in this trial is accelerating. As of now, almost all of our clinical sites are fully up and running and active. I believe that only one academic site is still in the late stages of getting ready. We expect full enrollment of this trial in late February. And as such, we expect top line data readout in summer of 2026. As the trial proceeds, we accumulate -- we monitor what's going on with the subjects on a daily, weekly basis. First off, we have to monitor for safety, just to make sure that there's nothing crazy that's going on. And there's been no safety concerns in what we see so far.
So blinded data is accumulating on a daily basis. And we would expect to have sufficient blinded data in late first quarter to be able to start to see if there are any patterns on the efficacy side of the equation. And what we're hoping to see, of course, is a spreading, a spread to start to develop, right? We hope to see that those -- a group of people who are getting better and then there's a different group of people who are getting worse or not improving. If there's a beginning to be of a spread, that starts to give us a signal that the drug may be doing something and that may give us a sense of what to expect when the data becomes fully unblinded, right?
I underscore May here because when data is blinded, there are still many things that could go wrong, right? But if there's no spreading patterns and so forth, that gives us a different thing to start thinking about versus when there's a spread in the pattern of the data that we start to see.
And lastly, on our ascites program, you may recall that we terminated the trial early so that we can engage the FDA in a conversation on what it would take to move to Phase III and register the trial. As of now, we have received all feedback that we need to move into Phase III from the FDA. And since we already have orphan and fast-track designation, we only need one Phase III trial to be completed and to register the drug. And that's up now. We are exploring partnering and other funding options that we hope to give a further update in the first half of next year, right? So that's the quick update. Now let's go into deeper a bit.
As you know, we believe that inflammation is the root of all things that start to go wrong in the body, and it really starts with TNF alpha. TNF alpha is considered to be the master regulator of inflammation. When you have TNF alpha it drives the release of a bunch of cytokines, chemokines and other things that leads to a pro inflammatory inflammation cycle, which has been implicated by a number of these diseases that you see here, right?
And once you have TNF alpha and inflammation, it drives something called IKK and JNK, which leads to insulin resistance, which, of course, is associated, implicated in diabetes, metabolic diseases and Parkinson's. We'll come back and discuss Parkinson much more, right? TNF alpha draw also drives something called DNA methyltransferases 3A and B which drives DNA methylation, which essentially is playing with the biological aging process. And it turns out that our drug candidate affects inflammation in just the central node and involving ERK and NF-kappa B.
It's been known for decades now that ERK plays 2 very different roles in virtually every cell in our body. The first involves insulin signaling. Insulin goes through a series of steps involving insulin substrate receptor 1 and 2 IRS 1 and 2, and eventually activates ERK for all of its cellular growth repair and regeneration activities. This is known as homeostatic ERK. But ERK is also involved in inflammation, right? So different extracellular signals such as cytokines, chemokines, who enter the body will activate MEK and activate ERK. When ERK is activated, which then that incurs activate NF-kappa B and triggers the production of TNF alpha. When you have TNF alpha, it creates a forward-feeding pro inflammatory cycle that leads to more inflammation.
So it's obvious what you want to do. You want to block inflammatory ERK, but not touch homeostatic ERK. Unfortunately, that is much easier said than done because dozens of teams have tried over the years, but they all failed. All of those drug candidates failed due to toxicity because no one was able to create a selective enough inhibitor of just inflammatory ERK. It was touching insulin signaling as well. And whenever you touch insulin signaling, you're going to create toxicity problem. And that's where bezisterim comes in, right? And to be frank, we did not set out to do this. We got lucky, right? We just had our eyes open when we observed this effect.
It turns out that bezisterim blocks the activation of ERK and NF-kappa B, as you can see here. And when you block the activation of NF-kappa B and ERK, you do not have the production of TNF alpha, right? And when you do not have TNF alpha, you do not have insulin resistance, right? As I mentioned before, insulin resistance is triggered by TNF-alpha, activating ERK and NF-kappa B. It is triggered by the insulin substrate receptor 1 and 2. It turns out that naturally insulin binds insulin substrate receptor 1 and 2 on a tyrosine residue, which then goes through this natural process here. But when you have inflammation, JNK and NF-kappa B also activate and it binds to the IRS 1 and 2 on the serine residue, right?
So when the serine residue is activated and occupied, it blocks the tyrosine site and insulin is not then able to bind into the tyrosine site. And that's how you have insulin resistance, right? It's inflammation, activating JNK and IKK, activating the serene site and IRS 1 and 2, which then creates insulin resistance. And by bezisterim's ability to block NF-kappa B and thus the production of TNF alpha, it's believed that we're actually alleviating the activation of JNK and IKK. We're opening up -- we're freeing up the serine site, opening up the tyrosine cytokine.
So I know that's a lot of science and a lot of mechanism there, but that's critical to understand how the drug works. And I have to first turn to Parkinson's. Two things need to be true at the same time for a person to develop Parkinson's symptoms. And this has been known for decades. The first is that you need to have low dopamine levels in the brain and then the second is you need neuro inflammation and the associated insulin resistance in the brain. If you're somehow able to reverse the insulin resistance in the brain, the brain is then able to make use of whatever level of dopamine that's there and help to restore muscle control.
So people have tried for a long time and tried lots of different ways, lots of crazy things as well to try to reverse insulin resistance in the brain, including this study, which used inhaled insulin just to try to get more insulin through the nose up into the brain so that you can free up more glucose. And it works. If you're able to get enough inhaled insulin up into the brain, you create more glucose and the brain then is able to make use of the dopamine that's there. That's what you see here. But of course, that's not a very practical thing -- solution to use, right. Inhaled insulin to treat Parkinson's. It's not a very recognized solution. And nothing else has been very practical.
And as a result, the drug levodopa remains the standard of care for treating Parkinson's over 5 decades after it was introduced, right? Now levodopa is a great drug. It is a really, really good drug in restoring muscle control, and that's why it remains a standard of care. But there are a few problems associated with levodopa. The first is that it has a short half-life. Such that Parkinson's patients end up having to take it every 3, 4 hours or so which then becomes a real problem overnight when you go to sleep, right. When you go to sleep, levodopa wears off, so that in the morning, you're rigid, your muscle is rigid, you can't get out of bed. So that's one problem associated with levodopa.
The second is that the effect of the drug wanes over time. So the longer you take the drug, the higher the dose you need to have just to get that effect. But as you get into higher and higher doses because of something called levodopa-induced dyskinesia, which is the tremors that you see many Parkinson's patients have. And when you have the dyskinesia, all you can do is to cut back on the levodopa dose and wait for the dyskinesia to pass. But as you cut back on the levodopa, you lose muscle control and you are rigid, right? So it's a terrible, terrible situation that Parkinson's patients have faced, right? And that's why that we believe that bezisterim may play a role in addressing this problem.
In our preclinical research, we found that in mice and rodents, and in nonhuman primates, in monkeys. We found that bezisterim was equally effective as restoring muscle control as bezisterim. Now that is a huge statement right? Because over the decades, nothing has been able to equal levodopa in its restoration of muscle control. But here, we found that bezisterim alone was equally effective as levodopa in restoring muscle control in rodents and in monkeys. And what we also found is when you use it in combination, when you give bezisterim in the combination of levodopa, you saw a synergistic effect. We saw a significant -- a statistically significant improvement in muscle control when used in combination, right?
So this is a disease score, so a lower score is better. The other thing we found is that when we use it in combination, we saw a reduction of dyskinesia. This is a very complicated chart to read, right? So when those monkeys that were given a placebo and levodopa right? So levodopa alone, as shown in the thin green line here, you see many abnormal involuntary movements. So very many tremors, right? So the distribution skews to the right, but when you give bezisterim and levodopa, you see that the distribution skews all the way to the left. It dramatically reduced the incidence of dyskinesia.
And what got most people, all of us, very excited is that at the end of the study when we sacrificed the monkeys and looked at their brains. We found that those that were treated with bezisterim retained twice as many dopaminergic neurons as those that were in control, suggesting that there is a neuroprotective property to the drug. And this to us is not at all surprising, right? Because we know that bezisterim reverses insulin resistance, which means that there is greater glucose availability in the brain.
We also know from various imaging studies and other ways that bezisterim enhances blood flow to the brain, which means there's greater oxygen availability in the brain. So when you have greater oxygen and glucose availability, it only bodes well for better neuronal health, right? So this was terrific work that, frankly, we were pointed in this direction by funding that was provided by the Michael J. Fox Foundation. So we're grateful to them and they continue to help us for our trial today.
So based upon this research, we wanted to go into the clinic. And so we enrolled 40 moderate-to-severe Parkinson's patients and essentially replicated in human patients, a study that we did in monkeys, right? So what we did is we gave half the patients placebo and levodopa, essentially just levodopa alone, as shown in red here. And we get the other half, bezisterim and levodopa. So the combination is shown in blue. And after 28 days of treatment, we saw that those that we treated with the combination experienced a greater than 3-point advantage on the Part III score or the muscle or motor score of unified Parkinson's disease rating scale.
Now that's a clinically meaningful difference. But if you look at patients who are younger than 70 years old, which is about half the population that was in this trial, that difference was somewhere between 4 and 5 points. Now that's a big difference that you do not see very often. But the other thing that was very terrific to see has to do what's called the morning on symptoms. As I mentioned before, levodopa wears off overnight. So this happens every day in life and have been in this trial. In this trial, patients would come into our sites, they would take their evening medications and go to bed. And then first thing in the morning before they take their morning medication, we would measure the muscle control again. And what we found is that none of the patients that were given to placebo and levodopa, so just levodopa alone. None of those patients have their muscles in the on state, none of them could move, none of them could get out of bed.
So their muscles were considered to be in the off-state, but about 1/3 of the patients that were given the combination of bezisterim and levodopa had their muscles in the on state, right? And this was an unexpected statistically significant finding, right? This was a small trial, so we did not expect statistical significance on this, but we had statistical significance, partly all driven despite the magnitude of the impact that bezisterim has. So this gives us human proof of concept that you can use bezisterim and levodopa to help moderate and severe Parkinson's patients improve their muscle control, and it establishes the first half of what we want to show in Parkinson's.
We are -- right, which is you can use bezisterim and levodopa in combination to help moderate-to-severe Parkinson's patients. We are now embarked in the middle of another trial to establish the second half of what we want to show, which is bezisterim can be used as monotherapy. So it can be used alone earlier in the disease progression, right? So we are now enrolling 60 patients in a trial of Parkinson's patients who needs to go on to treatment for the first time to treat their disease symptoms. Half of patients will be enrolled, it will be randomized to placebo, and then the other half will be randomized to just bezisterim alone. This trial will run for 3 months and what we're hoping to show is that those patients that are treated with bezisterim will have a slowing of their loss of muscle control, right? Whereas we believe that those on placebo will have greater muscle loss -- motor control loss, right?
And the other thing is that we expect -- we think what may happen is that a greater proportion of patients that are in the placebo arm may need to drop out of the study as well so that they can go on to levodopa, right? This is a short and small study. It is only 3 months long. It's only 60 patients large. In many ways, we do not expect to see statistical significance in the Part III score here because it's an exploratory study. And what we're hoping to see is the magnitude of the impact at the Part III score so that we can power the Phase III study. But we are fairly confident that when we'll see statistically significant changes in many of the component pieces that lead that contribute to the Part II score, the Part III score and the various other endpoints that we are doing.
So this trial is almost fully enrolled, right? And we expect this to be fully enrolled by the end of the year. We expect to have data coming out of this trial in the May time frame of next year that would allow us to power the 2 Phase III trials that we would expect to run. The Phase III program will have 2 parts to it. The first part would demonstrate symptomatic relief, right? So that would be a short 3, possibly 6-month study to demonstrate that we could give symptomatic relief to new, to patients who need to go on to therapy for the first time. But we will continue to follow these patients in an open-label study for another year in anticipation of demonstrating disease modification, right?
And when taken in totality, we believe that this would allow bezisterim hopefully to become the first newly -- the first new therapy for Parkinson's patients in over 5 decades. The first new therapy that would be able to restore muscle function in a manner that's equal to or better than levodopa, and also could be used in combination with levodopa for more severe patients in the disease -- with the disease. When fully developed, we believe this could represent a $3 billion to $5 billion annual sales opportunity in the United States alone, right.
With that, let me move on to our work in Long COVID, right? And you may think and ask the question, why Long COVID, what does Long COVID have to do with Alzheimer's and Parkinson's. And the short answer is really very little, except the mechanism of action, and it's also CNS related. Long COVID is defined as having persistent symptoms, at least 3 months after the infection has been beaten, but many of us have moved on. The world has moved on, believing that Long COVID -- believing that COVID has been beaten. It's behind us. But the reality is thousands of patients are still getting infected with COVID every single day in the U.S. right now, right? And 17 million Americans are currently suffering from Long COVID symptoms, primarily brain fog, fatigue and malaise. And roughly 3 million of these Americans have the conditions so severely that they have had to quit or change their job simply because they can no longer keep up with the physical demands of the job, right?
There are people who have difficulty staying out of bed for more than 2 or 3 hours, right? Or people who just cannot focus and have the cognitive function that they used to, right, 3 million. And this is part of the reason why there are so many large Long COVID centers out there, particularly at academic medical sites, right? We did not know any of this, right, until some of the experts we were working with turned us on to it, right? We were really focused on Alzheimer's and Parkinson's. But what the experts really turned us on to is that within the last couple of years, researchers have tied Long COVID symptoms to inflammation. And it's really working through 2 things: the spike protein and the envelope proteins.
What happens is that for these 17 million and perhaps even more Americans even after we have beaten the infection, you could have fragments of the spike protein and the envelope protein continuing to circulate around in your bloodstream, in your body. And as a result, the body's immune system believes that there's still an infection. So it's constantly mounting an immune response. And as such, it's creating inflammation, okay?
Because the spike protein is working through the pathway TLR4 or toll-like receptor 4 and then the envelope protein is working TLR2, both TLR2 and 4 is a couple of steps upstream from the activation of ERK and NF-kappa B. And that's the reason why we and many other experts out there believe that there's good reason to believe that bezisterim may be able to block the activation and the production of TNF alpha and block inflammation for these patients.
We were made about known -- we were alerted to this by our experts. And they also told us of the various grant programs that were available out there. So we applied for a grant and we were given a $13 million grant to fully fund an exploratory trial in Long COVID, right? This grant allows us to enroll over 200 patients. Half of the patients will be given placebo and the other half will be given bezisterim twice a day. And as of now, this trial is enrolling very rapidly. Acceleration -- the enrollment is accelerating. We have one academic site that is still in the final stages of getting activated. And as such, we expect this to be fully enrolled by the end of February, which puts us in a position to have top line data readout in the summer time frame.
To be fully transparent, we do not know what to expect in this trial. We have no preclinical studies to give us a hint. There are no preclinical models of Long COVID that's reliable in this arena. So we do not know what to expect. But mechanistically, we know that this makes sense. But the other thing that we do know is that there are a good number of patients out there who do not qualify for this trial. For example, you may not qualify because of the age, right? And you may not live near one of our sites. So over the years, we also have had -- we have also supported something called investigator-initiated trials. Where we would support various researchers who may have a plausible theory as to how bezisterim may contribute to addressing a need that their patient may have. And as such, we have supported trials in traumatic brain injury, ALS and so forth.
We've had some researchers come to us asking for support for their patients in Long COVID. So we've had several patients supported through this. And through those IITs, all the patients have gotten better, right? All the patients in these investigator-initiated trials have gotten better in their cognition and their malaise and fatigue. We know of 1 patient who frankly was just not able to stay up for more than 2 to 3 hours at a time. This was a very accomplished person who was older in age, but thus could not qualify for a trial. And just could not stay up and rarely left his house. He got so well that he was able to go on vacation this past summer, right? And we basically -- he and his clinician are very happy with his progress. His various endpoints, the scores that we measure in this, it's all improving, and his clinician is preparing a case study right now that we hope to publish relatively soon, just to tell the world on how this one particular patient is improving, right?
So that is a sample of one. But if that sample, coupled with some of the blind data -- blinded data that we're beginning to see in this trial continues to develop the way that we think it would develop. We believe that there's a very good chance that we may see statistically significant improvement for patients in either fatigue and/or brain fog. So we're looking at all combinations of fatigue alone, cognitive functions alone or the combination of the two. And if should we see that results, statistically significant result in either of those -- in any of those combinations. One thing that we're working towards right now is a conversation with the FDA sometime later next year about an emergency use authorization for an accelerated approval. Because there are many, many patients out there, 13 million -- 17 million patients affected, 3 million very severely.
So the unmet medical need is very high. There are no approved therapy for this condition. So patients really have nothing to help them, right? And that's why the emergency use pathway is there, right? And we are eagerly working and eyeing that pathway as a possibility of what may unfold next year as we unblind the study next summer. So with that, let me stop here and be mindful of time, let me move very quickly on to ascites, which is end-stage liver disease. Ascites is condition that you get to when the liver has been very, very scarred. By this point, you've gone through fibrosis, cirrhosis and so forth and the liver has been so scarred that it is no longer able to process fluid the way it normally does. And the kidney is also implicated at this point in time. So as such, fluid accumulates in the abdomen, right? And since there's no therapy for this, the only solution is to stick a large needle into the abdomen and physically remove the fluid, right?
And since nothing has been done to address why the fluid accumulates in the first place. These patients are back in the emergency room at the hospital every 1 or 2 weeks to get another 5 to 10 liters of fluid removed. So you can just imagine the strain that this puts on to the body. This is just not water, right? This is -- think of it as serum, so there's protein. There's lots of other things that are in, nutrients that are in this fluid. So these patients end up being malnourished and many other things, right? And so frankly, the only treatment objective at this point in time is to try to keep the patient alive for as long as possible so that they may qualify for a liver transplant. And since there are not enough liver transplant to go around the mortality rate in this condition is over 50% in 12 months.
So it's a horrible condition where there's no current therapy. We believe that our drug candidate, BIV201 may be that first therapy. It is a novel formulation of a drug called terlipressin. Terlipressin is a very, very potent vasal constructor that was first approved in Europe in the mid-80s, right? It's -- first of all, it's a peptide. So it has to be refrigerated. It's very potent. So you only need 2 milligrams of this drug. So how it works is that you would measure 2 milligrams and put it into an IV bolus and you give it to the patient, which then causes a severe constriction of virtually all the blood vessels in your body. And this was first approved to treat a condition called bleeding esophageal varices, which is a complication from ascites, whereby the patient is just bleeding out, right?
So terlipressin represents the last chance a clinician has to try to stop the bleeding and prevent the patient from bleeding out, and you would use it despite the fact that this creates a big blood spike of the drug, which then creates all kinds of side effects. You'll put up with side effects if you can try to keep the patient alive. So this has been used abroad for a very long time. So we know that the drug works. But it was not approved in the U.S. up until very recently because of this dramatic severe side effect, right?
And what we do instead is we take the same 2-milligram. Through a novel formulation, we're able to put it into a continuous infusion pump, keep it and use it at room temperature instead of having to be refrigerated. We can use that at room temperature, cook it up to a portable infusion pump and drip it into patients slowly over the course of the day. We conducted a Phase IIa starting to evaluate safety, and we found no drug-related safety concerns. We then moved on to a Phase IIb trial to try to measure the magnitude of therapeutic impact here. So how much can we actually affect the efficacy side?
We intended to enroll 30 patients, but after just 15 patients, we already had statistically significant data to show that those completing treatment saw a 50-plus percent reduction worth of ascites fluid, with just the treatment. Whereas those on standard of care saw no change, right? And in fact, one patient did not have to go back for another paracentesis, right? And since the mortality rate is so high here, we concluded that the only ethical thing for us to do as a company is to stop the study early and to engage the FDA in a conversation to see what it would take for us to go into Phase III trial to get this drug registered. And since we already have orphan and fast track designation, we only need to complete 1 Phase III trial to get this registered.
As of now, we have received all the feedback we need from the FDA to proceed with this program, right? And we are in conversations and exploring various partnering and other funding opportunities to launch this program. We hope to give a further update in the first half of next year as to when this program could be launched. And from funding, from the moment we have funding, we believe this drug could be on the market in as short as 3 years, right, because we only have one trial to conduct. But this is a complicated trial.
This will be a global trial that will be led out of Europe by our principal investigator out of Italy, who is the most experienced person in this field. All right. So let me just stop here and summarize. BioVie has 2 drug candidates. Bezisterim is currently in the clinic for Parkinson's and Long COVID. The Parkinson's trial is expected to be fully enrolled by the end of this calendar year. As such, we expect to have top line data readout by May of 2026. Our Long COVID trial is currently enrolling. We expect that to be fully enrolled by the end of February, which puts us in a position to have top line data readout by the summertime. And in addition to that, our liver disease program or ascites program has received all the feedback we need from the FDA to proceed. We are now exploring funding options, and we hope to start that program sometime next year, and we'll give additional feedback and update in early part of -- the first half of next year.
With that, and being mindful of time, let me open it up for any questions that you may have. Thank you very much.
Great. Thank you for a great presentation, Cuong, as always. We are, as Cuong just said, going to open up the event for questions. [Operator Instructions] What company manufactures levodopa?
Levodopa is currently a generic drug. It has been on the market for quite some time, right? So that is generic. Many manufacturers make it.
And our next question, how can somebody in Virginia get into the Parkinson's study?
It may be too late right now. But the Parkinson's trial actually has a very innovative design and a very unique design, whereby we send patient -- nurses out to see the patients. One thing you should know is that Parkinson's is actually a very difficult trial to conduct because by the mere fact that a person is going into a clinical site, that person thinks that he or she is going to get better treatment, and that may trigger a placebo effect, right? And many trials have failed or have seen less stellar results simply because of this placebo effect.
To avoid that placebo effect, we did 2 things in the innovative design for current trial. One of those is that we send patients out to their homes. So that we can actually keep everything as constant and as stable as possible. And when the nurse goes out to the house, everything is videoed, right? And all the videos, both from the in-home visits as well as those from our in site visits are then centrally rated by a team of 3 expert reviewers. That way, we try to keep everything constant for the patient, and we keep everything consistent across all the sites, all the patients because we have the same 3 people reviewing. I give that context because you can go to our website, there's a place that you can contact.
You can put in a contact to see if you may qualify. And if you qualify, we can send a nurse out to your home in Virginia or anywhere else in a contiguous 48 states in the U.S. But as I said, we are very close to full enrollment right now. So you need to hurry and there's no guarantee that there's still time to qualify for the trial. Hopefully, that answered your question.
And our next question, do you think you have an open-label extension on the current Phase II Parkinson study?
We are currently not planning on an open-label extension. But again, in the design of the trial, we do have a data monitoring committee that is unblinded to the data as we go along. And it may be very possible for any number of reasons that data monitoring committee may come back to us and may recommend doing an open-label extension of the trial, particularly if there is -- if the patients are getting benefit from a treatment, it would be a shame to discontinue treatment that those patients are benefiting. So we may consider an open label, but that really depends on the data monitoring committee.
Thanks, Cuong. And couple of related questions there. Are any Alzheimer's trials under consideration? And where does the Alzheimer's program fit with the Parkinson's trial.
The Alzheimer's program right now is on hold. You may recall that we conducted an Alzheimer's trial a couple of years back and when we unblinded the data, we found that those treated with bezisterim for 6 months saw a 68% slowing of cognitive decline compared to placebo, right? And the #1 reported safety concern was a mild headache reported by fewer than 8% of patients, right? And that mild headache disappeared after a day. And to give you context between those -- for those numbers. You may also note that there are 2 drugs currently on the market to treat Alzheimer's. These are known as antibodies to the amyloid plaques. And as a class, these drugs have been able to demonstrate a 30% slowing of cognitive decline after 18 months of treatment. And that's part of the reason why clinicians question whether or not there's a real therapeutic benefit at all with these drugs, 30% slowing of cognitive decline after 18 months of treatment compared to bezisterim slowing of 68% of slowing of cognitive decline after just 6 months of treatment.
In addition, the monoclonals have significant concerns about safety. Particularly around brain swelling and brain bleeding. And that's why these patients have to be monitored by PET scans. Now I get these numbers, not to mean, not to imply a head-to-head comparison has been conducted. No head-to-head comparison has been conducted. These are just results that have been reported by various studies out there, right, that they are the best that we can have at this point in time to try to draw any kind of comparative -- comparisons. But the problem we ran into then is that we had a problem whereby we had to exclude patients from 15 clinical sites who did not properly follow a protocol. And by excluding those patients, we missed our statistical significance, right?
We are convinced that drug works, but we just missed on statistical significance 2 years ago. So we have every intention of redoing that trial. So what we are doing now is waiting until such time as funding becomes available and we will restart the trial at that point in time. Funding could come from a partner, but it could come from when we have positive results from Parkinson's and Long COVID that may create a catalyst for us to go and raise some additional funding as well. Hopefully, that answered your question.
Thanks, Cuong. And next question, have you had any discussions about partnering with a large pharma company to help fund more accelerated trials?
That's a great question. We've had a good number of conversations with big pharma companies, larger -- big pharma, right? And we know that a number of them are very intrigued with this mechanism and they're waiting for data. They were waiting for the data that we hope to have had 2 years ago. And when we missed on statistical stats, they basically wished us well, urged us to redo the study, and they'll be waiting for us when the data completes.
And I get it. I've been on their side of this table, right? The world out there is littered with promising molecules in Alzheimer's that failed in Phase III. And in big pharma, no one has ever been fired for playing it safe. You only can get fired for playing -- for essentially going out on a limb and losing, right? So that's why big pharma like to play it safe. And they eventually will overpay and so forth, but that's okay. So it's really -- the onus is now on us to go and do the Phase III in Alzheimer's. If we get positive statistically significant data from this Phase II trial, there may be an opportunity for engagement with big pharma, but more likely, we'll probably need to go and do the phase III trial in Parkinson's ourselves as well.
But if we get statistically significant data in Long COVID and guidance from the FDA for how -- for emergency authorization, I think, that is we will be high target for a takeout or a partnering conversation at that point in time.
And our next question, given the changes at the FDA, are you concerned that there will be any delays in obtaining the approvals you need to move forward?
That is always the risk, right? But maybe we are experiencing an anomaly or frankly, what we are experiencing may not be the kind of stuff that makes headlines. But what we're experiencing right now is timely engagement with the FDA. We have not seen any meaningful delays in our conversations with the FDA on the trials that we are conducting or have been in discussions with them, right? So we're hopeful that, that will keep up.
Thank you, Cuong. And back to the previous Alzheimer's drug trial, did you ever take legal action against the testing centers that disrupted the trial?
Great question. This actually ties into the conversation about funding for Alzheimer's drugs. All -- many investors have asked me to basically unleash the lawyers, go after the 15 sites and get compensation that could be used to help support the next Alzheimer's trial. And we will do that. We have not done it so far. The approach that we have taken is to support the FDA and the DOJ, the Department of Justice in investigating these sites. We know that the FDA has set up investigators to the sites that have been the most active enrollers of patients in our trials. We know that the FDA has been handing out 483 citations, which basically says that you failed to adhere to the protocol and did not conduct a trial in the, right? And we're just waiting for them to take the appropriate next step and render their findings.
Because the -- they have far more investigative power than we do. So we'll give them a little bit more time in hopes that they will make their findings public at such time, we will initiate our steps, but we only have so much patients. So we will rethink how long we wait for the FDA and we'll give you more guidance on that over the course of the first half of next year.
Thank you, Cuong. And I think we have somebody with their hand raised. We'll get to you in one moment. Another question we had. You've been talking to major pharma companies for the liver ascites drug for quite some time. Why have there not been any takers?
Great question. I wish I had a good answer to that, too. There is one midsized pharma company that is the most logical partner for us to work with on this program. But unfortunately, they have been held up by their own internal changes, management changes, mergers and so forth. But luckily, that is now behind them. And hopefully, management is able to focus a bit more on some other things beyond the internal challenges. We know that the scientists in those -- in that company really likes this program. We've been in discussion with the scientists for years, and they really want to work with us, but they have never been able to get this thing up and get the attention and support of management.
And now that, that is behind us. We're hoping to essentially get this elevated in management to initiate those conversations. The companies that have shown much greater interest, unfortunately, are smaller companies. And they, like us, have had to go through a very difficult fundraising environment, right? And we need to raise $25 million to really initiate this next phase. And for small companies, raising $25 million to supportive program that you're going to partner on has been a tall order, and that's the reason why all these companies have been trying to raise the funds and have not been able to raise the funds to come back to us to initiate this program.
Thank you, Cuong. And we're going to go to Mike, you can help me here to let -- [ Mike Samuels ], you have a question here.
2. Question Answer
Cuong, a quick question. In the past couple of years, we've been doing these tests. We've had multiple reverse splits, multiple money raising. Currently, do we have enough funds on hand to get us through how far?
Yes, great question. Thanks for that. As of now, we have sufficient cash to go to 4Q of next year towards the tail end of 2026, right, which is plenty of runway to allow us to have the 2 data readouts that I mentioned before. The Parkinson's readout is expected in May, the Long COVID readout is expected in the summer, right? We do not anticipate needing to raise cash between now and those readouts. I can never say that we will never do it. Somebody comes to me with an offer that cannot give up, cannot refuse, I'll consider it, right? But at this point in time, we are not considering nor are we actively trying to seek additional funding. We have sufficient cash to go beyond the 2 catalysts that we expect for next year.
Thank you, Cuong. And coming up on an hour now. So I think we will just try to wrap things up here. Just sort of a final question. What milestones should investors expect over the next 12 months?
We have a good number of milestones coming up and this is part of the reason why I believe BioVie represents a terrific investment opportunity for investors who have a 12- to 18-month planning horizon. We have a Parkinson's trial that is almost fully enrolled. That trial is expected to read out in May, which will provide a catalyst at that point. Our Long COVID trial is expected to be fully enrolled in February, which puts us in the position to have top line data readout in the summer. We also have an ascites program that is frankly the most derisked program I've seen in my career of developing drugs, right? And we are exploring a number of funding opportunities that may be able to allow us to start that trial sometime next year.
Any one of those 3 programs will create significant upside for shareholders and put the numbers on to these. Parkinson's, we believe, could become a $3 billion to $5 billion annual sales opportunity in the U.S. alone for bezisterim.
Long COVID, if you just take 10% of the 3 million Americans who suffer from the most severe form of Long COVID. This could become a multi-tens of billions of dollars drug as well. And the ascites drug could be easily $1.5 billion to $2 billion annual sales drug. In any way you want to discount, risk adjust these drugs. It will lead you to a value that is significantly more than our current market cap of $10 million to $15 million. We unfortunately have been left for dead and essentially been ignored just like many other micro-cap biotech companies. We are trading, our market cap is less than half our cash, right? And that's the reason why we spend a lot of time trying to tell the market about our story, and we try to prepare everybody for the catalysts that are coming in 2026.
So 2 major data readouts in 2026. That would hopefully provide catalyst for our share price in our stock. With that, I thank you very much for your time this afternoon. If you are a current investor, thank you for your support. We hope that next year will be a pleasant one for our shares, right? And the -- for those that are not shareholders, really study what we do and considering -- weigh the facts. And we hope that you fully get to understand our story.
There was a question we did not get around to as to why there has not been more insider buying. The reality is I have participated and bought every time that the lawyers have allowed me to do so, right. I am a strong believer in this drug, not only because I'm the CEO, right? I'm putting my money where my mouth is. At one time, I was one of the largest shareholders in the company as well. But because I have nonpublic material information, I often cannot invest and buy more shares, right? And that is the same with much of the management team.
All right. So with that, I thank you for your time. I wish you a great day. Thank you very much. Bye-bye.
Thank you, Cuong. And for our audience, if you do -- for more information on BioVie, you can always give us a call here at RedChip at 1-800-REDCHIP or you can e-mail us at [email protected], which is the ticker symbol [email protected]. And of course, you can also visit the redchip.com website to the BioVie page at biviinfo.com. There you can actually view and download the investor presentation, fact sheet. You can also sign up for news alerts to stay abreast of all the developments as they happen.
With that, I want to thank all of our attendees for their time today. And again, thank you, Cuong, for once again joining us, and we look forward to having you back.
Thank you. Have a good day.
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BioVie Inc — Special Call - BioVie Inc.
1. Management Discussion
Hello. This is Craig Brelsford with RedChip Companies. Thank you for joining today's event with BioVie, which trades on the NASDAQ under the ticker BIVI. With us today, we have Cuong Do, President and CEO of BioVie. We will begin with a brief presentation in a moment and then we will answer your questions. Submit your question at any time by using the Q&A tool at the bottom of the Zoom window.
Before we begin, please allow me to read the safe harbor statement. This call may contain forward-looking statements within the meaning of the Private Securities Ligation Reform Act of 1995. All statements pertaining to future financial and/or operating results, along with other statements about the future expectations, beliefs, goals, plans or prospects expressed by management constitute forward-looking statements. Any statements that are not historical facts should also be considered forward-looking statements. Of course, forward-looking statements involve risks and uncertainties.
I now turn this webinar over to Cuong. Go ahead.
Good afternoon, Craig. Thank you for joining. Good afternoon, everyone. Thank you for joining our update today. I'm very excited to share with you this afternoon some exciting progress at the company, right?
As you know, we have several clinical trials underway right now, which makes it -- I believe we have one of the most exciting biotech companies around with an exciting portfolio. You may know that we have a Parkinson's clinical trial that is underway. It is a Phase II trial, and now we can pick up -- enrollment has picked up relatively quickly within the last few weeks, and we can now project to be fully enrolled by the end of 2025. As a result of that, we would expect top line data readout in the May April time frame of 2026. So this is a very exciting progress for us, and I'll describe more about this trial in a moment.
You also may note that we have a long COVID Phase II trial that is underway as well. That trial is enrolling very well, and we expect to have further guidance when that will be fully enrolled in about a month. The enrollment is going very well. We just really want to see how that is coming along in the next few weeks and when we'll get full guidance at that point in time.
We also like to announce that our ascites Phase III clinical trial has been submitted -- protocol has been submitted to the FDA, and we should hear back from the FDA any moment now. We submitted this 3 weeks ago, and we typically hear within 30 days. So that 30 days should be coming up next week. And the exciting thing to announce is that we have significant cash runway. We have sufficient cash to go to the end of 2026, which is, of course, well beyond the timing of when the Parkinson's and the long COVID trial are expected to read out.
So just an overview. As you know, our lead asset is a drug candidate known as bezisterim that modulates the production of TNF-alpha, which is considered to be the master regulator of inflammation. In clinical trials, bezisterim has shown to be able to reduce inflammation and the associated insulin resistance.
In our Parkinson's trial, Phase II trial, Parkinson's patients have seen improved muscle control. Alzheimer's patients have seen improved cognition and function, and all of these patients have seen the lower levels of DNA methylation, which is essentially changing the biological aging process.
Our second drug candidate is BIV201, which hopefully will become the first therapeutic for ascites, which is a terrible condition that has over 50% mortality rate within 12 months.
As you know, we believe that chronic low-grade inflammation is the starting point for many things that go on in the body. And it all starts with TNF-alpha, which is considered to be the master regulator of inflammation. When you have TNF-alpha present, it just drives more inflammation. It creates a forward-feeding pro-inflammatory cycle that just creates -- that produces more TNF-alpha, more cytokines, chemokines, which of course has been associated with a large number of diseases.
When you have inflammation, TNF-alpha, it derives its resistance that's been associated with Parkinson's and diabetes. And of course, TNF-alpha drive something called DNA methyltransferase 3A and B, which accelerates DNA methylation, which has been associated with a large number of diseases. And our drug candidate directly impacts the production of TNF-alpha. It's been -- and it works through something called ERK and NF-kappa B.
Now it's been known for decades that ERK plays two very different roles in every -- practically every cell in our body. The first involves insulin signaling, whereby insulin works through several pathways and essentially affects all -- and ERK helps affect all the cellular growth repair and regeneration activity that goes in all of ourselves. But it's also been known for all this time that ERK plays an important inflammatory role as well, whereby different extracellular signals, such as different cytokines, chemokines would come into the cell, it would activate ERK, which then activates NF-kappa B to lead the production of TNF-alpha. And as I mentioned earlier, TNF-alpha then drives a forward-feeding pro-inflammatory cycle, that leads to more TNF-alpha, right?
And so it's obvious what you want to do, you want to block what's called inflammatory ERK without touching hemostatic ERK. But unfortunately, that's much easier to than done because dozens of teams over decades try to do it, but no one was able to find a selective enough inhibitor of just inflammatory ERK. And that's where, frankly, we got lucky. We did not set out to do this. We just had our eyes open to recognize that our drug candidate, bezisterim, blocks the activation of ERK and NF-kappa B, right?
So we are not blocking ERK itself, right? And that's why we do not suffer from the toxicity problems that have plagued all the prior ERK inhibitors. What we are doing, bezisterim is blocking the activation of ERK into its active form, something called PERK. And without PERK, you're not going to have activated NF-kappa B and as a result, you block the production of TNF-alpha, which is a huge deal because this is the starting point for many things that comes after it in various disease cascades, one of which, of course, is Parkinson's.
It's been known for quite some time now that 2 conditions need to be true at the same time for a patient to develop Parkinson's symptoms. The first is you need low dopamine levels in the brain. And the second is you need inflammation and the associated insulin resistance. If you're somehow able to just reverse the insulin resistance in the brain, the body is able to make use of the dopamine that's there to restore muscle function. And over the years, people have tried lots of crazy things, including using inhaled insulin as shown here, right, to try to get a little bit more insulin into the brain so that you can reverse the insulin resistance and free up that glucose. And it does work, right?
As you can see from this data, if you're able to get enough insulin into the brain, it reverses the muscle loss, right? The body is able to make use of the dopamine that's there. But that, unfortunately, is not a very practical solution. And that's why to this day, the standard of care in Parkinson's remain the drug called levodopa, which was introduced over 5 decades ago. And now levodopa remains the standard of care for a reason. It is a very, very, very good drug in helping to restore muscle control.
But it does have some deficiencies. The first is that it has a short half-life, which means Parkinson's patients have to take it every 3 to 4 hours or 5 hours because it wears off very quickly. That has a short half-life. The second is that it's activities wanes over time so that the longer you stay on the drug, the higher dose you need to get that effect. So over time, as you get higher and higher doses, it causes something called LID, levodopa-induced dyskinesia, which is a trembling effect.
So when you have LID, all you can really do is cut back on the dose of levodopa and wait for the dyskinesia to pass. But as you cut back on the dosage, you lose muscle control and therefore, are not able to move, which is a terrible trade-off that Parkinson's patients have to go through. And what we found in our preclinical studies in rodents and nonhuman primates is that bezisterim is equally effective as levodopa in restoring muscle control.
Now that is a huge statement in and of itself because no drug over the decades have been able to be equal or better to levodopa in restoring muscle control. But what we also found is that we give it in combination with levodopa, you saw a synergistic effect, whereby you saw the greatest muscle control. Now that is a huge deal.
What we also found at the end of the trial, when we sacrifice the monkeys and look at their brains, we found that those that were treated with bezisterim retained twice as many neurons as those that were treated with placebo, right, suggesting that there is a neuroprotective property to the drug, right? And we're grateful to the Michael J. Fox Foundation for having funded this trial to help us understand that there is a potential neuroprotective property to the drug, which for us is actually not at all surprising because we know that bezisterim reverses insulin resistance, which means there's greater glucose availability in the brain. We also know that system enhances blood flow to the brain, which means there's greater oxygen available in the brain. So when you have greater glucose and oxygen availability in the brain that only bodes well for better neuronal health.
So based upon this preclinical data, we went into the clinic and enrolled 40 patients with moderate to severe Parkinson's, and we essentially replicated in these patients, the study that we did in the nonhuman primates, whereby we treated the patients for 28 days. We gave half of them a placebo and levodopa or essentially just levodopa alone, as shown here in red.
We also gave half the patients bezisterim and levodopa as shown in blue, and as you can see, those that were treated with levodopa experienced about a 3-point improvement or a superiority on what's called the Part III score or the motor control score of the unified Parkinson's disease rating scale. Now that is a clinically meaningful difference, right?
And what more is that, that is on top of the tremendous improvement that levodopa produces by itself. So this is a huge deal. But if you look at patients who are younger than 70 years old, who -- presumably whose disease has not progressed quite as far, that difference is closer to 4 to 5 points, which is a huge deal that hasn't been seen very often. But the other thing that we observed has to what's called the time 0 muscle state.
As I mentioned to you earlier, bezi -- I'm sorry, levodopa has a short half-life. So the studies -- the way the study is conducted, and this happens every single day for hundreds of thousands of Parkinson's patients is that they would take their evening medication and go to bed. And overnight, levodopa would wear off. So that first thing in the morning before they take the evening -- the morning medications, none of the patients that were in our trial that were given placebo and levodopa, essentially levodopa alone, had control of their muscle first thing in the morning. So their muscles were in the off state. So they were stuck and rigid and so forth. And they had to wait -- take their morning medication and wait until it kicks in before they had restoration of muscle control.
In contrast, roughly 1/3 of the patients that were given as bezisterim have control of their muscles first thing in the morning. They can move. They can get out of bed. Now that has just never been seen before. And that, of course, is all because bezisterim has a long half-life. It lasts for 12 hours, whereas levodopa last for maybe 3, 4 to 5 hours at most post. So this trial gives us the first proof of concept in humans that shows that we can give levodopa alongside bezisterim and help moderate to severe Parkinson's patients improve their muscle control. And this establishes the first half of what we're trying to do in Parkinson's.
And we are now conducting a second study to demonstrate the second part of what we're trying to do in Parkinson's, which is to show that bezisterim could be used as first-line therapy as monotherapy in Parkinson's. So we are enrolling 60 patients in a clinical trial where we will give half the patients placebo, and then the other half, we will give them bezisterim alone. And the trial will go for 3 months and what we're hoping to show in this trial is that over the course of 3 months, bezisterim can help slow the progression of the disease, slow the progression of muscle loss for patients who are treated, whereas those that are on placebo will continually have a worsening of their muscle control, right?
This trial, as I say, is underway. This trial is expected to be fully enrolled by the end of the year, and this trial is expected to have data readout in April or May 2026, right? So this is a very exciting development for us. This trial was slow to enroll. The enrollment has picked up significantly in the past few weeks, months, and we are now expecting full enrollment at the end of the year.
So now let me move on to long COVID. The world really has moved on from long COVID. Everybody assumed that it's behind us and so forth. But the reality is thousands of Americans are getting infection every single day. And 17 million Americans currently suffer from the debilitating symptoms of brain fog, fatigue and malaise associated with long COVID, and 3 million Americans have these conditions so severely that they have had to quit or change their jobs, simply because they can no longer put up with the physical demands of their job, and there are no treatments out there today, right?
And it is unbelievable to me. I did not know this, but there are huge, huge long COVID centers and major academic centers around the country that are trying -- such as Yale, Mayo Clinic, Stanford, UCSF and so forth, that are trying to treat these patients using any combination that they could simply because there's really no treatment for long COVID as it stands. And within the last couple of years, researchers have been able to tie the symptoms of long COVID through inflammation, right?
It's believed that long COVID, essentially despite proteins that remain in the body after the infection has been thought fought off, works through something called the TLR4, the toll-like receptor 4, and the spike protein, the E protein, the envelop proteins, also works through the toll-like receptor 2, both of which worked to activate NF-kappa B and ERK in the cell. And since bezisterim has been shown to modulate the activation of ERK and NF-kappa B, there is good reason to believe that bezisterim may be able to modulate the inflammation that comes from these -- the spike protein and the envelope protein that continues to circulate around in the body. So that is the reason why that we have been given a $13 million grant. In fact, we are the only company that has been given a grant to test a therapeutic to see if it can address the CNS symptoms of long COVID. The CNS symptoms, of course, being brain fog, malaise, fatigue that goes along with long COVID.
So we are currently enrolling 200 patients in a trial that is fully supported by our grant. We are randomizing the patients half to placebo and half to bezisterim, okay? And this trial essentially runs for 3 months and then that trial, like I said, is currently underway, and we hope to give full guidance on when the trial will be fully enrolled, but we expect this to be on track for a data readout in the middle of next year, right? Again, a second data readout in 2026.
With that, let me also move on to ascites. Ascites is the origin of the company. This is where the company started before we made the acquisition of the bezisterim that brought us into the CNS realm. Ascites is a terrible, terrible end-stage liver disease. By the time you develop ascites, the liver has gotten so scarred that it's not able to process fluid the way it normally does. And it then also starts to affect the kidney. And as a result, fluid starts to build up in the abdomen, right? And there are no therapies approved for ascites. And so literally, the only thing that you can do is stick a large needle into the abdomen and physically remove the fluid that has accumulated there. But since nothing has been done to address why the fluid accumulates in the first place, these patients are back in the hospitals every 1 to 2 weeks to get another 5 to 10 liters of the ascites fluid removed. Now you can just imagine the strain that that's up to the body, right?
And so frankly, the only treatment objective at this point in time is to keep the patient alive for as long as possible so that he or she hopefully may qualify for a liver transplant. And since there are not enough livers to go around, the life expectancy, the mortality rate, I should say, for this condition is over 50% over the course of 12 months. And our solution is a drug called terlipressin. We put our drug into a small saline bag. We hook it up to a portable infusion pump that patients wear on their belt, and we slowly infuse the drug in over the course of the day. We conducted a Phase IIa trial that showed that the drug was safe. There were no severe adverse events that were drug related.
We then continued on to a Phase IIb trial to try to demonstrate efficacy of the drug. The plan was to enroll 30 patients in a trial, right? But halfway through the study, after 15 patients were enrolled, we had already received statistically significant data that show that those that treated with our drug, BIV201, saw a 50% reduction in the ascites volume, whereas those that are under standard of care saw no change. And as I mentioned, this is a condition that has 50-plus percent mortality rate within 12 months. So that's why we concluded, the only ethical thing to do was to stop the trial early and to engage the FDA in a conversation in what -- on what it would take for us to proceed to Phase III trials for registration.
And since we've already received fast track and orphan designation from the FDA, we only need one trial to register the drug. So as of now, we have had conversations with the FDA. We have received all the feedback we need from the FDA to design the trial, which we have done, and we have submitted the Phase III protocol for the trial to the FDA that was done 3 weeks ago, and the FDA has 30 days to respond to our protocol, and that 30 days is expected to be up next week.
So by the end of next week, we expect to have comments back from the FDA on any changes, if any, and we don't expect any, which would then allow us to proceed to Phase III. This trial is currently underway pending different fundraising mechanisms. We are in various discussions to see how we can raise $25 million to fully fund all of the work that would need to be done between now and registration for this drug, right? And this, for us, is a big deal that would save a lot of lives.
To kind of give you a sense of the magnitude that we're talking about here, there are about 1 million Parkinson's patients in the U.S. today. And if with just 10% penetration of that market, we believe that our bezisterim for Parkinson's could be at least the $3 billion to $5 billion drug annually in annual sales in the United States. And with ascites, we believe this could become a $1.6 billion to $2 billion drug in annual sales in the U.S. alone, right? So any way that you want to risk adjust or discount these numbers, it would still lead you to a number that is substantially larger than our current market cap of $15 million, right?
So that's why I continually tell people that I believe we represent a great investment opportunity. We are the rare biotech companies that has significant cash runway well beyond catalyst. We will have 2 data readouts in middle of next year in second quarter and maybe over the summer of next year. Our Parkinson's trial is expected to read out in April or May. And our ascites trial is certainly going to be reading out in the middle of next year, right? So we represent that company that has cash. We have a company that has multiple data readouts, right? So I believe we represent a terrific investment opportunity for investors who have a 12- to 18-month planning horizon, investment horizon because we believe there is good reason to believe that these trials will be positive, right?
With that, let me stop it here to see if we have any questions in the audience.
Thank you very much, Cuong. [Operator Instructions] As some persons already have done, Cuong. What is the size of the market for your ascites drug? What do you expect the FDA to do by the end of the month?
Right. For ascites, there is no drug available on the market right now. So we have taken a very conservative estimate to size the market, right? And that leads us to believe that our ascites drug, BIV201, could have annual sales of somewhere between $1.6 billion and $2 billion in the United States alone, right?
And there was a question about what do we expect from the FDA by the end of the month right? Frankly, we expect them to not have any comments, which essentially means that we are then authorized to move on to Phase III when we have the funding to do so.
Okay. Well, we have someone who wants to speak to you, Cuong. Lance, please unmute your line and Cuong is ready to speak to you.
2. Question Answer
Yes. I was wondering with the ascites, do you have any information or any follow-up on the patients that were in the Phase II trial, the ones that did so well? Do you have -- is there any follow-up on that information?
We have some anecdotal follow-up. We did not follow up as there was no post-study surveillance, if you will, that was put in place. But we do have anecdotal information that those patients continue to do well. In fact, we have one patient who actually never had to go back to have another centesis because the drug essentially helped address her ascites fluid buildup, which is exactly what we want to see, right? So that was some -- that is very encouraging. And part of the -- that gave us some of the information that we needed to go and design the Phase III trial the way that we did. So hopefully, that answers your question.
It did. I have another one, quick one. with the ascites, you've been looking for a partner ever since that trial, which I don't remember was it a year, 1.5 years ago or along the lines, and you haven't found anything. What makes you think that you'll be able to find somebody now?
That's a very good question. As we look at out there, the most logical partner for us is a company that was going through its own difficulty, right? They were bankrupt. They've been acquired by someone else. And for that company, this represents a small opportunity. That's why I think that just doesn't quite work out for them. There are 2 other companies that we were in discussions with, and they're highly interested in this. But frankly, they, like us, have had difficulty raising capital as well. And as you know, the Baltic capital market has been brutal, absolutely brutal over the course of the last 2 years. So those companies, like us, have been keeping their eyes open for opportunities to fundraise. And we believe that the capital markets are beginning to open up a little bit that gives us some hope that over the course of the coming months, capital will become available for us to either individually -- jointly fund the trial or to essentially fund the trial through a different kind of partnership that we're exploring.
Thank you very much, Lance. Cuong, what is the status of the once daily bezisterim pill?
Yes. That is the -- let me -- to explain that, bezisterim is currently administered twice a day. Patients would take one capsule in the morning, one capsule at night, right? And the -- obviously, when you're trying to address a population like Parkinson's or Alzheimer's, once a day would be much, much better. So we have initiated and frankly, we have slow walked the development effort for a once-a-day drug, simply because we know we can -- we know it can be done, we know what needs to be done, but it will cost us about $1 million to $2 million to fully develop that once-a-day version, right? And so right now, what we want to do is to focus our resources on the Parkinson's trial and the long COVID trial just to make sure both of those readout. And once that is behind us, we'll then pick up on a once-a-day version. And that is probably the version that we will take into the Phase III trial for Alzheimer's and for Parkinson's as well. Thank you for the question.
Cuong, you may be having bandwidth issues and I'm suggesting that you cut your video and go only -- yes. Thank you. Thank you. [Operator Instructions] Cuong, we already have some questions here written in. Why do you only feel 10% of Parkinson's patients would take bezisterim? I would think, given the results so far, every patient would want it.
I share that optimism, right? But I think the safe thing to do is to forecast a modest penetration and then let it grow over time. I would much, much rather under deliver -- under promise and over deliver rather than overpromise and underdeliver. It goes back also to the fact that bezisterim is a -- I'm sorry, levodopa is a very, very good drug. And if you're already on levodopa, you then would need to take bezisterim top of it, right, which at the time that we launched the drug, we would not have the indication for it. We expect to launch bezisterim as a monotherapy to go after newly diagnosed patients, right? So that is a population that needs it the most.
The reason I say that is that the moment you are diagnosed with Parkinson's, you know you are on a one-way road of neurodegeneration and your muscle control will only go down. So the faster that you can get on to a drug that can help slow that decline, the better for the progression of your disease. And so frankly, from a good of humanity standpoint, that's exactly what we want to do is to help those patients slow the progression. So that is our first indication.
Our second indication is we will go back and redo our Phase II, which gives bezisterim in addition to levodopa to essentially get the market as a combination, right? But early on, we want to be safe, conservative, small forecast. Of course, there's going to be some off-label use where clinicians will give bezisterim to patients who are already taking levodopa, we're not going to forecast for that. We're going to wait until we actually conduct the Phase IV trial that gives bezisterim with levodopa, show that it works, get the claims for it, then we will go and market for it. But that, of course, will take us into well beyond the 10% that we're conservatively forecasting now. Like I said, we would rather under promise and over deliver rather than the other way around. I hope that answers your question.
Thanks, Cuong. Have you had any discussions with any major pharmaceutical companies for funding?
Thank you for that question. Over the last couple of years, we have had a number of conversations with large pharmaceutical companies that are interested in Alzheimer's, Parkinson's and CNS diseases. A number of them have actually shown interest, great interest in this mechanism of action because this is a very novel mechanism of action because as you know, in Alzheimer's, everybody has been talking about amyloid and to some extent tau for decades, and there's never been a drug that can essentially address amyloid and tau, which then also significantly alter the disease and slow cognitive decline, right? And we represent the first new mechanism at work in Alzheimer's and Parkinson's in decades.
But in pharma, and I understand where they're coming from, because I've been on that side of the table as well, the road is littered with promising drugs for Alzheimer's and Parkinson's that fail in Phase III, right? And in big pharma, no one is ever fired for playing it safe, right? And so the reception that we have gotten from big pharma has been they wish us well, right? They really are wishing us well. They're monitoring us, and they basically tell us come back when you have positive Phase II or Phase III data on this trial and then we'll pick up the conversations there, right? So there's interest in our molecule, they're monitoring it, and they're just really waiting for Phase III data in Alzheimer's and possibly Phase II, more ideally Phase III data from Parkinson's trial. And so I do not expect us to ever commercializing these molecules. My expectation is that when we demonstrate positive data, we will out-license the molecule to big pharma or frankly, they will acquire our company, right? That's really the realistic outcome to expect in for our company. I hope that answers your question.
How long would a Phase III trial last for ascites?
This trial is unfortunately a trial that's going to take some time to enroll. We need to follow the patients for 6 months, right? So in theory, this trial is short, but the enrollment rate here will be relatively slow, right? And so we would expect to start the trial, to conduct the trial in both Europe and the U.S. with some significant centers that work with these patients, right? But even that, this trial will take us at least 18 months, possibly 24 months from beginning to end, right? And that's part of the reason why we did not prioritize this earlier is that it will take some time and $25 million to fully complete the study and to register the drug. Hopefully, that answers your question.
What is the status of the class action? And is it a large drain on resources?
To be honest, it is not a large drain on resources, right? Where it stands right now is we are in discovery stage. We had essentially -- the way it works is that we put in our petition or motion to dismiss the trial at the very first stage. And frankly, the way -- and that was not granted. And the way it was not granted is, frankly, the judge bought our answers to virtually every argument, but the judge had a couple of other questions that were still unanswered in his mind -- in her mind. And the -- and as a result, she asked us to go into discovery and essentially answer those questions. So we are now in the discovery mode, whereby both the plaintiffs and us are essentially look -- answering questions, putting up answers to various questions that were being asked. And once the discovery stage is over, we will then essentially put in place another set of questions for summary judgment.
And as you should know that, unfortunately, many of these class action lawsuits is an insurance game, right? When any time that -- there's significant movement in share prices, there is an entire group of ambulance-chasing lawyers out there that essentially goes out and try to find 1 or 2 plaintiffs or shareholders who've lost some money to essentially try to get a class action suit started, right? Their objective is never to actually sue us or essentially win. Their entire objective is to get an insurance settlement, right, and get essentially payout by the insurance company. And as a result of that, over 2/3 of these suits are dismissed at the motion to dismiss stage or dismissed summarily without ever going to trial, right? And then a small minority of these -- and then the remainder essentially are negotiated settlements, right?
And the way it works is that we're now in the discovery phase, as I mentioned, we will eventually file our motion to dismiss. We believe that will be granted. And you should also note that our attorneys that are working on this has an 80% dismissal rate of these suits compared to the industry average of about 2/3. And even if the suit is not dismissed, we believe we'll settle it and we are still well within our insurance policy for the settlement of this suit. So frankly, it's not taking that much time. It's really the lawyers going to the motions of it right now. Hopefully, that answers your question.
Could you clarify ascites please, Phase III trial begins mid-2026, and lasts 18 to 24 months?
That is our hope is that if we get the funding for the trial such that we are able to initiate it mid -- whenever we initiate it, the trial, let's say, will be expected to run for about 2 years. That is how the Phase III trial will work for ascites.
We have time for a few more questions. [Operator Instructions] Cuong, INmune Bio was another company focusing on TNF-a and its recent clinical results were not as good as hoped. Did that trial provide any useful information about the method of action of bezisterim?
Thank you for asking that question. And INmune Bio, of course, is one of a small handful of companies that work on inflammation as the mechanism to address disease. And that trial rebolstered our conviction that bezisterim is the correct approach, okay? The INmune Bio's approach is not to block the production of TNF-alpha as bezisterim does, but to actually hope to try to mop up and block the activity of TNF-alpha after it has already been produced, right, which is downstream. And once you have all the TNF-alpha in the body, mopping it all up is very, very difficult, right?
And you know that is -- excuse me, the case from the entire class of antibodies now known as anti-TNF, right, such as Humira, Stelara and so forth. These anti-TNF antibodies have been around for decades. That's why Humira still remains a multibillion-dollar drug that it is right now. And the experience with these antibodies, these anti-TNF drugs, shows that it's actually very difficult to mop up and block the activity of TNF-alpha once it's already there. And unfortunately, INmune Bio is doing exactly what those antibodies are trying to do in a different and a small molecule. It just doesn't work quite as well, although we know there is a signal, and we know that blocking TNF-alpha can work.
And what I -- the reason I say that is there have been large population of studies out of the NHS and here in the U.S., Mayo Clinic and other places, that looked at patients who are taking anti-TNF medication. And what they found is that for those that are taking anti-TNF medications, those patients have a 50% lower risk of developing Alzheimer's compared to those who are not taking anti-TNF medications, right? And of course, the Humira and so forth, do not cross into the blood brain barrier, right?
So it doesn't really help directly with getting into the brain. But the mere fact that you're reducing the TNF-alpha burden systemically for these patients also help reduce some of the CNS burden of TNF-alpha, right? So it just shows that if you're able to reduce TNF-alpha, you reduce the potential risk of developing Alzheimer's. And that is part of the reason why we have even greater conviction now that bezisterim is the right way to go. And by blocking the production of TNF-alpha, we believe we block all the downstream negative consequences that comes along with that, which then should help us not only with Alzheimer's, but with Parkinson's, the long COVID as well.
Thank you very much, Cuong, and thank you, everyone, for all of your questions. For more information about BioVie, reach us at 1-800-REDCHIP or e-mail us at [email protected]. Please visit RedChip's Investor Information page for BioVie. It's biviinfo.com. There, you can view and download the investor presentation and fact sheet and sign up for news alerts on BioVie.
Please be sure to watch Small Stocks, Big Money, RedChip's program featuring exciting small cap companies every Saturday night at 7:00 p.m. Eastern on Bloomberg USA.
Join RedChip's next webinar with FatPipe on Wednesday, October 15 at 4:15 p.m. U.S. Eastern. Register for all RedChip webinars at redchip.com/events, where you can also view an archived version of today's webinar. Thank you to our many participants today, and thank you, Cuong.
Thank you. Have a great day.
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| Hauptsitz | USA |
| CEO | Mr. Do |
| Mitarbeiter | 13 |
| Gegründet | 2013 |
| Webseite | www.bioviepharma.com |


