KemPharm, Inc. Aktie

It's been a long day for you. We'll get through this efficiently, but also be able to have an opportunity to explain your story to the folks here today and those listening in.

So maybe before we get started, Neil, your mission is to redefine what's possible for people with rare diseases. What does that mean to you and your team?

Yes, that, first of all, thank you for having us. We've had a great day today. We really appreciate the support of Moving Family. I will be making forward-looking statements, so I'd ask for everybody to take a look at recent SEC filings to ensure that you get the most up-to-date information. On behalf of Justin and the rest of the team, I really appreciate answering this question because what we do in the rare disease world is clearly a challenging part of pharmaceutical development and bringing medicines to people who otherwise have no other opportunity to medicines. Now, rare disease is not as unique as it once was 20 or 30 years ago, but what we do hasn't changed much. We're trying to develop medicines and redefine how we get them to patients to then impact their lives. For us, we're doing that in a commercial sense today, primarily with commercial programs in Niemann-Pick disease type C, ultra-orphan, along with UCD. But in addition to that, we're also expanding globally to patients outside of the U.S. And we have a Phase 3 program also going in vascular Ehlers-Danlos syndrome. So for us, it's ultra-orphan diseases. We're talking about prevalence rates of less than 1,000 for some of them and less than 7,500 for others, but I'm looking forward to getting into that more with you as we move forward. But it's a real privilege for us to be able to be working in the rare disease space and thinking differently about how we impact the lives of patients living with rare diseases.

Great. Thank you for that. How would you describe Zevra to an investor hearing the story for the first time?

Yeah, we've taken a little bit of a different tact. We are a commercial stage organization that has been a commercial stage organization for just a few years. Today we have a U.S. opportunity with Miplyffa for Niemann-Pick disease type C, about 900 patient prevalent population in the U.S., about 300 to 350 of those patients. This is a commercial opportunity for us. We've been now at it for about six quarters in terms of the launch. We'll get into some more of this detail later on. But in addition to that, that's 1 leg on the stool. The other leg on the stool for us is our global expansion. We're trying to be able to get Miplyffa (arimoclomol, I should say) into Europe through an MAA process that we'll talk about a little bit more as well. And we're globally expanding into regions and territories where we can leverage the European, sorry, our U.S. label into other markets. And then we have our third leg on the stool, which is our pipeline, our Phase 3 development program for vascular Ehlers-Danlos syndrome, which has enrolled about 66 patients out of 150 today. And we'll talk a little bit more about that later on. But that is in a place for us today that we can really go to the agency and start to have some conversations around how do we accelerate the development. Zevra today is 3 legs on a stool. It's a U.S. opportunity that has a lot of uncertainty. Untapped potential. It's a global expansion opportunity in our second leg on the stool, and thirdly, it's around celiprolol in the pipeline.

Great. So maybe, um, we'll start with Niemann-Pick Type C. Now, you are the first and only FDA-approved treatment. And you've been, you know, you said in the market for about six quarters. Where are you in that journey today? And frankly, what are you most excited about from?

to go from here? Yes, most excited about the learnings we're having. As I mentioned, 900 patient prevalence in the U.S. About 300 to 350 of those patients were diagnosed. And now the opportunity for us, six quarters in, at the end of Q2, we had 184 prescription enrollment forms so far. That is an amazing impact that our team has been able to drive so far. With only 350 patients, getting 184 of those in six quarters is pretty good. Now, that's primarily those patients. We also are even more excited today than we were 1 year ago because we've been learning. We've been learning, number one, that it's not only a pediatric disease anymore. We've, about 50% of our patients are adult patients. That learning is now informing how we try to get newly diagnosed patients into the mix. I'll give you an example. We have had patients that have been misdiagnosed with multiple sclerosis, adult patients that then have progressed with other symptoms that are more attuned to Niemann-Pick disease type C, get them genetic tested, and then they've eventually gotten on the product based on the fact that they had NPC, not MS. We've had a Wilson's disease patient that has been, you know, recently published, actually about a quarter or two ago, that was misdiagnosed. So with this, we're learning. And in any rare disease, the more you learn about the journey of patients that are getting them to the ultimate and then on treatment, is it better off that we can then tailor our development program, sorry, our patient finding programs to being able to then get more newly diagnosed patients. If I look back at the last six quarters, 1 year ago today I would not have told you that 50% of our patients would be adult patients. You know, they used to coin Niemann-Pick disease type C as childhood Alzheimer's. And today we're understanding more, we're learning more, and now those newly diagnosed patients are giving us a lot more confidence that the market is somewhere between that 350 currently diagnosed patients and the newly diagnosed patients, that quarter over quarter we're seeing more of those now that we're learning. So that's been probably the biggest surprise for us that there is real growth, very similar, real opportunity for growth beyond the 350, and that's very similar to Europe. In Europe today, there are about 1,100 patient prevalence, and the majority of those patients have been diagnosed on some type of therapy. And the reason for that is, Miglustat was originally approved well over a decade ago. The previous company went out and invested in disease state awareness and diagnostic modalities in publishing the suspicion index, and that that market has been more mature over the last decade of identifying and diagnosing patients closer to the 1,100 patient population in Europe. So we have a lot of confidence that if we can continue to invest in disease state awareness, continue to get diagnosis earlier, that we too will be able to continue to build newly diagnosed patients to get closer to 900 and 350.

Yeah, and I think, as you said, as you're kind of uncovering new adult patients, the way people think about the overall number of patients that are out there, it's probably only going to grow and you'll hopefully find more patients that really need your therapy. So, maybe shifting a little bit in terms of your launch. You're now generating about $30 million a quarter, only just, you know, less than 2 years into launch. What is driving that momentum and how's your patient finding approach reaching newly diagnosed patients, including the roughly half who turn out to be adults, as you said. And maybe I'll just add for the audience, you know, even recently a colleague of mine reached out to me to be referred to you all as their son is having, suffering from NPC. So I think it really speaks volumes in terms of kind of your ability to continue to.

execute and find new patients. But yeah, just walk me through kind of the momentum that you've had with your launch process. Yeah, it thank you for talking about that personal journey. This is what makes it really special what we do. The opportunity to impact the lives of people suffering from NPC, but it's also important to have something that we can offer them. And that's been 1 of the drivers of success. We have recently been able to get published our open label extension study in addition to our clinical trial data that showed about the halting of the progression of the disease through 12 months, based on the Niemann-Pick C severity scale with our data in combination with Miglustat. Now we've been able to also get the published data from our open-label extension study going out 4 years that supports that, that, uh, that, that, uh, trajectory, along with our expanded access program now that's also out 4 years with some pediatric data, that, uh, part of our pediatric investigational plan that's out there too in the literature. We package all of that together with, now that we've got newly approved therapies, the clinicians have now published consensus guidelines. And they've updated that based on the fact that there's now a product that's approved and multiple products that have come to market here recently. So these clinical guidelines have been able to reinforce the need to treat early, to diagnose with genetic testing so you hopefully don't have misdiagnosis, and then to treat with combination therapies, which is what we have in our label, and disease modification. So all of this longitudinal data helps to be able to get patients on the therapy and then have that therapy paid for by payers because you're able to provide convincing evidence of efficacy. On top of that, you know, we're able to now work with physicians who may have never, if you've never seen NPC before or understand what it looks like, we can get them engaged with other experts that are in there, but we can also provide reference to clinical guidelines that show disease-modifying therapy, use combination therapy, and Miplyffa in combination with Miglustat has a potential benefit for those patients. It's a great safety profile, I should add.

I think you're really changing the kind of your position as a potential foundational therapy. But maybe just on that note, as you said, NPC historically had no approved options. Now, as you mentioned, starting to see some new entrants. Um, now, and as the first FDA approved therapy and with treatment guidelines starting to be updated to support combination use, how do you fit into that evolving landscape? What kind of work that you're doing? How are you, you know, what is your kind of approach to physician education?

Now, we want to continue to learn. And every day that we have the opportunity to identify the journey of a new patient, because 1 thing is for real, and I have been in this industry for a while in the ultra rare space in multiple different companies, you know 1 patient, you know 1 patient. Every patient's journey to getting to the ultimate diagnosis of a rare disease and then a treatment, if there is 1 available, is unique. And for us, you know, having the mentality that we can continue to learn from what we're seeing in the marketplace for every patient and try to understand from the physicians, even the payers by the way, and the rest of the community how best to be able to take that learning, move their diagnosis journey earlier to then allow them to be genetic tested earlier and then get them on therapy earlier, that allows for us today, based on our therapy, to be able to halt the progression of the disease and have a better outcome for patients. So, if I think about the other products that are being developed and the rising tide that is kind of lifting all boats around disease state awareness, expert connect programs that we have to get people who are not, you know, aware of what NPC is and get them connected with somebody who's an expert. And that expert, by the way, might only have 2 patients. You know, it's not like a lot of physicians have ever heard or seen of NPC. So that rising tide, getting these case reports out, having the ability to tell the story about each and every journey, each and every patient's journey, then get somebody else to think NPC. And that's what we're trying to do.

Got it. Thank you. And so on that note, you mentioned it is a progressive disease where staying on therapy matters enormously. Now, once patients start Miplyffa, what are your strategies for keeping them on therapy long term?

Yeah, we have a fairly robust patient services group that helps through our specialty pharmacy and through our own efforts, it really helps to be able to decrease the burden of getting their shipments, decrease, you know, we call it white glove, you know, service, but the reality is that it's almost the cost of doing business for ultra rare disease patients. They have a challenging time getting multiple therapies for their disease, for Niemann-Pick disease type C specifically, but for all rare diseases. It's not like it's just you're just on 1 product. They're all specialty products. So we have a patient's services group that really tries to handhold and support through reimbursement support, through monthly shipments, through contact directly with the families to be able to get the shipment to their homes at the time that they're going to be there, that they can receive the shipment and actually sign for the shipment and get it there. Because this happens every month. And we're learning there too, by the way. There's certain things that we did really well that we're doubling down on, certain things we weren't doing so well, so the surveys that we get back from patients and families, we can then actually improve the quality of the service that we provide to those patients and caregivers.

Got it, thank you.

There's 1 additional thing that I think is important. We have, uh, we don't publicly talk about the persistence rates or the compliance rates, but I think, uh, you know, we're asked that question on a regular basis in in these meetings. And, uh, and whilst when you're talking about 1 number, you know, difference of a patient that's, uh, persistent, uh, you know, month over month versus 1 that might discontinue. It can skew your numbers, because it's hard to be able to provide that. But what I can say is that we saw very high levels of persistency in our expanded access program. And we saw very low levels of discontinuation rates. And primarily, these are patients who their disease progressed and unfortunately passed away from their disease. We're seeing very similar types of high persistency, high refill rates, and low discontinuation rates that we saw then and as we continue our launch and this trajectory that we're on, this high level of trajectory that we're on in identifying new patients, once we get to some steady state, which I don't foresee any time soon, then we'll be able to potentially provide more persistence and or adherence rates.

Got it. Now, on Europe, we talked about, you know, kind of the significant identified patient population base there and, you know, the work that you're doing through obtaining approval with EMA. How are you thinking about the pathway? board and the opportunity to bring the product to European patients. Now, I will say I believe what you have is 1 of the largest NPC data sets that's ever been assembled. So just maybe you can fill me in on kind of your EU application process.

Yes, a couple of things that I'll try to mention, and obviously we're in the process right now, so I can't get into a lot of detail. What I will say is that we remain highly confident in the robust nature of the data in our dossier. This has been supplemented with the additional that I mentioned earlier throughout our process with open-label extension data out 4 years, expanded access program data out 4 years, the pediatric investigational plan was complete and submitted as well. So we feel highly confident about that. Today we're in the re-examination process, which we've filed for re-examination as well as an ad hoc meeting similar to a scientific advisory group meeting that will address the remaining issues under the current, um, regulatory framework that exists in Europe. So we are, we are, um, bringing that data to life through our reassessment process, or re-examination process, and they'll have experts there to be able to help to, yes, get to the bottom, if you will, of the questions. In regards to getting the product to European patients, you know, we have a global EAP program ongoing today. At the end of Q2, we had about 132 patients that were enrolled. Primarily, those are in Europe in a very small number of markets. This has grown tremendously over the last couple of years from where we started with just not a few, but maybe half that number of patients. And that continues to show us that there's a real pull for the product post FDA approval in the U.S. for Europe, as well as other markets in the globe too that we've been addressing through name patient basis reimbursement utilizing the U.S. label. So that pull is giving us a lot of confidence that there remains, you know, unmet need. Um, going forward, how we go to market in Europe or how we, you know, assess what our strategy is, it really has got to be taken care of after the FDA, sorry, the EMA process is done. We'll understand what a label could look like in the event of a positive outcome, and then we can address what that market approach will be in terms of go-to-market.

Great. Now, shifting gears beyond NPC, maybe we can talk a little bit around vascular Ehlers-Danlos syndrome, which is really a serious condition with no approved therapy in the U.S. Now, can you elaborate a little bit around where the program currently stands and what excites you about this program?

Yes, so this is another 1 of those ultra rare diseases. About 7,500 patients from a prevalence perspective in the U.S. Celiprolol is currently under investigation in our Phase 3 program for that disease. It is a 150, 2-to-1 randomized trial that that we have been enrolling in. We have about 66 patients in that trial as of the end of Q2. And I think importantly, and we've said this publicly many times, this is a therapy that exists for patients outside of the U.S. today. It's never been approved anywhere for vascular Ehlers-Danlos syndrome. It's been approved for other indications, but it's utilized primarily as a standard of care in a lot of ex-U.S. markets. We're running this trial under a SPA. with a decentralized process of enrolling this trial. But we also recognize that the event rate has been slower than we had wanted to see it. That has led us to being able to engage with the FDA earlier this year to let them know that we are running the trial, the enrollments are coming, the events are not coming as fast as we'd like, and we'd like to be able to work with you to understand what are some options for us to accelerate getting this product to U.S. patients. left us with some homework, very informative meeting that we had earlier this year, and we've said publicly that we have a second half of this year time to re-engage with the FDA after some of the homework that we've done, and we're on track to be able to do that. But our goal here is to be able to try and find ways to accelerate getting the product to patients in the United States.

Thank you. Maybe talking a little bit around your corporate strategy, and maybe 1 area to start is your unique financial position. You're very well capitalized. You, I believe, ended the quarter with $230 million of cash. You haven't been burning that much capital, if at all. Which is very, frankly, unusual for a company in your stage. So how does that position you and how are you thinking about your capital allocation going forward?

Justin? Yes, thank you. Thank you for having us again. We are in very good shape, and Neil and the Zevra team have really positioned us well over the last couple of years with the success that we've had, particularly around the Miplyffa launch. We finished the second quarter $260 million in cash. And we are very focused on our spend. We're very disciplined in all we do. But as we started our conversation today, our three priorities are very crystal clear. Growth of Miplyffa for the U.S., rest of world expansion, and moving celiprolol forward. And we really take that to heart. However, we're always looking to see what else we can do. And so as an example, earlier this year, the team monetized a future royalty stream and some non-core assets to generate some money that we used to repay double-digit interest rate debt. And so now we're debt-free, which is fantastic, and that balance sheet for the future is going to be flexibility that it's now afforded to us gives us a chance to do what we need to do to stick to those three core messages I mentioned, as well as take a look to see what else we can do to grow our company.

Got it. Um, I think, you know, what you have been able to do is, you know, run your company in a very, uh, with capital light milestones to come. But yeah, I think maybe thinking about, um, you know, how investors, you know, many of the equity investors sitting here today, how should they think about what are the right value inflecting milestones? What will be important for them as they are kind of trying to build their own investor investment thesis?

Yes. Yes, you know, we have three legs on the stool, the most today. The focus point for us is around this TAM and the expansion of the TAM for the U.S. business. 1 year ago today, I could not have sat here with the conviction that I have today around what this addressable market is. Today I can tell you it's somewhere between that 350 and the 900. 1 year ago I could not have said that to you outside of the fact that I knew where those diagnosed patients are and a lot of that is because we're now identifying these newly diagnosed patients. I would have expected this three or four years from now. I wouldn't have expected it today. That is telling me that the U.S. opportunity is worth looking at, and if you do the math, I think it's also 1 worth investing in. The opportunity for the second and the third leg on the stool around our potential to impact the lives of patients in Europe with an approval, and also the number of patients we're impacting through our global expanded access program today, shows us that there's a real pull for the product ex-U.S. To date, we've been able to do that to expand markets outside of Europe at U.S. WAC pricing through this named reimbursement program. We're going to continue to expand those markets and open access to patients through that mechanism. But then also from the celiprolol perspective, that's another upside for us. Investment thesis for this company today is the three legs on the stool with really the focal area around the U.S. business today and expanding that TAM. That is... We've got a lot of conviction around that.

Great. Um, you've said in the past that, uh, you want to be a preferred partner for rare disease assets that fit your commercial infrastructure and your vision. Um, what are you looking for if you can comment on it and also, how do you think about continuing to grow the portfolio from here?

Yeah, the vision for us is to become a leading rare disease company. It starts by exactly what we started this conversation with, around how can we make a difference in the lives of the patients with the three focus. The three legs on the stool or the three areas that we're focused in today. If we do that really well, we'll have earned the right to be able to go out and be a partner of choice for other parties. But that really comes off of the back of executing on our mission today. Anything that we could do in the future, we'll have to be able to leverage what we've built and what we're good at. Today we're earning that right by executing 184 prescription enrollment forms in six quarters post-launch. I mean, it's more than 50% of the diagnosed patient population. That's a big deal in the second full year of launch to be able to do that. So we're kind of earning our stripes around becoming a commercial rare disease, high value therapeutic that can pull through patients through, keep them on product, and really help them through this process. I think anything we look at in the future, we'll have to leverage that infrastructure that we've built.

Great. Um, two last questions that I have. One is we've touched on some of this, but back to the point about how investors should think, care about, and maybe starting to do some work on Zevra in the story. What are the key milestones or events that they should be watching out for? I think we've touched on all of those, but just to recapitulate.

Yeah, Justin mentioned the fact that we have resources to be able to invest in the Three Legs on the Stool. I think the important events for investors to look at is number one, our continued growth in the U.S. business. Quarter, you know, quarter over quarter, it's going to be lumpy. It's rare disease. We're not going to be able to have linear growth in anything we do. But I think on an annual over annual basis, which is the lens of which we look through in terms of building new newly diagnosed patients, addressing the patients that are diagnosed and getting them, you know, in physicians educated with the awareness around Miplyffa. I think that's going to be key. That's number one. Number two is we have in Q4 this year, we'll get clarity around the re-examination process when it comes through to Europe. Along those same lines, we call that our geographic expansion strategy, you know, we're hopeful to be able to also bring on board new markets that that will be able to support through our named patient program. And then lastly, we have a second half of this year celiprolol engagement with the U.S. FDA. We're looking forward to that, to understand how we can then accelerate the clinical development of the program to patients in the U.S.

Got it.

Did I miss anything? We're going to continue to take a disciplined approach to how we can supplement our products. It's not core, but, you know, we, as Neil said, we want to earn that right to be a partner of choice. We've built a great infrastructure. We have a strong balance sheet. And so, you know, again, look for those catalysts in the fourth quarter that we just described. And we'll.

try to move from there. Great. And last question. If investors remember just 1 thing about Zevra from today, or anything that we haven't talked about, what would that be?

It's our ability to redefine what's possible in taking care of patients living with rare diseases. What we do in this industry, not just us, but others that are in the rare disease world, is not easy. I mean, actually, we have more failures than successes in what we do in this business, and our ability to try and redefine how we take care of patients living with rare diseases. That's what motivates us every day. And I think that, uh, our entire team, runs through walls for patients on a daily basis. So that's who we are, and that's what we want to be able to continue to drive.

Great. Well, um, thank you very much for your time today, and congratulations on all of your progress and success, and we're very excited about what's to come for Zevra.

Thank you. Thank you.

This live transcript is auto-generated without human intervention or review.

Good afternoon, and thank you for joining Zevra's Second Quarter 2026 Financial Results and Corporate Update Conference Call.

Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our second quarter 2026 financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investors section of our website.

Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with Zevra's business.

Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and our other filings with the SEC.

This call will also reference certain non-GAAP financial measures, including adjusted net income and adjusted net income per share. Quantitative reconciliations of these measures to the most directly comparable GAAP measures as well as information regarding the usefulness of these measures to management and investors are included in the earnings press release issued today, which is available on our website.

I am pleased to welcome Zevra's management team members participating in today's call, Neil McFarlane, Zevra's President and Chief Executive Officer; Josh Schafer, our Chief Commercial Officer; and Justin Renz, our Chief Financial Officer.

Now it's my pleasure to hand the call over to Neil.

Thank you, Nichol, and welcome to everybody joining our quarterly call this afternoon. At Zevra, our mission is to redefine what is possible in bringing life-changing therapies to people living with rare disease. Our strategy to unlock long-term value through focused execution has translated into measurable results across our business.

For the second quarter, our net revenue was $39.7 million, a 53% increase year-over-year. This was driven by $30.2 million in net U.S. revenue for MIPLYFFA. Our commercial progress reflects continued momentum in reaching patients and successfully establishing MIPLYFFA in combination with miglustat as a foundational treatment for Niemann-Pick disease type C, or NPC, in the U.S.

As of June 30, we had received a total of 184 prescription enrollment forms, 14 of which were received in the second quarter, bringing the total in the first 6 months of the year to 23.

Additionally, we have strengthened our intellectual property position for MIPLYFFA with an Orange Book listing through 2041. We published long-term data from our U.S. Early Access Program and the findings from our pediatric substudy, further demonstrating the efficacy and safety of MIPLYFFA.

We requested a reexamination of our arimoclomol Marketing Authorization Application, or MAA, by the Committee for Medicinal Products for Human Use, or CHMP, in the European Union. And we continue to build a strong financial foundation with more than $260 million in cash, cash equivalents and investments.

Our strategic focus is reflected in the thoughtful portfolio decisions we are making, while maintaining an unwavering sense of urgency on behalf of patients.

Most recently, we announced that following our oral explanation, the CHMP issued a negative opinion regarding the arimoclomol MAA. Subsequently, we are advancing the reexamination process, which will provide the opportunity to address the specific grounds cited in the CHMP opinion and have our comprehensive evidence package reviewed by a new rapporteur and co-rapporteur. This process typically takes 120 days from the submission of a reexamination to the opinion from the CHMP, which is expected in Q4.

Importantly, as we advance through the European regulatory process, our commitment to the patient and the NPC community remains steadfast. We will maintain access to arimoclomol for eligible patients through our global Expanded Access Program, or EAP, which is comprised of compassionate use and reimbursed named patient programs that have enrolled 132 patients, with 10 added in the second quarter.

As previously guided, we expect reimbursements beyond the French EAP to be highly variable in the first few years until the patient base has stabilized. Ordering patterns for patients outside of Europe remain unpredictable with some territories ordering a full year supply and others ordering on a monthly or quarterly basis.

Simultaneously, we are evaluating opportunities to expand access to MIPLYFFA through the global EAP in response to strong interest from additional countries. This reflects our commitment to helping eligible patients outside the U.S. access treatment while building meaningful partnerships across the rare disease community.

Additionally, we strengthened our U.S. intellectual property position for MIPLYFFA with an Orange Book listing that provides protection through 2041. We are still awaiting the determination for the separately requested patent term extension through the U.S. Patent Office, which if granted, would fall within the Orange Book coverage period. Together, our IP portfolio provides a strong foundation for the long-term commercial opportunity for MIPLYFFA. And we continue to invest in expanding and strengthening our protection beyond our current portfolio.

At the same time, we are advancing celiprolol, a selective adrenoceptor modulator that induces vascular dilation and smooth muscle relaxation for the treatment of Vascular Ehlers-Danlos Syndrome, or VEDS. VEDS is a rare genetic connective tissue disorder caused by changes in the COL3A1 gene, leading to fragile walls in blood vessels and hollow organs, which can cause arterial rupture or dissection among other complications. Celiprolol has been shown to reduce the mechanical stress on these tissues.

In our ongoing Phase III DISCOVER trial, we've enrolled a total of 66 patients, including four who were enrolled in the second quarter. As an event-driven study, 28 events are required to trigger the interim analysis, and we have three confirmed events thus far.

We are focused on accelerating this program by implementing strategies to drive enrollment, including expanding our network of genetic testing centers and exploring options to advance our clinical development by engaging with the FDA. Following a type C meeting in Q1, we remain on track to reengage with the FDA in the second half of this year.

In summary, our priorities remain clear: delivering innovation for people living with rare disease; creating sustainable value for our stakeholders; and fulfilling our commitment to the patients, healthcare providers and communities we serve.

I'll now turn the call over to Josh to share more details regarding MIPLYFFA's commercial performance. Josh?

Thank you, Neil, and good afternoon. NPC is an ultra-rare genetic disease that causes significant neurological impairment and places a substantial burden on patients and families. Clinical manifestations can include dysfunction of ambulation, fine motor skills, swallowing and speech. The disease and its symptoms vary significantly across patients, reflecting its complexity and severity.

Earlier this year, the NPC clinical practice treatment guidelines were updated to further characterize the disease and reflect new advances in treatment since its last publication in 2018, including the addition of MIPLYFFA. Importantly, the guidelines recommend considering combination therapy for all patients with confirmed NPC.

As a reminder, MIPLYFFA is differentiated as the only FDA-approved treatment for NPC with established efficacy and safety indicated for use in combination with miglustat. The randomized controlled pivotal study demonstrated the combination halted NPC disease progression at 12 months, as assessed by the rescored 4-domain NPC Clinical Severity Scale, a validated measurement of NPC progression. The onset of benefit was rapid with clinical effects sustained over the long-term.

MIPLYFFA's clinical data, combined with a growing awareness across the NPC community, continues to support its adoption. As Neil mentioned, we have received 184 prescription enrollment forms since launch through June 30. The 14 we received in the second quarter included patients from Puerto Rico, reflecting our reach into U.S. territories.

As we have noted previously, an enrollment form is a prescription submitted to our specialty pharmacy, which then begins the benefits investigation process to determine reimbursement eligibility. Notably, patients initiating treatment are being enrolled from centers of excellence and community practices with representation across both newly diagnosed and previously diagnosed patients as well as adult and pediatric populations. This distribution supports our confidence in the estimated prevalence of 900 patients living with NPC in the U.S., of whom 300 to 350 are diagnosed.

Our commercial strategy is centered on three key priorities: accelerating diagnosis and treatment; driving demand; and facilitating access to MIPLYFFA. And the progress we are seeing reflects the growing impact of initiatives being implemented across each of these areas.

Our disease awareness campaign, Learn NPC, Read Between the Signs, is driving the identification of patients based on symptoms and has led to new patient enrollments for MIPLYFFA. Our genetic testing collaborations and our custom AI-driven targeting model are helping to support the identification and diagnosis of patients with NPC, allowing for earlier intervention.

We also launched our Expert Connect program, which has enabled health care professionals who are less familiar with NPC to consult one-on-one with clinical experts to further strengthen knowledge and confidence. Additionally, we expanded and enhanced our digital engagement through the launch of NPC Knowledge Hub, providing resources to help patients and caregivers navigate the treatment journey.

From a market access standpoint, payer coverage remained at 69% of covered lives, consistent with our expectations. We continue to share presentations with payers on the recent update to the NPC clinical practice guidelines as well as the growing body of newly published clinical data on MIPLYFFA. Additionally, we convene payer advisory boards with key decision-makers to inform our strategy on how best to support prescribers and patients as they navigate the reimbursement landscape.

We maintained strong relationships with the rare disease community and assembled steering committees to gather expert insights and helped shape NPC disease awareness. In the second quarter, we participated in national and regional congresses, including the American Academy of Neurology, the Society of Inborn Metabolic Disorders and Annual Pediatric Neuro Symposium, to enhance scientific visibility and educate clinicians and researchers. This strong engagement has continued into the third quarter with our participation at the National Niemann-Pick Disease Foundation Conference, where the importance and interest in a combination strategy for NPC treatment was highlighted prominently.

Before turning the call over to Justin to review the financials, we want to reiterate our deep gratitude for the opportunity to make a meaningful impact in the lives of patients and their loved ones. We remain committed to being a trusted partner in the community by providing compassionate, patient-centered support. Justin?

Thank you, Josh. Underpinning our achievements is a robust cash position, which provides the flexibility to invest in commercial execution, advance our pipeline and pursue future growth opportunities while maintaining financial discipline.

In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file later today.

In the second quarter of 2026, we generated total net revenue of $39.7 million, which was an increase of $13.8 million or 53% compared to $25.9 million in Q2 2025. As a reminder, we recognize U.S. commercial revenue when MIPLYFFA shipments are received by the specialty pharmacy. Of note, channel inventory has returned to target levels as compared to Q1 when it fell below the low end of our range.

The second quarter 2026 total net revenue is comprised of $30.2 million from MIPLYFFA net sales in the U.S., $0.2 million from OLPRUVA, $9.0 million in net reimbursements from the global EAP for arimoclomol and $0.3 million in past due royalty revenue from Commave.

Revenue from MIPLYFFA in the U.S. grew 23% compared to the first quarter of this year and 40% compared to the second quarter of last year. During the second quarter of 2026, our operating expenses were $21.0 million, which was a decrease of $3.2 million compared to the same quarter a year ago.

R&D expense was $4.5 million for Q2 2026, which was an increase of $1.1 million compared to Q2 2025 due primarily to ongoing arimoclomol efforts. SG&A expense was $16.6 million for Q2 2026, which was a decrease of $4.2 million compared to Q2 2025, primarily due to a decrease in professional fees and third-party spending, partially offset by an increase in personnel-related costs. We allocated $4.0 million for our Q2 2026 income tax provision.

Net income for the second quarter of 2026 was $8.8 million or $0.14 per basic and diluted share compared to net income of $74.7 million or $1.24 per basic and $1.21 per diluted share for the same quarter of a year ago.

This quarter, we incurred a mark-to-market fair value noncash charge of $6.4 million related to our outstanding common stock warrants. Excluding this adjustment that impacts period comparison, net income for Q2 2026 would have been $15.2 million or $0.25 per share. In Q2 2025, we would have had an adjusted net loss of $2.4 million or $0.04 per share by excluding onetime transactions and other adjustments that contributed a net positive of $77.1 million to Q2 2025's results.

As of June 30, 2026, total cash, cash equivalents and investments were $260.2 million, which was an increase of approximately $23.4 million compared to March 31, 2026.

In summary, we remain well positioned with the financial capacity to execute on our strategic priorities independent of the capital markets.

Now I'll turn the call back to Neil for his closing remarks. Neil?

Thanks, Justin. Our strong second quarter performance demonstrates the strength of our strategy, disciplined execution and continued momentum across the business. We remain steadfast in our commitment to putting patients first, a principle that is reflected in our approach to growing our U.S. commercial business, expanding our global presence and fueling our portfolio to deliver long-term shareholder value.

With that, operator, please open the line for questions.

[Operator Instructions] We will take our first question from Kristen Kluska with Cantor Fitzgerald.

I had a couple this afternoon. First, you're sitting on a very robust balance sheet and have a continued line of sight into more revenues as the MIPLYFFA business grows. So curious how you're leveraging this cash and ways you're looking to potentially explore using that?

Second, I wanted to talk about the EMA. You mentioned there's a new rapporteur here. Can you tell us a little bit about how that process works when there's really a new team here looking? Is there anything that you can do differently? And we have seen some recent reversals specifically in the rare disease space. So curious about your thoughts there.

Thank you, Kristen. Maybe I'll start with your second question, and then I'll pivot over to Justin to talk a little bit about the strong financial foundation we've got.

So you are correct that in our reexamination process we will have the opportunity to bring in new rapporteur and co-rapporteur to further interpret the evidence that we have submitted so far. I can't actually comment on what that process will look like because I have to be thoughtful in the level of details we provide.

But our confidence is grounded in the totality of the evidence and the clear unmet patient need. As you saw, we continued in our global EAP to add an additional 10 patients, up to 132 patients. Those are primarily patients that are in Europe. And I think that as we continue to move forward with our reexamination process, we're looking forward to bringing new expert voices into the procedural discussions and allowing for the comprehensive evidence package to shine.

On that note, I'll ask Justin to talk a little bit more about our strong financial foundation and what we're doing moving forward.

Thank you, Neil, and thank you, Kristen. Yes, as you noted, we ended Q2 with $260.2 million in cash, cash equivalents and investments, and that really provides us meaningful flexibility. Our priorities are unchanged as we execute against our strategic plan, namely we're driving MIPLYFFA growth, pursue global opportunities for MIPLYFFA, continue to advance celiprolol and again, evaluate, of course, aligned external opportunities. So we're going to maintain that disciplined capital allocation strategy we've always talked about. We're going to be prudent in our investments and really focus on value-creating opportunities when we can find them.

And we will take our next question from Sumant Kulkarni with Canaccord Genuity.

This one is about the EMA process. What exactly could you do to strengthen your -- I guess, the request for reexamination? Is there anything new you can file in terms of the real-world data that you have generated? Or I guess the best way I could ask that is what is going to be new in this request versus what was denied in the prior documents that you sent in?

Thanks, Sumant. So I think it's a really important question. The opinion that the CHMP provided reflects their assessment on the submitted evidence under this European regulatory framework that they have. Under the reexamination process, we'll have the opportunity to focus specifically to address these specific grounds that have been cited for the refusal.

We believe strongly that the comprehensive evidence package, I mean, we've got an open-label extension study that goes out 4 years, actually just this week, the Expanded Access Program data that has been published has gone out for 4 years. The pediatric sub-study has been published now, along with a very comprehensive safety database.

So this robust and comprehensive package that we've resubmitted as part of this application, we're now going to go into the further interpretation of the evidence that are specific to the questions that they -- or the specific grounds that were cited for the refusal and bring it to light. And that comes with new expert voices into the procedural discussion that will allow us to continue to educate the CHMP on this -- again, I've said it a few times, but robust and comprehensive evidence package.

And we'll take our next question from Eddie Hickman with Guggenheim Securities.

Congrats on all the progress so far. Just two from me. On the U.S. side, can you just give us maybe some directional color on which of the segments you're seeing a rebound in? Is it sort of the GeneDx contributing to more diagnoses? Are you seeing a different level of new versus previously diagnosed, adult versus pediatric? Just help us understand sort of where the biggest opportunity remaining is?

And then on the ex U.S. side, can you sort of maybe help us bracket the sort of variable non-French EAP numbers sort of -- I know it's going to be sort of up and down maybe, but can you maybe sort of give us a min-max or some sort of bracketing for the next couple of quarters on how we should model that?

Let me start with your second question in regards to the global EAP. And I think you're specifically asking about the reimbursed section of that. We've said previously that our French program has been very consistent. It's approximately $2.5 million net per quarter or $10 million net per year. And that program has been ongoing for a multitude of years, and we have about 30 patients, plus or minus, that remain very consistent.

The variability in ordering patterns that come through other named patient reimbursement programs can come in -- it's one patient at a time, and it can be one patient that orders for an annual basis, one patient that orders for quarterly or a monthly supply. So it's really hard for me to tell you or guide you in regards to the brackets that you're asking for because it remains unpredictable. It's driven by the timing, the order volume, the program type and also the geography. So I can't -- the only thing that I can tell you is that we remain very committed in the French EAP program, which has been consistent at about $10 million net per year or $2.5 million -- approximately $2.5 million per quarter.

Let me switch quickly to the U.S. business. Really great questions. The U.S. business we're learning more about every day. We are seeing an equal mix between children and adults. As you know, this has been primarily a child disease for many years and kind of characterizes that. Now we're seeing quite a bit of adult patients. So those learnings are coming through.

We don't have a single tactic that is working to uncover the number of patients we have. And remember, we're at 184 enrollment forms now with 14 in the quarter. This is really a integrated strategy, and I'll ask Josh to talk more about some of the things he's seeing on the ground, but an integrated strategy versus a single tactic, that is continuing to drive this and kind of unlocking the TAM that we think is between the 350 and 900. Josh?

Yes. Thanks. Eddie, thanks for the question. As Neil mentioned, I wouldn't attribute the enrollments to any one tactic. In fact, we have a really well-integrated commercial strategy that's focused on accelerating diagnosis and treatment. And we're seeing that really take place with the 23 enrollments that we've seen in the first half of this year.

Those enrollments are coming with a mix of newly diagnosed patients as well as previously diagnosed patients. It's a mix of patients in centers of excellence as well as now we're able to reach out into the community and find patients who are in community practices. And we're seeing patients who are young as well as adult patients. So we're really pleased with the diversity and the distribution of enrollments that we're seeing, most notably because it really gives us confidence in the overall addressable market that we've been talking about, which is, we believe, somewhere between 350 and 900 patients.

And we will take our next question from Jason Butler with Citizens.

Just wondering if you could give us some kind of perspective on the age distribution of newly diagnosed patients? And then secondly, when you look at the tools that you're using like the AI targeting and the Expert Connect program, are you seeing any growth in the number of prescribers over the last couple of quarters?

Yes, sure. In terms of the age distribution of newly diagnosed patients, we are seeing patients who are coming in, who are both under the age of 18 as well as some who are over the age of 18. And just within the past -- this first half, we have some very specific instances. There was a newly diagnosed child who is about 2 years old, symptoms have just been presented and the parents had her tested and confirmed with NPC.

On the other hand, we have a patient who is in his mid-20s and has been misdiagnosed with epilepsy for 5 or 6 years and was finally tested and confirmed to have NPC. So we're really seeing a diversity of instances and age groups reinforcing the heterogeneity of the disease.

And this is largely driven by the tactics that we put in place, the AI targeting, our disease awareness campaign, all of these things are really helping to drive enrollments and again, giving us confidence that there are more patients out there yet to be diagnosed.

Yes. Jason, just to put a little bit of a finer point on this. We've said that the age distribution between adults and kids is about 50-50 in what we see today. So of these 184 enrollment forms, you should think of that as half adults and half children. When it comes to the newly diagnosed patients, it's really difficult for us to be able to say -- and provide that level of guidance.

So for us, you should consider to say this is not just a childhood disease as we've been thinking about this for many, many decades. These adult patients that are coming in today have got delayed diagnosis. They've got complex disease histories, and we're starting to unlock that. So the 50-50 ratio remains of our total population.

[Operator Instructions] We will move next to Brandon Folkes with H.C. Wainwright.

Congrats on another good quarter. Neil, maybe just how do you feel about enrollment forms today halfway through 2026 when we think about the full year compared to the enrollment forms you generated in 2025? It does look like things are trending up on an enrollment basis. But do you have better visibility into enrollment trends today? And are you starting to get more comfortable with sort of a quarterly level of enrollment forms you expect going forward?

Thanks, Brandon. So yes, you asked a question in regards to 2025, we had 52 enrollments for the year. And halfway through this year, we're at about 23 enrollments. I think what gives me a lot of confidence is really around the opportunity to unlock newly diagnosed patients.

We started seeing that around this time last year. And then in Q3, we saw more newly diagnosed patients, Q4 newly diagnosed patients. And these efforts that Josh just mentioned previously around our bespoke AI model that's getting the EMR data along with the claims data together and helping our reps get to places where they can educate physicians that believe -- that we believe have NPC patients, it's really driving the -- both newly diagnosed as well as the previously diagnosed patients.

So I'm pleased with where we are through the year, but I'm even more confident about the TAM being somewhere between that 350 and 900 because we're seeing these newly diagnosed patients earlier in the launch process than we would have expected today. And I'm hopeful that with continued investment on our side and others in the area in NPC, we'll continue to drive that forward.

And then maybe just one more for me. I heard your earlier commentary on celiprolol. But any additional color you can provide coming out of that meeting, that 1Q meeting and sort of the continued dialogue with the FDA? Did you come out of the meeting more confident in going ahead? Or what gives you confidence in terms of sort of moving that program forward coming out of that meeting? Any color would be helpful.

Well, I would call the meeting as informative. We went in to try and find ways to accelerate the clinical development program. And the questions we asked were responded to with opportunities for us to go out and do some homework. And we kind of have several approaches that we're working on here. It's a parallel track, right? One is that we want to try and find ways to accelerate the clinical development through regulatory pathways.

And the other is really around us and the ability to drive enrollment so we can get to the events and get to this interim analysis. As you saw last quarter, we had two confirmed events. We have three confirmed events now in Q2. We're up to 66 patients enrolled of 150 patients in the study, but we'd like to be able to find ways to put our foot on the gas, and that's what our second half plans are going to be, discussing with the FDA.

And we will take our next question from Lachlan Hanbury-Brown with William Blair.

Maybe the first, Neil, going back to sort of some of the prior questions. But just wondering what gives you confidence that you can be successful in the reexamination? I understand your point about confidence in the overall package, but sort of confidence in maybe being able to get the rapporteurs over the line or beyond the hurdles that they've seen in this initial review because they typically associate AMU with being totally flexible?

And then maybe a second question, just wondering if the IP win for MIPLYFFA in the U.S. changes how you think about your ability or maybe appetite to invest in the MIPLYFFA franchise in the U.S.?

So let me start with your first question. I'm going to be thoughtful in the level of detail I provide, but our confidence is fully grounded in the totality of the evidence and the unmet need, right? We mentioned again, 132 patients in our global EAP. The majority of those patients are patients that are in the European Union. And I think that, that's an important perspective. So we continue to see the demand from patients.

When it comes to the CHMP and their assessment, this process that we're working through and the stage that we're in around the reexamination process, is really around now focusing on that further interpretation of the evidence that we've provided around the open-label extension, the EAP and the pediatric sub-studies, but it's also -- it starts with our pivotal study, right? It's primarily centered on the analysis of the pivotal study as the foundation of this application.

So we've got a positive, clinically meaningful and statistically significant clinical study. That's what gives us the first part of confidence. The rest of it is around the comprehensive evidence package. So I can't give you a probability, but what I can tell you is that we're bolstering our clinical trial with long-term clinical experience supported by multi years of safety database with no new safety concerns, and we believe strongly that the benefit risk is in favor of MIPLYFFA for patients in the EU.

The second question that you had was in regards to -- I'm going to take that as life cycle management and IP. Yes, we're very happy with the longer duration of intellectual property protection today. And that does give us a lens towards looking at how we can further invest the capital Justin was talking about earlier today, into the U.S. business, but also into the European and global expansion along with the celiprolol program. So I kind of leave it at that at this point in the game. I don't want to say that we have a specific action that we'll take with the current extension, but we are looking forward to maximize the value of MIPLYFFA over the long haul.

And we will take our next question from Kambiz Yazdi with BTIG.

Congratulations on the quarter. Just one for me. On the reexamination, how are you thinking about the role of the SAG meeting and KOL testimonies potentially in the process? And also maybe on your strong cash position, can you speak about your philosophy on share buybacks?

I'll take the first one, and I'll ask Justin to comment on our capital allocation plans. The answer to that question is we did resubmit for reexamination, and we have requested a SAG meeting. That's part of bringing new expert voices into the procedural discussions, and we will take advantage of that. Justin?

Yes. Thank you. And again, reiterating what we said earlier, we're in growth mode. Our priorities remain unchanged against our strategic plan right now, and that's driving MIPLYFFA growth, pursuing global opportunities, advancing celiprolol. And again, as we just discussed, prudently at least exploring the opportunities that we might be able to invest in perhaps arimoclomol and other indications. So we're going to be very much open-minded to things. So we have a Board and we discuss these topics. But right now, that is not part of our current capital allocation strategy.

And we will take a follow-up question from Sumant Kulkarni with Canaccord Genuity.

So is there anything you can share about what your latest competitive intelligence tells you about the remainder of the diagnosed and treated patients in the U.S. that are not yet on MIPLYFFA, especially in terms of their being on the other approved products like IntraBio's AQNEURSA?

Yes. I recall this question. It's important. We believe there are complementary mechanisms of action. And I think the treatment guidelines reinforce what we've been saying for many years now that when you have a heterogeneous disease that has multi symptomatology and just devastating progression, it's important to have as many complementary mechanisms as possible that you can utilize.

I'll ask Josh to talk a little bit more about what we see in the marketplace. But what we have been seeing is the guidelines along with our current real-world evidence that patients and physicians want options, and we're seeing combination therapy being a -- not just a recognized approach, but more common than not approach.

Yes. As Neil mentioned and as we talked about previously, Sumant, the market is really moving towards a combination therapy because the treatments that are available are very complementary to one another. And this was reinforced in the treatment guidelines that came out earlier this year. Also important in those guidelines, it was recognized that MIPLYFFA was a disease-modifying treatment and is the only treatment that FDA indicated in combination use with miglustat. So as the market moves towards combination therapy, I think it really supports MIPLYFFA's ongoing use as foundational therapy.

To your question about other products out there, we don't really see this as an either/or market. Many patients -- certainly, we know that many MIPLYFFA patients are receiving miglustat and perhaps other treatments. And so we look at this really as an opportunity to continue to grow our share of those diagnosed patients and more importantly, the undiagnosed patients. The activities that we're doing now, we're seeing new enrollments coming in on a regular basis for newly diagnosed patients, all of which gives us real confidence that the overall market size is what we think it is of 350 to 900 patients.

And we will move next to Jonathan Aschoff with ROTH Capital Partners.

Solid quarter, guys. Can you -- just two things. Can you tell us anything about patient attrition, any commercial patients who are no longer taking the drug and maybe a percentage there? And how did the end of 2Q inventory compared to the end of 1Q?

Jonathan, I'll ask Josh to talk a little bit about the refill rate persistency and what we see in the market today and then I'll add a little bit about the ending inventory.

Yes. With regards to attrition, first of all, as we noted, we've got about 69% of covered lives. So we are very confident in our -- in the access that we're able to provide for patients, either through direct formulary for those 69% of covered lives or through some sort of medical exception pathway, and we're seeing that most of the patients who are enrolled to receive MIPLYFFA do indeed receive MIPLYFFA. Our refill rates and compliance rates or persistence rates are really quite high.

The patients that we do lose, unfortunately, are -- it's really a reflection more of the severity of the disease. It is a oftentimes fatal disease and patients will progress to the point of dying. And the patients who we have lost, they're no longer taking MIPLYFFA, the vast majority of those are patients who have unfortunately passed away.

Yes. Jonathan, just to put a finer edge on that, we had discussed that patients that get on MIPLYFFA seem to stay on MIPLYFFA. Our EAP data was very supportive of that effort. The rates that we saw in terms of the patients who, as Josh mentioned, unfortunately passed away, those rates are really super small. We're continuing to see those similar kind of rates in our long-term data so far. Now we're almost 2 years in. At the end of this year, we should be able to start seeing some more persistency and maybe we'll be able to provide that metric later on. Justin, do you want to talk a little bit about inventory?

So in general, we have a channel inventory target that we work with our specialty pharmacy partner on and our channel inventory fell below our targeted range at the end of Q1, was truly because of shipment timing and the dynamics around that. And for Q2, it was essentially back to the normal target level that we aspire to. So I believe you'll see a normalization going forward of this level.

Okay. So just a wee bit higher, back to normal is what you're saying?

Correct.

And this does conclude the Q&A portion of today's call. I'd like to turn the call back over to Neil McFarlane for any closing remarks.

Thank you for joining the call today, and we look forward to keeping you apprised of our future progress. Have a great evening.

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

Good afternoon, and thank you for joining Zevra's First Quarter 2026 Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our first quarter 2026 financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investors section of our website.

Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied in any forward-looking statements due to risks and uncertainties associated with Zevra's business. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and our filings with the SEC.

I am pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; Josh Schafer, our Chief Commercial Officer; and Justin Renz, our Chief Financial Officer. Our Chief Medical Officer, Adrian Quortel, will also be available for today's question-and-answer session.

Now it's my pleasure to hand the call over to Neil.

Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. We are building a durable rare disease company grounded in disciplined execution, financial strength and a commitment to patients. We continue to advance our strategic plan, delivering strong performance and positioning ourselves for long-term growth. We made substantial progress in establishing MIPLYFFA as a foundational treatment for Niemann-Pick disease type C or NPC in the U.S. by delivering meaningful clinical impact to patients and pursuing multiple pathways to expand patient access globally. We are also advancing our late-stage asset, Celiprolol, through a Phase III study for the treatment of Vascular Ehlers-Danlos Syndrome, or VES, and executing several approaches to accelerate its development.

To sharpen our focus on high-impact activities and remove operational distractions, we optimized the portfolio by divesting noncore assets with the sale of the SDX portfolio, to Commave Therapeutics for $50 million and concurrently resolved the legal dispute. Our balance sheet is strong with a cash position of $236.8 million and no outstanding debt, providing financial flexibility to drive growth. In the first quarter, total net revenue was $36.2 million, which is a 78% increase over Q1 2025.

We've now reached a total of 170 prescription enrollment forms for MIPLYFFA from launch through March 31, nine of which were received in the first quarter. Recall, the estimated prevalence of NPC patients in the U.S. is approximately 900, of whom 300 to 350 are currently diagnosed. Thus, we have successfully reached roughly half of this patient population and continue to have traction in newly diagnosed patients. This is a significant early launch achievement and remains consistent with the meaningful opportunity ahead for continued growth.

As a reminder, we've established a solid patent position for MIPLYFFA. It received orphan drug designation in the U.S., enabling marketing exclusivity through 2031. Consistent with our strategy to maximize the potential growth drivers for our business, we're pursuing a patent term extension through the U.S. Patent Office and await their decision, which could provide coverage beyond 2031.

Through our global Expanded Access Program, or EAP, we are able to deliver a much-needed treatment to patients with NPC in certain European countries and select territories outside of Europe, including the U.K. As of the end of the quarter, we have a total of 122 patients enrolled in the EAP across geographies. Our global EAP is comprised of multiple access programs, including compassionate use and named patient reimbursement. Within each, we expect variability in enrollment and reimbursement over the first few years until the patient base has stabilized, consistent with our experience in the French EAP program.

In Europe, there are an estimated 1,100 individuals with NPC. Diagnosis rates exceed those in the U.S., largely because the earlier approval of miglustat established physician awareness and enabled patient identification.

To support the geographic expansion with a potential approval of Arimoclomol in Europe, we have a marketing authorization application under review by the European Medicines Agency, or EMA. We submitted our responses to the EMA's 120-day list of questions within the 90-day clock stop period and are progressing along the standard review process to make Arimoclomol available to the European NPC community.

As a reminder, we have also received orphan medicinal product designation in Europe for the treatment of NPC. Turning to our late-stage pipeline. Our Phase III DiSCOVER Trial is evaluating Celiprolol for the treatment of beds, a rare inherited connective tissue disorder caused by COL3A1 gene mutations that weaken the walls of blood vessels and hollow organs and can cause arterial rupture or dissection among other complications. Approximately 90% of patients experience an event by the age of 40. There are roughly 7,500 individuals living with beds. Celiprolol is a selective adrenoceptor modulator that works by inducing vascular dilation and smooth muscle relaxation and thereby reducing mechanical stress on tissues of the arterial wall and hollow organs.

Currently, there are no approved therapies for beds. Our commercialization strategy for Celiprolol is focused exclusively in the U.S., where there's a clear opportunity to fill an unmet need. While not approved in Europe for beds, Celiprolol is the primary off-label treatment in several European countries. This use is supported by the results of several studies, including long-term European cohorts.

We've enrolled a total of 62 patients in the DiSCOVER Trial with 10 patients enrolled in the first quarter. This is an event-driven study, and we have two confirmed events out of the 28 events required to trigger the interim analysis. We continue to implement activities aimed at driving enrollment, including building a network of genetic testing centers to improve diagnosis as well as strengthening connections with key specialists who manage these patients.

In parallel, following the FDA Type C meeting we had in the first quarter, we are preparing for a follow-up meeting in the second half of the year to explore pathways to accelerate its clinical development.

In summary, we have a clear vision to become a leading rare disease therapeutics company, and we're motivated by the momentum and the opportunity that this phase of growth brings.

I'll now turn the call over to Josh to review our commercial performance in more detail. Josh?

Thank you, Neil, and good afternoon. Before reviewing the quarterly progress with MIPLYFFA, I'll provide a quick background on NPC. NPC is a rare lysosomal storage disorder caused by mutations in genes that impair intracellular cholesterol and lipid trafficking, leading to the abnormal lipid accumulation in the brain, liver, spleen and other organs. The onset and course of disease are heterogeneous, ranging from infancy to adulthood with progressive neurodegeneration that can vary in both speed of onset and clinical presentation. The extensive data generated for MIPLYFFA and NPC has shown long-term meaningful patient outcomes through the most expansive clinical development program in NPC to date.

We have more than 5 years of data across more than 270 NPC patients worldwide through clinical studies, including our pivotal trial, open-label extension study, global EAP and pediatric substudy. All demonstrating MIPLYFFA's efficacy and safety. Notably, MIPLYFFA in combination with miglustat is the first and only disease-modifying therapy shown to halt disease progression at 12 months in a randomized controlled trial based on the validated NPC clinical severity scale.

The onset of benefit is rapid with improvements noted at the first clinical evaluation time point of 12 weeks and has durable efficacy with treatment effects sustained for over 5 years. We are pleased to announce that MIPLYFFA was added to the NPC clinical practice guidelines, which were recently published in the Journal of Inherited Metabolic Disease, marking the first update to the guidelines since initial publication in 2018. These guidelines discuss the heterogeneity of the disease and reinforce that the NPC Clinical Severity Scale and genetic testing are the most reliable clinical endpoints and confirmation of diagnosis.

The guidelines also point out, consistent with our messaging that early detection is critical to delay disease progression. MIPLYFFA's mechanistic and clinical differentiation is resonating with prescribers and patients and is driving adoption. As Neil shared, we have received a total of 170 prescription enrollment forms since launch with 9 enrollment forms coming in Q1.

Our commercial strategy is focused on three key priorities: accelerating time to diagnosis and treatment, driving demand and facilitating access to MIPLYFFA. NPC remains significantly underdiagnosed and often diagnosed late due to heterogeneous symptoms. To enable earlier diagnosis, we have focused on education and engagement within the medical community through a strong presence at medical present data and through our ongoing disease awareness campaign called Learn NPC, Read Between the Signs.

Additionally, we have built a custom AI-driven targeting model to find likely NPC patients and have collaborations with providers of genetic testing to accelerate their diagnosis. As a result, we continue to see new enrollments from previously diagnosed as well as newly diagnosed patients. We are also finding patients and seeing demand for MIPLYFFA increase outside of the centers of excellence.

Our prescriber base is expanding to include community-based prescribers, which we believe reflects the success of our targeting and education efforts. Many of these new prescribers did not know that they had NPC patients in their practice and were previously unfamiliar with the disease and treatment options. We help facilitate medical education efforts through various initiatives such as the recently launched Expert Connect, which connects HCPs unfamiliar with NPC to experts who can address questions regarding disease state and available treatment options.

Our patient mix has grown to include adults and children equally. These trends give us confidence in the estimated prevalence of 900 people in the U.S. living with NPC, and we remain focused on reaching as many patients as possible and expanding the total addressable market.

From a market access standpoint, we have stable coverage of 69% and continue to achieve reimbursement through the medical exception pathway. Payer engagement continues to be focused on emphasizing our robust clinical safety and efficacy data and the extensive real-world evidence seen in clinical practice that supports MIPLYFFA's value. We believe clinical benefit support services and broad patient access and independent market research suggests MIPLYFFA is the preferred NPC therapy most trusted by clinicians and shown to improve balance, swallowing, cognition, speech and reduce falls.

We received heartwarming letters from families noting how positively impacted they have been by MIPLYFFA and the support of our Amplify Assist patient services program. Together, this feedback reflects the impact of our commercial activities and sets the stage for continued growth.

With that, I will turn the call over to Justin to review our financial results.

Thank you, Josh. In addition to the financial details included in today's call, we encourage you to refer to our quarterly report on Form 10-Q for more detailed information, which we intend to file shortly. As Neil mentioned, in the first quarter of 2026, we generated net revenue of $36.2 million, which was an increase in total net revenue of $15.8 million compared to $20.4 million in Q1 of 2025. This is comprised of $24.6 million from MIPLYFFA net sales in the United States, $0.3 million from OLPRUVA, $10.2 million in net reimbursements from the global EAP for Arimoclomol and $1.1 million in royalty revenue. It is worth noting that we had 1 less shipment week of MIPLYFFA in the U.S. due to the first quarter delivery calendar. And as a result, channel inventory fell below the low end of our targeted range.

Turning to recent business transactions. In March, we executed an agreement with Commave Therapeutics for the sale of the SDX portfolio for $50 million, monetizing assets that were not central to our core investment thesis. Under our contractual obligations, Aquestive Therapeutics received 10% or $5 million of gross proceeds. In the first quarter, we received $40.5 million of the $45 million in net proceeds, and we received a final payment of $4.5 million in April. In connection with this transaction, we reviewed our capital allocation strategy and retired our debt early, saving on average approximately $8 million a year in future interest expense. We are now debt-free and strategically positioned for growth, supported by a clean balance sheet. These onetime transactions impacted our first quarter financials.

Accordingly, we recorded a onetime gain of approximately $43.3 million, partially offset by an approximately $10 million expense associated with the early extinguishment of debt, which is noted in the other income and expense section in our financial statements. Pivoting back to normal operations. During the first quarter of 2026, our operating expenses were $25.2 million, which was an increase of $2.4 million compared to the same quarter a year ago. R&D expense was $4.4 million for Q1 2026, which was an increase of $1.1 million compared to Q1 2025 due primarily to increases in third-party costs and professional fees.

SG&A expense was $20.8 million for Q1 2026, which was an increase of $1.2 million compared to Q1 2025, primarily due to an increase in professional fees, partially offset by a decrease in third-party spending.

We have utilized the vast majority of our usable net operating loss carryforwards. And as a result of the multiple onetime transactions that we recorded in the first quarter, we incurred an estimated tax provision of $6.9 million.

Net income for the first quarter of 2026 was $37.9 million or $0.62 per basic and $0.60 per diluted share compared to a net loss of $3.1 million or $0.06 per basic and diluted share for the same quarter a year ago. Excluding the onetime transactions as well as the related tax provision for clear comparability across periods, the estimated quarterly net income reported would be $11.5 million or $0.18 per diluted share. As of March 31, 2026, Total cash, cash equivalents and investments were $236.8 million, which was a decrease of $2.1 million compared to December 31, 2025.

As mentioned earlier, this decrease is attributable to deleveraging driven by our debt payoff, partially offset by the non-dilutive capital proceeds from the sale of the SDX portfolio and supported by our operating income. Collectively, these factors have further fortified our balance sheet, and we remain well positioned with the financial capacity to execute on our strategic priorities independent of the capital markets.

And now I'll turn the call back to Neil for his closing remarks. Neil?

Thanks, Justin. Our corporate profile has evolved significantly with the execution against the strategic pillars we introduced a little over a year ago. We are delivering strong commercial execution while thoughtfully monetizing assets that are not core to our business. We relocated our corporate headquarters to Boston, a hub of biotech innovation, and we strengthened our leadership by attracting seasoned professionals to our executive management team and Board of Directors, bringing valuable experience and perspective. These efforts, combined with prudent financial stewardship, are positioning us to deliver on our vision. As we advance through 2026, we are anchored by a clinically meaningful commercial product with multiple opportunities for global growth, a late-stage pipeline and a strong financial position.

Our collective team at Zevra is energized by the numerous opportunities we have to expand our impact for people living with rare diseases. Operator, please open the line for questions.

[Operator Instructions] Our first question today comes from Kristen Kluska with Cantor Fitzgerald.

This is [ Ian ] on Kristen's line. Congrats on the quarter update here. First, now that MIPLYFFA has been added to the NPC clinical practice guidelines, we're just wondering what this means for physician adoption. I mean are these doctors now formally advised to consider MIPLYFFA when treating these patients?

We're really pleased. And I think as we've been previously communicating, these guidelines were in process and really came out quite fast after the introduction of new products that were approved in the U.S. It really reinforces the NPC severity scale as the tool that shows disease progression, the genetic testing as a key endpoint and diagnostic tool. The complexity of the disease. When we think about the heterogenicity of both infantile version and adult versions of the disease, it's really important to early detect in order to be able to delay progression. But really importantly, the combination therapy being considered for NPC was one of the major takeaways. I'll ask Josh to talk a little bit about some of the impact that we see and quite frankly, the hurdles that are lowered with these guidelines that have been published.

Yes. Neil, just to add to what Neil said, we're also really pleased that the guidelines addressed the need for early detection and that early treatment helps delay progression. I also went on to talk about the importance of using combination therapy for patients who have been diagnosed with NPC. And this is really important because these are the opinions of a select group of key opinion leaders that we're now able to use and communicate and help build confidence and consistency in the way that some HCPs who might not be as familiar with NPC can now use this as a consistent guide for their treatment.

And then second, do you have a sense of the proportion of the patients that are identified through the genetic testing that you're conducting and the AI-driven predictive model that ultimately convert onto the drug? I guess -- and just... go ahead.

No, sorry, please finish your question.

Yes. Just related to that, I was wondering what feedback you're hearing from the physicians that are managing these patients in terms of like how receptive they are being to the data-driven identification approaches that you're using?

Wonderful question, and thanks for outlining our commercial strategy really clearly for us. Let me ask Josh to touch on some of the key priorities that we're executing against and some of the feedback that we're getting.

Yes. As we mentioned in the prepared remarks, we have three key priorities from a commercial perspective. The first is to accelerate the time to diagnosis and treatment, and we're seeing that through our disease awareness campaign as well as some of the collaborations with the genetic testing companies. Our second priority is to really drive demand and you're seeing that in the enrollments that we've been able to get over the past quarter and more and then facilitate access with 69% of covered lives -- or 69% of covered lives currently able to access MIPLYFFA and the others we're able to do through medical exception pathways. So we feel really confident in our line of sight for more patients, given what we're seeing today, which is a nice mix of newly diagnosed as well as previously diagnosed patients.

And our next question comes from Kambiz Yazdi with BTIG.

EAP revenue growth was quite robust. Is France driving that growth or other countries? And how should we think about geographic composition of the EAP as a leading indicator for where commercial demand may concentrate post EU approval?

And welcome to the analyst coverage. As we reported, 122 patients enrolled in Q1 2026. This really encompasses multiple access programs. We have and multiple territories. In Europe, we've had outstanding for quite some time, our French EAP experience, which we've previously guided and consistently receive about $10 million net per year now that our patient base has stabilized in that territory, and that was per year. Today, we have new markets that are coming on. And that variability in ordering pattern and the rates of new enrollments and the reality is that we've got both compassionate use as well as named patient reimbursement. All of these new attributes lead to the variability that we continuously guide towards. It's early. We have new distributors. We're really pleased, though, with our new distributors and actually all of our distributors and how fast they're able to be able to get the named patient request for individual patients, then drive that back through to being able to get through our system and the product to patients in these territories.

So we're really pleased with the continued inbound in these markets. And our goal is to really expand access to as many markets as we can while still focusing on our key territories in the U.S. and expanding the diagnosis and treatment, along with our global expansion, i.e., Europe and the MAA as well.

And then maybe quickly as a follow-up, can you share with us any quantitative milestones or leading indicators you're tracking internally around your bespoke AI-driven patient identification and genetic testing efforts in identifying newly diagnosed NPC patients?

Let me ask Josh to work on some of those strategies that are starting to really give us confidence in the TAM.

Yes. And we monitor and measure a number of things, which will remain internal and metrics that we use just internally. But we are measuring how many patients are we finding that are newly diagnosed. We're looking at how many new prescribers we're able to bring into the mix as well. And then other interesting dynamics about the patient's journey, perhaps any other treatments that they may have been on, other diagnoses and other symptoms, all of that is tracked as we look to really understand as much as we can about these patients. This program is working extremely well, and it's allowing us to look at those patients who we know are diagnosed with NPC and find like patients who have not yet been diagnosed and be able to work with clinicians in accelerating their diagnosis and treatment.

Yes. Let me add a little bit to that. I think it's important because we talk about the heterogeneity of the disease. The patient journey in NPC, unlike a lot -- so I should say, not unlike a lot of other rare diseases, it is you know one patient, you know one patient. We're talking about children and the primary data that has been developed over many, many years in NPC has been really child-based. In our experience and the expanded access program in the U.S., we built that out, and we saw that it was about 50% of the patients that were children and 50% of the patients that were adults. In our real-world experience moving forward, we still see that. Now that means that we have to continue to learn about the journey of the patient that is the adult patient and understand how we can then make the tools and continue to evolve our commercial strategy and educate physicians that patients in later ages also can present with NPC. So this is early in our launch. We're really pleased with the 170 total enrollments, the 50% children and adults, but we're still learning a lot about what it means for adult patients.

Our next question comes from Sumant Kulkarni with Canaccord.

I have two, one on MIPLYFFA, one on Celiprolol. You mentioned that MIPLYFFA is now at almost 50% share of the diagnosed and treated patients in the U.S. What does your competitive intelligence tell you about your share of the competitor AQNEURSA in the remainder and your ability to gain further share in this 300 to 350 patients? And do you know of any cases where payers are allowing concurrent use of both MIPLYFFA and AQNEURSA in the same patient?

We'll take one question at a time here in regards to the MIPLYFFA question. You are correct, and I'll ask Josh to double-click on this a little. You are correct that also based on the treatment guidelines that were just recently established that a lot of patients, and I can't give you the exact amount, but a lot of patients because of the complexity of the disease as well as the symptomatology of the disease are on multiple therapies, and we're actually seeing success in getting those patients on miglustat and MIPLYFFA and other therapies to be able to get them covered from a commercial perspective. These guidelines, I think, are going to continue to reinforce that combination therapy should be considered for NPC patients. We believe that, that bodes really well for us.

Our label is in combination with miglustat. We see that's where our clinical differentiation is in terms of disease modification, halting the progression of the disease and the durable effects that we see. So we see this as a real positive.

And I'll ask Josh maybe to talk a little bit about what he's seeing on the ground.

Yes. Just to add a little bit more color there. We have been seeing in clinical practice that clinicians want to be able to treat patients with as many different modalities and to approach the treatment from as many different angles as possible. And so we've seen this combination therapy for some time. And now that opinion is really reinforced by the treatment guidelines. This -- what this means is that those 50% of diagnosed patients that we're treating now, some of them very likely may be on other therapies. And this is not an either/or marketplace. This is really an and marketplace. And so we're seeing that continuing to grow. We're also seeing that coverage is continuing to exist for patients. We have 69% of total covered lives today. Those who are not able to get that through direct formulary, we're able to get coverage even for combination therapy through medical exception pathways.

Got it. On Celiprolol, we know you plan to meet with the FDA again in the second half of this year, but what are some of the specific options that you might have on the table in terms of your ability to move faster to bring this product to VEDS patients?

Yes. We mentioned in our last call that we had a Type C meeting in Q1 and that we're exploring pathways to accelerate the clinical development of Celiprolol. I would categorize the conversations as constructive, also informative. We're now -- this is the early stages. We're now in the process of preparing for the follow-up meeting in the second half of 2026. And quite frankly, it's too early for us to tell you what strategies we're going at. But the reality is that we're going in two directions. One, to continue to boost enrollment activities and the other is to accelerate the clinical development through additional pathways with additional data.

Our next question comes from Eddie Hickman with Guggenheim Securities.

Congratulations on all the progress. Apologies if this has already been asked, I'm jumping between a few calls. But really nice performance outside the U.S. as well. So I'm wondering if you can give an update on the type of questions that you got from the 100 day, day 120 day. And are they clinical CMC or more commercial? And then what's the sort of time line for that updated CHMP opinion?

Yes. Eddie, and you have been the first to ask the question in regards to our marketing authorization application. As we mentioned in the prepared remarks, we did respond to the 120-day list of questions within the appropriate clock stop period. We provided the standard responses that are there. I won't get into the specifics of what it is the questions were or how we address them. But what I would continue to say is that since we've submitted a new application to the agency, we have not actually seen any new questions that we did not see out of the previous European submission and the FDA submission that we were able to then provide.

So the substantial amount of data that we have now and the robustness of the package. And as a reminder, it's the totality of the data is over 270 treated patients. including data from our Phase II/III study, the open-label extension study that's got 5 years of data, our EAP that's got over 5 years of data, along with the pediatric substudy. So it's new data and the engagement is fairly standard. So now we're continuing to engage along the path, and we look forward to our next engagement with the European regulators.

Our next question comes from Jason Butler with Citizens.

Two for me. Can you talk about the profile of the newly diagnosed patients that you're seeing? And again, speak to heterogeneity here. How early in the disease are they versus patients that may have had disease for a long time, but the diagnosis is delayed?

And then second question, you mentioned that the inventory was below the lower end of the range for range at the end of the quarter. Has that reversed so far in 2Q?

Yes. Jason, let me start with the first question, the profile of the newly diagnosed patients. And I know I say this a lot, but when you know one patient, you know one patient because of this heterogeneity of the disease, it's really important. One patient may present with a primary symptom that's an epilepsy. Another may present with gait instability. Another may present with cognitive challenges. And because of that, we're continuing to refine our -- as Josh talked about, the predictive modeling and claims data and all the things that we're doing, but we're learning along the way. So every time we get a new patient, it's further informing how we go out and try to continue to expand. And as I mentioned, this 300 patients to 350 patients, we have a really good line of sight to those diagnosed patients already. We've talked about the prevalence of the 900 in the United States.

We now have a lot more confidence that, that 350 and the 900, we're somewhere in between those according to the TAM and what we see as the addressable patient population. So let me ask Josh to talk a little bit about some of the characteristics we see, but the confidence is getting -- is growing every time we see newly diagnosed patients.

Yes. Just to bring this to life a little bit. We recently saw the diagnosis of a toddler who had an enlarged spleen and then went through genetic testing and was confirmed to have NPC. On the other end of the spectrum, there was an adult who had been misdiagnosed with MS for years and then changed physicians who happen to be aware of NPC and did genetic testing and confirmed that it was NPC. So it really runs the gamut, but there are some similarities and some characteristics both in terms of the symptoms as well as the journey that these patients go on that we're able to really learn from. And this is feeding our disease awareness campaign that we're continuing to educate physicians. We have a strong presence at medical conferences. We published a lot of data on this as well as the strong commercial efforts that we're putting in place.

And Jason, let me hand off to Justin to keep him going here this evening on the inventory question.

Yes. Jason, the way the Q1 delivery calendar fell, we just had one less shipment of MIPLYFFA in the U.S. than typical. And so as a result, our inventory in the channel was a little less than the low end of our targeted range. And so as a result, we do expect this to fall back within our targeted range by the end of the second quarter.

[Operator Instructions] Our next question will come from Brandon Folkes with H.C. Wainwright.

Congrats on all the progress. Maybe just two for me. Any color on the time from submitting an enrollment form to getting on product and how that is trending at this stage of the launch? And then secondly, is there a difference in net revenue realized per patient if it goes through a medical exception versus a patient whose insurance falls into the 69% of covered lives currently?

Brandon. let me ask Josh to talk a little bit about the enrollment numbers that we're seeing now.

Yes. I think your question was really around the time from an enrollment comes in until once a patient receives therapy. And it's varied. It is largely dependent on the payer type, whether it's government or whether it's commercial. We are seeing -- in Q1, we had a number of patients who went through a reauthorization process, which is very typical at the beginning of the year as patients might either change plans or plans might change their policies. And so that had an impact on the first quarter, and we're working through those reauthorizations. So it's a little difficult to give you kind of what the average or the standard is just because of that reauthorizations that took place in the first quarter.

Your second question, might have to -- this was about net revenue, and I'm not sure that I quite got that. Can you repeat that question for us?

I guess what's the priority to grow the 69% of covered lives? Given your success under medical exceptions, is there a difference in net realized revenue per patient that goes through insurance versus the medical exception?

Yes. No, that's a great question. I'll ask Josh to opine. But the 69% of covered lives allows for you to be on a formulary. It doesn't necessarily have a preferred position on the formulary. And it does speed up the process. A lot of our education that's gone to payers so far has really allowed us to be able to educate on the clinical benefits with the medical directors of this [ varying ] plans and achieve what we believe is a really good covered life plan. The important component of the question I think you just asked, which is maybe get into the potential for gross to net as well.

We do not contract currently today. So it's standard government discounts along with distribution margins and the like. So the net price hasn't really changed except for the variability in gross to net on a quarter-over-quarter basis.

Yes. And I think you're asking a very salient question around the 69% and our intent to grow that. We absolutely do, and we are making steady progress in that area, largely by talking about the clinical differentiation of MIPLYFFA. The guidelines certainly will help those discussions as well. But as you point out, it really doesn't impact the overall ability for a patient to receive MIPLYFFA because every plan has a medical exception pathway. What it does is it reduces a little bit of the time, it reduces some of the burden. We have a very robust patient services resources that we provide to help patients and offices navigate this. And so our goal is to make MIPLYFFA as accessible to patients as possible. And we do that both through increasing the covered lives, but also providing these patient services.

Thank you. This concludes the Q&A portion of today's call. I will now turn the call back to Neil for any additional or closing remarks.

Thank you for joining our call today. We look forward to keeping you appraised of our future progress. Have a wonderful evening.

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

Good afternoon, and thank you for joining Zevra's Fourth Quarter and Full Year 2025 Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications. Please go ahead.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our fourth quarter and full year 2025 financial results. I encourage you to read the financial results news release, which was distributed this afternoon and is available in the Investors section of our website.

Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by forward-looking statements due to risks and uncertainties associated with Zevra's business. Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from the projections made, and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K, and other filings with the SEC.

I'm pleased to welcome Zevra's management team members participating in today's call. Neil McFarlan, Zevra's President and Chief Executive Officer; and Josh Schafer, our Chief Commercial Officer; our Chief Medical Officer, Adrian Quartel, and our Senior Vice President of Finance, and Corporate Controller, [ Timothy Sangiovanni ] will be available for today's question-and-answer session.

Now it is my pleasure to hand the call over to Neil.

Thank you, Nicole. And welcome to everyone joining our quarterly call this afternoon. I want to start off by welcoming our newly appointed Chief Financial Officer, Justin Renz, whose official start date is today. Justin brings more than 25 years of biopharmaceutical financial leadership experience with a strong track record supporting global commercial operations, along with extensive experience in capital markets and Investor Relations. He will share a few brief remarks at the end of the call. However, we will not be taking questions on today's call.

At Zevra, we're on a mission to redefine what's possible in delivering life-changing therapies to people living with rare diseases. As a commercial stage company with a late-stage pipeline, we have a unique opportunity to make meaningful impact for patients. Our vision comes to life through our strategic plan and our core values of patient centricity integrity, accountability, innovation and courage. Guided by the execution of our plan and demonstrating our values, 2025 was a banner year with net revenue of $106.5 million driven by the performance of MIPLYFFA, which delivered $87.4 million in revenue. Specifically, in the fourth quarter, we generated $34.1 million in total net revenue, which included $26.4 million from MIPLYFFA sales. This momentum sets the stage for multiple growth drivers in 2026.

Our success was reflected in the strong commercial performance of MIPLYFFA, with 24 prescription enrollment forms received in the fourth quarter, bringing the total in the first full year of commercialization to 52 enrollment forms, and a total of 161 enrollments since launch. MIPLYFFA's is our foundational treatment for [ Niemann-Pick Disease ] Type C, or NPC. And this increasing penetration means that more patients are gaining access to the therapy they need. Importantly, this growth is being driven by previously diagnosed patients and increasingly by newly diagnosed patients. This trend reinforces our confidence in the estimated U.S. prevalence of approximately 900 affected individuals, of whom 300 to 350 are currently diagnosed. Later on the call, Josh will review some of the initiatives that are fueling our traction in the identification of newly diagnosed patients.

As a reminder, MIPLYFFA has exclusivity in the U.S. through 2031 under the orphan drug designation, and we are diligently pursuing a patent term extension, which is currently under review by the U.S. Patent Office. It's important to note that additional exclusivity would represent positive upside to our existing runway. We're expanding access to MIPLYFFA, or arimoclomol, beyond the U.S. through our global Expanded Access Program, or EAP, which had 113 patients enrolled at the end of 2025. Over the past year, we've built a strong reputation across Europe, in particular, through our global EAP, enabling clinicians to gain first-hand experience with arimoclomol.

In the fourth quarter, we established a new distribution agreement to extend the global EAP to select territories outside of Europe and initiated shipments of named patient supply prior to year-end. As is common with these types of distribution agreements, we anticipate variability in ordering patterns and the rate of new enrollments in the first few years until the patient base has stabilized. We're pleased with the rapid execution by our partner to ensure swift delivery to patients.

Importantly, in late July of last year, we submitted our marketing authorization application to the European Medicines Agency, supported by a robust data package, including our pivotal Phase II/III trial, open-label extension study, expanded access programs and our pediatric substudy. As a part of the standard review process, we received the 120-day list of questions from the EU regulators at the end of 2025. At this time, we are fully prepared to respond to these questions within the regulatory 90-day clock stop period, and look forward to our continued engagement with the agency to make arimoclomol available to the EU NPC community.

In Europe, NPC prevalence is estimated at approximately 1,100 individuals. And our current EAP is supporting nearly 10% of this population. Diagnosis rates are significantly higher than in the U.S. due in large part to the European approval of miglustat for the treatment of NPC, which provided a clear treatment pathway and has led to stronger position awareness and higher patient identification. As we continue expanding access and strengthening relationships with key opinion leaders, we are well positioned to maximize our commercial impact in Europe through strategic partnerships or independently.

Beyond MIPLYFFA, we're advancing [indiscernible] for the treatment of vascular [indiscernible] syndrome, or [ Vets ], a rare inherited connective tissue disorder caused by [ CALL3A1-GENE ] mutations. These mutations lead to defects in type 3 pro collagen in the walls of blood vessels and hollow organs, resulting in arterial dissections and organ ruptures. In the U.S., approximately 7,500 individuals have been diagnosed with [indiscernible]. [ Soliprelol ] acts as a selective [indiscernible] modulator, reducing mechanical stress on collagen fibers. Data on [indiscernible] generated outside of the U.S., including results from the [ BBEST ] study, and 2 long-term European cohorts demonstrated a reduction in major vascular events and improved overall survival among patients receiving treatment.

Our ongoing Phase III DISCOVER study designed to confirm the clinical benefit observed in prior studies. We enrolled 8 patients in the fourth quarter, bringing the total number of enrolled patients at year-end to 52 of the planned 150 patients to be enrolled in the study, and 1 confirmed event out of the 28 events required to trigger the interim analysis. We are continuing to implement strategies to boost our enrollment, including building a network of genetic testing centers to help diagnose individuals with beds as well, as connecting with specialists that [indiscernible] these patients, such as the vascular surgeons who intervene when a patient experiences an event.

In parallel, we recently engaged the FDA in a Type C meeting to discuss our regulatory options to accelerate the development program. While discussions are in their early stages, we appreciate the agency's responsiveness to our request and look forward to further engagement in the second half of the year.

Finally, from a corporate standpoint, we've moved our headquarters to [indiscernible] a strategic step that strengthens our foundation for long-term growth and positions us at the center of one of the world's most dynamic biotech ecosystems. This move enhances our ability to collaborate with industry leaders and access a deep pool of highly specialized talent, ensuring we remain well equipped to deliver on our strategic plan.

Before turning the call over to Josh, I would like to recognize that February was a rare disease [indiscernible] month. And to coincide with this, we were invited to ring the opening bell at NASDAQ. It was a powerful tribute to the patients who inspire our mission and drive us forward every day. It's especially important to connect with the community. And during the month, we also held an all-hands meeting, where we have the privilege of hearing from a mother of 2 children with NPC. Her story reinforced the importance of expanding access to diagnosis, treatment and support for the community. And it was a moving reminder of why we do, what we do for people living with rare diseases.

Josh?

Thank you, Neil, and good afternoon. We are thrilled by the commercial performance and the momentum we are seeing in the U.S. market. With 52 prescription enrollment forms in 2025, and 24 of them received in the fourth quarter, as well as our strong refill rate, we are building confidence in the continued growth and long-term potential of MIPLYFFA. We believe this reflects the compelling clinical benefit demonstrated by MIPLYFFA as the only disease-modifying therapy for NPC shown to have both a rapid onset of improvement within 12 weeks of treatment, and to halt NPC progression at 12 months, with durable efficacy and safety maintained for more than 5 years.

This clinical differentiation is resonating across stakeholders and is catalyzing adoption of MIPLYFFA. And we are seeing strong adherence to therapy once a patient begins treatment, similar to what we observed in the long-term U.S. EAP. As a reminder, NPC is a devastating and often fatal genetic disorder caused by mutations that impair intracellular lipid transport. As a result, cholesterol and other lipids progressively accumulate within cells, leading to widespread neurological and [indiscernible] dysfunction. NPC is clinically heterogeneous with significant variability in symptom presentation and rate of progression. Although it has traditionally been considered a pediatric disease due to its genetic origins, approximately half of the people treated with MIPLYFFA are adults, underscoring the need to recognize NPC across the lifespan.

As Neil mentioned, the growth we saw in the fourth quarter was driven not only by the increased adoption among previously diagnosed patients, but also meaningfully by people who were previously undiagnosed. This progress within the newly diagnosed segment strengthens our confidence in the overall market potential. We attribute this success to our 3-pronged approach to education and diagnosis. Through our disease awareness campaign called [ Learn NPC ], read between the signs, our custom AI-driven targeting model, and our collaboration with providers of genetic tests to confirm diagnosis.

Our disease awareness campaign has become a trusted go-to resource to effectively educate the community on the need for early detection and treatment of NPC. Whether through engagement at medical congresses or providing resources for individuals seeking reliable information online. Additionally, we have developed a bespoke AI predictive model to identify potential patients who may be misdiagnosed or undiagnosed. Using claims data and electronic health records, we analyze the symptoms and diagnosis and treatment journeys of patients who are confirmed to have NPC. We then apply these insights to a broader claims database to find undiagnosed patients with similar profiles.

And we recently expanded our partnership with [ GeneDx ], which adds to our existing collaborations with companies that provide genetic testing to confirm a diagnosis of those suspected to have NPC, serving to accelerate time to diagnosis and new patient identification. Eligible patients will be able to receive GeneDx's exome DX test at no charge, and results will be provided to clinicians and their patients in as little as 3 weeks to facilitate clinical decision-making.

This strategy revealed a number of unique patients who match these patterns and were previously unknown, allowing us to engage physicians with targeted education, turning data into action, and giving newly identified patients a chance for earlier intervention in their disease. We continue to educate the community on our robust clinical data and differentiated mechanism of action. At the [ WORLD ] Symposium last month, we presented 4 posters highlighting new data on MIPLYFFA. The growing body of evidence continues to support the impressive results we saw in our clinical trials, reinforced by the long-term real-world data we are generating. There were multiple presentations resulting from the data generated through our U.S. EAP program.

In a multiyear subgroup analysis of participants with up to 4 years of follow-up, MIPLYFFA demonstrated durable treatment effects, sustained clinical benefit and safety with significant patient adherence over that time. At [indiscernible], we also presented new analyses evaluating the adult NPC population and found those treated with arimoclomol in the U.S. EAP showed disease stabilization over the 4 years while maintaining a favorable safety profile consistent with the broader results.

Importantly, our market access strategies remain strong, and we continue to drive access for our patients. We are at 68% of Total Covered Lives, which is in line with where we expected to be after the first full year on the market. For the remaining patients, we have been able to secure high reimbursement rates through the efforts of our Market Access and Patient Services team. Our success reflects the benefits demonstrated by data generated in the clinical studies, as well as long-term real-world experience, which show MIPLYFFA's ability to halt disease progression with a favorable safety profile. In just 4 full quarters in the field, the team has built meaningful momentum and delivered measurable impact, and we couldn't be more proud.

We are expanding access to diagnosis and enabling disease-modifying treatment, while demonstrating clear leadership through our comprehensive support services and commercial execution. As a result, we are steadily establishing ourselves as a trusted partner of choice in the rare disease space.

Now I will turn the call back over to Neil.

Thanks, Josh. I'll briefly review a summary of our financial performance and encourage you to refer to our press release and SEC filings for more detailed information.

As I mentioned, in the fourth quarter, we generated $34.1 million in total net revenue. This included $26.4 million from MIPLYFFA sales, $400,000 from OLPRUVA, $5.6 million in net reimbursements from our global EAP, $1.8 million royalties and other reimbursements under the [indiscernible] license. Operating expenses for the fourth quarter was $23 million, which was a decrease of $1.5 million compared to the same quarter a year ago. R&D expense was $2.6 million for Q4, 2025, which was a decrease of $5.8 million compared to the same quarter in 2024, primarily due to a decrease in personnel-related third-party costs.

SG&A expense was $20.4 million for Q4, 2025, which was an increase of approximately $4.3 million compared to the same quarter in 2024, largely due to investments to support our launch activities. Net income for Q4, 2025 was $12.2 million or $0.20 per basic, and $0.19 per diluted share. For the same quarter in 2024, we reported a net loss of $35.7 million, or $0.67 per share.

Turning to the full year. Our 2025 net revenue of $106.5 million included $87.4 million from MIPLYFFA sales, $800,000 from OLPRUVA, $13 million in net reimbursements from the global EAP, and $5 million from royalties and other reimbursements under the stars license. Operating expense for the full year 2025 was $90.4 million, which was a decrease of $6.6 million compared to 2024. R&D expense was $12.7 million, which is a decrease of $29.4 million compared to the prior year, primarily due to a decrease in personnel-related costs, combined with a decrease in third-party costs.

SG&A expense was $77.6 million which was an increase of $22.7 million compared to the prior year, mainly due to an increase in third-party costs related to MIPLYFFA, combined with an increase in personnel-related costs, professional fees and other expenses associated with our commercial, medical and launch activities. Net income for the full year 2025 was $83.2 million, or $1.40 per basic and $1.35 per diluted share, compared to a net loss of $105.5 million or $2.28 per basic and diluted share for 2024. As of December 31, 2025, total cash, cash equivalents and investments were $238.9 million, and total debt was approximately $61.9 million.

In closing, we have meaningful value inflection points squarely [indiscernible]. In the U.S., we're driving commercial execution to further unlock the NPC opportunity, as well as pursuing a patent term extension. In the EU, we have our [indiscernible] filing before the regulators with one of the most comprehensive NPC data sets, along with expanded access through our global EAP in markets beyond Europe. And for our pipeline opportunity with [indiscernible], we look forward to further engagement with the FDA regarding potential options to accelerate our development plan.

The key takeaway is that strong execution, grounded and [indiscernible] therapeutic benefit for patients is translating into tangible bottom line results. We remain committed to prioritizing our investments towards high-impact initiatives which gives us the flexibility to explore additional ways to create meaningful value for our shareholders and the rare disease community that we serve. As I mentioned earlier, today's Justin's first official day with the company, and we're very pleased to have him joining us.

Before we open the call for questions, I've asked him to share a few brief remarks. Justin?

Thanks, Neil. It's great to be officially starting at Zevra today. In a short time, I've already seen the depth of experience and the genuine dedication to bringing meaningful therapies to patients living with rare diseases. Over the coming weeks, I look forward to listening, learning and engaging with many of you while partnering with the team to support Zevra's continued execution and long-term value creation.

And with that, I'll turn it back to Neil.

Thank you, Justin. We're glad to have you on board. Operator, please open the line for questions.

[Operator Instructions] We'll take our first question from Kristen Kluska with Cantor.

Congrats on a great quarter and Justin, I'm looking forward to working with you. Welcome on board. So 24 start forms is certainly impressive. You tripled it from 3Q. Can you give us any more color or details around the split of how many of those -- or percent were new diagnoses versus those getting identified patients on drug? And then how should we be thinking about the cadence moving forward? How does the pipeline look now? Are you still seeing people undergo the genetic testing, diagnosis, or looking more into the AI predictive targeting model?

That's great. Thank you so much. Let me start off by saying that we've talked about this on a quarter-over-quarter basis. You're going to see some variability. 52 enrollment forms in the full year, I think the number that we're really lined into, and when we think about the total of 161 total enrollments since launch, it reinforces our confidence in the U.S. prevalence. Especially, I'll ask Josh to talk a little bit about this. Especially with the identification of newly diagnosed patients, which from Q2 to Q3 to Q4, we continue to see some growth there.

So Josh?

Yes. Thanks, Kristen, for the question. As Neil mentioned, we've seen pretty meaningful growth in the newly diagnosed patients from Q1 all the way through Q4. That is really attributable to the strength of the clinical data of MIPLYFFA. And moreover, the real-world experience that [ HCPs ] are now having with their patients. It's also attributable to the efforts that we've put behind our disease awareness campaign, all driving greater diagnoses, as well as some of the collaborations that we have with genetic testing companies. As we mentioned in the remarks, we have recently expanded collaboration. And so we do expect that we'll see continued diagnosis in 2026.

Yes. Kristen, just to add on that. When we talk about the opportunity to have these efforts that Josh and the team are working through in identifying newly diagnosed patients, or [indiscernible] diagnosed patients quite frankly, this is a reinforcement to us that the market opportunity is somewhere between the 300 and 350 patients that are currently diagnosed, and the 900 patient prevalence. Getting these newly diagnosed patients early in the launch cycle versus a year or 2 years from now is really providing that confidence.

Got it. And just now that you're collecting more quarter-over-quarter data, do you have like an average sense or a range of the time it's taking for an initial suspicion to be confirmed or not confirmed to an actual diagnosis?

That's a really difficult question for us to answer because, as you know, in the rare disease space, as you work with one patient and the myriad of symptoms that a patient may have may be very different from one to the other. So we're continuing to lean in and double down on obviously educating physicians. Our new AI model that we're working on and the team is really starting to help us to get the education to the right places to ID new patients. And then as we're thinking about the disease state awareness and all the work that we're doing, it's a trifecta things that we're working on that are allowing us to be able to see this moving forward.

The one thing that I will say, and that Josh has mentioned previously, is that once we're identifying patients, we're able to utilize the depth of data that we have to really work with the physicians and the payers to get patients on and then to get patients reimbursed.

Kristen [indiscernible] as we mentioned in our prepared remarks, with this new collaboration with [indiscernible] the expectation is that from the time there is a test to results available, it's estimated that physicians would know those results within about 3 weeks.

Okay. And just a quick one, last one. So for [indiscernible], I'm doing my math correctly, you should be submitting responses in the next couple of weeks here. So after that, you would expect 2 months later to get the day 180 questions. Is that what's next?

So you're spot on and your math around our submission within the 90-day clock stop period. We're prepared. We will make that happen. The next one is the 150 day, not 180.

We'll move next to Jason Butler with Citizens.

Let me add my congrats to Justin joining the team. Can you talk about persistency? And are you at a point now where you can -- you have enough data to give some estimate of the proportion of patients that stay on therapy for a long period of time?

Jason, thanks for that question. As you know, persistency is really defined by the number of patients who are on drug for an entire 12-month period of time. And it's too early in our launch to be able to give meaningful data on persistency. I can tell you that we're very encouraged by the refill rates that we're seeing and the number of patients that are staying on therapy. Again, this really speaks to the durability of response that we're seeing both in our clinical data and the availability of new data that we're publishing to support that, as well as the real-world experience that clinicians are having with their patients.

Great. And then can you talk about the initial experience ex U.S. with the [indiscernible] collaboration? And how should we think about that as a potential growth driver in 2026?

Yes. We're really pleased with the adding of [ Unipar ]. And as you know, we have multiple distributors that are helping us with our global [indiscernible] The opportunity for us to execute that agreement and at the end of the year and in Q4 actually ship and get product to patients in a very short period is exactly what we want to do as an organization. That will continue. We've actually had additional orders in Q1 already, and that will continue.

But as I mentioned in the prepared remarks, you're going to see some variability in the ordering patterns, and the new rate of enrollment as we continue to build on that. Much like what we saw in our French EAP program, which continues to be approximately 30 patients, up or down 1 or 2 on a quarter-over-quarter basis. But it took a number of years to get to that steady state. So we're really pleased with that, and we expect that we're going to continue to engage broadening access for MIPLYFFA outside of Europe as well.

Okay. Great. And then just last question. You've talked a couple of quarters now about the number of -- the encouraging number of new diagnosed patients that you're seeing. Do you have an updated sense of what the total addressable number of patients could be in the U.S. relative to the [ 900 ] you've historically talked about?

Yes, I have to say that in the early phases of launch, it's hard for you to say what your end state is, but what I can tell you is that we're seeing -- and we saw it in Q2, a few new patients, we saw in Q3, and then we saw even more in Q4. That's giving us a lot of confidence that the market opportunity of those patients were diagnosed already, the 300 to 350, we can grow that somewhere between the 300 and 350 and 900. And it's because of this effort that Josh has been talking about on getting the reps to the place where they can be most successful, as well as all of the genetic testing and disease state awareness. It seems like -- and you see this sometimes in rare diseases, you don't really know what you have until you get a product in the market and then the rising tide starts to lift all boats. We're having more and more confidence on a quarter-over-quarter basis that the addressable market is somewhere between that 350 and 900.

We'll move next to Lachlan Hanbury-Brown with William Blair. [Operator Instructions] We'll move next to Eddie Hickman with Guggenheim.

Congrats on a great quarter. I wanted to follow up on the EAP revenue, which was quite impressive this quarter. Can you clarify if this represents a new recurring quarterly run rate that we should take into account? Or does it include any sort of onetime payments? And then how should we think about the growth of this revenue stream going forward and beyond?

Yes. Thanks, Eddie. So I want to -- this is part of why we really wanted to make sure that we put into the prepared remarks that these are initial shipments. And again, this is named [ patient base ] is reimbursed sales. So the shipments that are going out are for individual patients. So we've identified a number of patients prior to signing the agreement, and we continue to, on a named basis, so a one-by-one basis, actually get product into market [indiscernible] patients. So this is the start. And as I mentioned, that was Q4. We have more that we have continued to ship into Q1.

It's going to take some time, and there will be variability on a quarter-over-quarter basis. But our goal right now is to make sure that we're leaning into those patients who have been identified, to get them through the system on a one-by-one basis, and actually get product to those patients. Really impressed with what we've been able to do in getting the speed of product to patients so far.

Understood. I appreciate that clarification. Have you said any more detail about -- like are these in France only? Or are there other patients in other countries that you're starting to include in [indiscernible]

Yes. So maybe -- I don't provide guidance very often, but this is one where we've had very consistent guidance. Our French EAP program has approximately 30 patients, plus or mine one a quarter-over-quarter basis. And that has generated approximately $10 million net revenue per year. Anything on top of that right now is coming from additional markets that we're able to service. So what you're seeing on top of the quarter-over-quarter basis that we've said with the $10 million net a year, $2.5 million or so a quarter, is coming from the new territories.

Got it. And then one thing, I apologize if I missed this earlier, but how is the gross to net evolving in the U.S.? And is there a variability in that price we should be taking into account now as the launch evolves further?

Yes. Our gross to net, as you know, has evolved through the year, we had a one-off in Q3 based on the Inflation Reduction Act. Moving forward, I think we can't really guide you to gross to net [indiscernible] We've taken that into account -- and nothing has changed in our contract in our patient base so far moving forward. So in terms of the commercial and government [ air mix ], we feel like we're going to have a quarter-over-quarter change, and I can't really tell you what that is today. It's too dynamic.

We'll move next now to Sumant Kulkarni with Canaccord.

I have a few here. So first, on U.S. sales of MIPLYFFA. Do you expect to see continued sequential growth on that? Or could that be lumpy as well?

Yes, I'll answer that one. We see variability in the -- with high-value therapeutics and a number of patients in the ultra-rare space, one patient makes a difference on a quarter-over-quarter basis. So we expect variability moving forward for the duration of the launch, and the duration of supporting the patient base out there as we grow the market. And again, the addressable market, we're feeling a lot more confident between that [ 300, 350 and 900 ]. That means there's variability in our future.

Understood. And what's the average age of patients on MIPLYFFA? And what's the average age of newly diagnosed NPC patients? I'm asking because there could be the potential availability of [indiscernible] for infantile [indiscernible] NPC as soon as maybe this year, and how prepared is the company for competition from that front?

Yes. I'll ask Josh to talk about some of the specifics. But it's important to note that our patient mix remains very similar to what we saw in our expanded access program in the U.S. Of the 83 patients that we had when we closed the program was approximately 50% adults and 50% children. And that mix has continued throughout our launch in our 161 patients since launch, it's approximately 50-50 still.

I'll reiterate what we've also said, we actually believe that our physician base, our patient base really wants to have as many options available to them that's out there. And I'll ask Josh to talk a little bit more about what we've seen in the market, what our market research says. And we believe that it actually is helpful for all patients out there.

Yes. And just to reiterate what Neil said and was in our prepared remarks, about half of our MIPLYFFA today for patients over the age of '18, or '18 and over. Again, very reflective of what we saw in our expanded access program in the U.S.

In terms of new products coming into the market, we are very much aware of [ adrabetadex ], and we welcome all new treatments for patients. We have been hearing from clinicians and seen in practice that they are looking for multi-modal treatments and being able to use as many treatments as they possibly can for these patients. But we also hear from them that they view MIPLYFFA as really the foundational treatment in the preferred disease-modifying treatment for patients with NPC. So we're continuing to execute on as we can, and continuing to bring new patients to MIPLYFFA.

Yes. Sumant, bottom line, it's a win-win for patients. It increases awareness. Physicians want as many as they can, and we're supportive of continuing to rise the awareness of the NPC market.

Got it. And on arimoclomol pending EMA applications, you're on track to submit your 90-day response. Is it fair to assume that the agency has not as for any new clinical data? And on [ celeprilol ], what could you do to accelerate that program, given that if the product works really well, the time to event should actually go up?

Yes. Let me start with the [ MAA ]question. So as I mentioned in the prepared remarks, we submitted a robust data package. It's a standard view process where we got a 120-day list of questions, and we have not seen any new questions that we were not fully prepared to respond to in that clock stop. So we're going to continue to engage and continue to move forward.

But recall, what package we've put forth is the largest data set of NPC patients ever treated. So we're continuing to engage. I won't get into specifics of what questions are coming or not. There's no surprises that we have seen in the questions that we've seen so far. And you asked another question in regards to accelerating [indiscernible].

We talked about this before. I'm really pleased that we've been able to reinitiate the study. We have got 52 patients enrolled as of the year-end. But I'd like to see more. And part of that, we've always said our goal is to accelerate the development program. And that 52 patients is great, but we also wanted to engage the FDA in a Type C meeting to discuss our regulatory options. They were very responsive. We're going to engage further in the second half of this year as we continue to invest in and boosted enrollment, but in addition to that, look to ways to get the product to patients as soon as we possibly can.

We'll move next to Brandon Folkes with H.C. Wainright.

Congratulations on a very good quarter. Neil, maybe just first, obviously, there's a tremendous number of enrollments this quarter. If we look at the annual enrollment of 52 in 2025, how do we think about annual enrollment numbers going forward relative to 2025 as we balance an accelerating enrollment figure that we've actually seen quarterly recently, some of these initiatives you've put in, but also the natural launch trajectory here. So understanding this variability quarter-to-quarter, how do we think about the annual enrollments going forward?

Yes. Let me thank you for that question, by the way, because there have been a lot of questions in regards to what we see on a quarter-over-quarter basis. But 52 patients in our first full year post launch, we think, is a really great number moving forward for us. Now will that be -- I'm not going to guide you as to what we see in 2026 because, as I mentioned, we've got a lot of diagnosed patients to continue to go after. [indiscernible] make sure that we give MIPLYFFA as a potential opportunity for them. But the real, I think, confidence boost is that the efforts that we are making in the newly diagnosed patients, or misdiagnosed patients that are becoming newly diagnosed NPC patients, is giving us a lot of confidence on the long runway be able to get that 300 to 350 patients, and the 900 prevalent, getting it closer somewhere in there.

I'm not going to tell you what that number is because we don't know. But it is removing the barriers that we've seen out there specifically when it comes to diagnosing patients with our efforts around genetic testing as well.

Great. And maybe just one follow-up for me. Any way to contextualize what portion of diagnosed NPC patients in the U.S., you have engaged through your programs? And maybe, sort of, how we should think about how quickly those patients could convert?

Yes, I'm going to ask Josh to tackle that one. That's a tough one.

Yes. That is a bit of a tough one. But we've talked about that we believe that there are somewhere between 300 to 350 diagnosed patients. And we have, at the end of the fourth quarter, 161 patients enrolled to receive MIPLYFFA. The majority of those patients are previously diagnosed patients with the -- certainly the number of newly diagnosed patients increasing. So I think that puts us in a good position where we can comfortably say that we have somewhere between 40% and 50% of the diagnosed patients currently enrolled to receive MIPLYFFA.

We'll move next to Lachlan Hanbury-Brown with William Blair.

Congrats on a strong quarter, and apologies if this was asked earlier, my line cut out for a while during Q&A. But on the 24 new patient starts in Q4, was there anything sort of new that you'd start up late in Q3 in terms of the identification efforts that maybe contributed to that increase? Or is this just kind of a great example of the lumpiness of quarter-to-quarter new ads where it sort of leads that you initiated in prior quarters coming to fruition?

Yes, Lachlan, thank you for the question. I'm glad that you're able to get on the call. There's no single book here in regards to what we did in 1 quarter. I've mentioned this previously, and this is a consistent effort that we've got to be able to continue to put forth to be able to identify earlier patients, to get them [ diagnosis is ] earlier and then support them through the process. I'll ask Josh to talk a little bit about some of the things that we've done. But it is really -- it's a quality of effort here that is driving this type of performance.

Yes. As Neil mentioned, I think it certainly is a combination of multiple things, no single magic bullet here. Some of it is by virtue of a full year on the market and giving clinicians enough opportunity to gain some clinical experience. Also enough opportunity for us to build the disease awareness, both through our campaigns as well as our publication strategies. And some of it was our enhanced targeting and commercial execution, where we created a very bespoke AI-driven targeting model. But we looked at known NPC patients and identified their symptoms, understood their journey of clinicians with whom they got diagnosed and received some treatment. And then we were able to find patients that looked like those patients in claims data and EMR data that had not yet been diagnosed to -- send our reps in a much more targeted way to clinicians' offices to help them identify these patients.

Yes. Josh talked about this a little bit in a previous question, but the centers of excellence that we really focus on, and then the community physicians, there is a back and forth that happens between those. That is what we are working with in terms of the next generation of patients and continuing to drive. That breadth, though, of physicians who are prescribing and ultimately supporting patients is widening. So as we think about the future, the breadth will continue as we get new patients. This is an ultra-rare disease. A lot of times, a physician may only see 1 NPC patient in their entire lives, their entire careers. And we want to make sure that we continue to expand that breadth with all the tools that we're working with today.

Great. And Neil, maybe another question for you. Just -- it's a different topic. But you've obviously got a very strong balance sheet and you are more or less sort of cash flow -- pretty close to cash flow breakeven [indiscernible] by the looks of things. So how are you thinking about capital allocation in terms of you may be approaching that where, as you said in the past, you've proven your ability to launch MIPLYFFA. So maybe you can start looking at if there are opportunities to add other things to the pipeline or the portfolio?

Yes. So we look at capital allocation on a quarterly basis. And I think it's important for us to reiterate that we have the cash to operate independently of the capital markets with $238.9 million in cash at the end of the year. It's important for us because we need to execute on what's in front of us right now. We have a number of opportunities we're executing against. The U.S. market and the launch and continued investment into ensuring that we broaden access to as many patients as we can in the U.S. We're talking about geographic expansion right now when we think about Europe and the continued patients that are coming into the EAP, but also the MAA and beyond Europe as we're looking at that.

And then again, we're also investing in our pipeline in [indiscernible]. So as we continue to invest and allocate our capital to the highest payoff activities that we can, we're well on our way to building a leading rare disease company. But we do evaluate it on a quarterly basis.

At this time, we have reached our allotted time for questions. I'll now turn the call back over to Neil McFarlan for closing comments.

Thank you, Leo. I want to appreciate the team here today for [indiscernible] effort in the year last year and the team that we have, that's supporting MIPLYFFA and all of our development programs, and we look forward to updating you on future calls. Thank you very much.

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.

Good morning, everyone. My name is Susmita Roy. I'm an associate on the JPMorgan Healthcare Investment Banking team. On behalf of JPMorgan, thank you all for attending the Healthcare Conference. And I'm thrilled and honored to introduce Zevra Therapeutics for their company presentation today.

A little bit about Zevra before we get started. Zevra Therapeutics is a commercial-stage company with a unique opportunity to impact people living with rare diseases. The company is in growth mode, with operating runway to execute on its commercial and development priorities.

Zevra is commercializing its lead product, MIPLYFFA, in the U.S. for Niemann-Pick disease type C. In addition, the company has an MAA under review by the EMA, is broadening access through geographic expansion opportunities and has a pipeline of rare disease programs.

Zevra has multiple revenue sources with net revenue generation of $72.3 million in the first 9 months of last year and exited Q3 with a strong cash position of $230.4 million.

We are delighted to welcome their President and CEO, Neil McFarlane, to the podium to tell us more. Thank you, Neil. And I'll turn it over to him. Please note that Q&A will be afterwards. And if you have a question in the audience, please wait for the mic. Thank you so much.

Thank you, Susmita, and thank you, JPMorgan, for allowing us the opportunity to present this week. I realize it's been a long week. And for all of you in the room, thank you for coming here on the last day of the conference.

At Zevra, we're taking a rare approach to taking care of patients. As you see on the screen here today, this is an adult patient with Niemann-Pick disease type C. And I'm looking forward to spending some time with you today talking to you about Niemann-Pick and what we're doing here at Zevra to help patients.

I'm going to be making some forward-looking statements today. So please take a look at our most recent SEC filings to get the most up-to-date information.

Moving forward, we're a purpose-driven company. I think it's important that I spend a little time on this slide. We have a mission to redefine what is possible in bringing life-changing therapies to people living with rare diseases. And we've got a vision to become a leading rare disease therapeutics company.

Our values, as you see on this slide, start with patient centricity. It's what we do when we make decisions. It's what we do when think about how to service the rare disease community. We're accountable to each other, we're accountable to the community and we're accountable to our shareholders as we move forward. Integrity. Innovation. Courage. All of these values drive how we operate as an organization.

We also have a very unique opportunity to impact people living with rare diseases. We're a commercial-stage company with a late-stage pipeline. We've got geographic expansion underway with an MAA, as well as an existing infrastructure that we can leverage to actually launch the products that we have and earn the right to go and bring new products in and be a partner of choice.

As Susmita mentioned, we have multiple revenue sources. And through the first 9 months of 2025, $72.3 million. We exited Q3 2025 with a strong cash position of $230.4 million. And we are in growth mode.

Spending a little bit of time on the portfolio, we have a diversified portfolio. As I mentioned previously, MIPLYFFA with Niemann-Pick disease type C. Importantly, we have that product that was FDA-approved in September of 2024. At that time, and today, we actually guide that we have orphan drug exclusivity, the regulatory pathway to keep exclusivity through 2031. I'll talk a little bit more about some recent evolution of our patent term extension application and what we are hopeful there's the potential to extend to 2031 and beyond.

The other product we have is OLPRUVA, and it's for urea cycle disorders, approved in 2022 with intellectual property through 2036. AZSTARYS is a product that we developed internally and licensed and have a partnership where we receive royalties and milestones through 2037. And as I mentioned, arimoclomol, which is the name of MIPLYFFA, for Niemann-Pick C is under review by the MAA.

Celiprolol is our Phase III program that is ongoing right now, with IP through 2038. And we've done some work in Phase II for sleep disorders with KP1077 in idiopathic hypersomnia and narcolepsy that allows for a Phase III-ready trial with very durable IP. So this diversified portfolio is fulfilling our mission, and it's just getting started to bring life-changing therapies to people with rare diseases.

We're doing this through intense focus on the things that we can drive and on the impact that we can make as an organization. The team has been executing very well. And we're innovating not only in bringing new therapeutics to market, but we're actually also innovating in how we bring therapeutics to patients ultimately.

We're prioritizing our resources and been incredibly disciplined with how we've moved forward through 4 pillars. Our marketed therapies that are in growth mode for MIPLYFFA, as I mentioned, as our lead product. A pipeline that's got synergistic growth opportunities with us to be able to leverage our infrastructure, but also develop and get approved products through the regulatory and clinical development periods.

And then we've got an experienced team. We call it talent and culture, and I think this is one of the most important parts of any company. And our experienced team that's continuing to grow and lean in to deliver for patients, I'm very proud of. And we're doing all that with a corporate foundation that's got a very strong balance sheet and the fiscal discipline to make investments that are paying off for us through focus, and not trying to, I like to say, we're not trying to boil the ocean, we're not trying to invest in everything. We're investing in things that can drive real value and sustainable growth for the company.

Let me talk a little bit about 2025, because it's really paved the way. It's a transformational year, no doubt, for us in 2025. But it's paved the way for some multiple growth drivers as we look into 2026. Our team has really established MIPLYFFA as a foundational therapy in NPC. In the first 12 months post launch, we've achieved approximately 40% of the diagnosed patient population. I've been in the rare disease space for a long time, and I've been incredibly impressed with the launch here. I've never seen 40% of the diagnosed patient population in 12 months. So we're really making an impact, and I think it's a testament to the team.

In our pipeline, we've got some significant growth opportunities. As I mentioned, we have our MAA that is under review by the European authorities. We announced that 120-day response would be coming at the end of 2025. We received that response. While I can't talk about all of what's in the response, what I can say is that we've seen no new questions or concerns that have come up from the response that we've got. And our team is incredibly well prepared for all of what we received. Very similar to the pathway that we've taken with the FDA to get the product approved. And I'll talk a little bit more about what we've done and the data and the sustainability of the data that we've put forth for the application.

Importantly, we actually have been able to have 92 expanded access patients in Europe and the U.K. in a very small number of markets. And that number is growing on a quarter-over-quarter basis. Very proud of the opportunity to be able to serve patients that expand access to MIPLYFFA in Europe. But this is also a testament to our ability to be able to expand the markets and get the product to patients.

The other perspective here is that we have our pivotal Phase III trial undergoing for celiprolol under the growth opportunities. I'll talk a little bit more about that when I get into the pipeline and innovation section.

And we're demonstrating leadership through advocacy, through community engagement, through patient and caregiver support services as we develop ourselves as a partner of choice in the rare disease products. And recently, over the last 6 to 9 months, we've actually established a presence in Boston, one of the leading biotech rare disease hubs, and it's allowing us to be able to tap into that ecosystem and then drive additional value for the company.

And as I mentioned, a very strong balance sheet. In 2025, we were able to monetize our pediatric Priority Review Voucher for $150 million that added nondilutive capital to our balance sheet. And as I mentioned, $230 million at the end of Q3, where we have not burned capital for a few quarters. So we're very proud of what we're doing in terms of setting the foundation for the future.

Let me spend a minute on our marketed therapies here, and I'm going to primarily focus on Niemann-Pick disease type C, which is an ultra-rare genetic disorder that leads to premature mortality. NPC, as I mentioned, devastating, fatal lysosomal storage disease. And what really transpires here is you get cholesterol buildup in the lysosome in cells that lead to cell death.

NPC is a gene that the mutations produce abnormal protein and it doesn't allow for the cholesterol to be -- go in and out of the lysosome. And this progressive buildup then leads to cell death. That cell death is in organs. It causes organ dysfunctions. It causes spleen, liver and brain buildup and then cell death.

The signs and symptoms of NPC are very heterogeneous. The age of onset is -- well, you have it forever. It's genetic. However, the symptoms can show up either as a child or as an adult. What we've seen so far in our experience, both in our expanded access program and in the 137 patient enrollments that we've had, is about a 50-50 split between children and adults, which a lot of times people see this as just a childhood disease. And as we are moving forward in our launch and engaging with patients with NPC, we're realizing that we're diagnosing patients who are adults for the first time or adults who have been misdiagnosed as well.

So the rate of progression is obviously different if you have these symptoms of cognition, speech, ambulation, swallowing, fine motor skills and the visceral, primarily hepatosplenomegaly. But these presentation of symptoms are challenging, like in every rare disease. It presents a diagnostic odyssey for a lot of physicians to be able to try and find out what is transpiring.

And for us, we've demonstrated through multiple trials the impact that MIPLYFFA has on NPC. And it's proven effectiveness now both as a short term in terms of improvement -- and what you see in this slide here is MIPLYFFA that's utilized in combination with miglustat. On the bottom, you see the Niemann-Pick C clinical severity scale of MIPLYFFA and miglustat, where you see no progression through 12 months and improvement as early as 12 weeks. I think that's an important perspective. You get it early and then you have the durability.

On the right-hand side, you see the same revised 4-domain Niemann-Pick C clinical severity scale, over a period of 48 months through our open-label extension study. And you see that limited progression and the halting of this devastating disease, durable through 5 years. This is, for us, a real game changer, when you can bring the -- with, by the way, I should say, a very acceptable safety profile, importantly. This allows us, though, to be able to establish MIPLYFFA as foundational therapy for NPC. The mechanism of action and up-regulation of the CLEAR gene network allows for the impact that we see on the halting of the progression of the disease, the strength of our clinical data, the magnitude of our safety database.

And we're continuing to expand the body of evidence as we're moving forward now through both our expanded access program that we had in the United States, our pediatric investigational study and substudy, which is complete, as well as our open-label extension going through 5 years. The magnitude of this data, it's -- and what we've submitted actually to the MAA is really the largest collection of data for NPC patients ever. And we're proud of the ability to be able to get that into the committee.

We're also driving patient access. Very proud of the organization right now. In payer coverage, about 66% of covered lives as of the end of Q3, which is very on target for us in terms of the first 12 months of launch. Patients are gaining coverage even when not on formulary through the medical exception pathway. And through the end of Q3, we're still having a very high adherence through our enrollments, and very proud of that. We'll get our first glimpse at the end of Q1, into Q2 on persistency, patients that have been on product for 12 months. And I'm looking forward to being able to continue to drive that.

This next section is an important section for us. We have been increasing the identification of patients. As I mentioned, approximately 40% of the diagnosed patient population, which equals 137 patient enrollments. We had 8 patient enrollments in Q3.

Importantly though, some of the work that we've been doing around the identification of disease state awareness, offering genetic testing and some of the collaborations have started to unlock newly-diagnosed patients. We saw some of this in Q2, very early in our launch. In Q3, we talked about it also on our earnings call that the identification of newly-diagnosed patients is really key for us to be able to establish ourselves, not with the diagnosed patients we have today, but the TAM being between that 300 to 350 patients that we know are diagnosed and being treated today and the 900 prevalent patient population. In Q3, we saw more. And in Q4, we've seen even more newly-diagnosed patients, which is giving me a lot of confidence that the TAM is somewhere between this 300 to 350 and the 900, which the team has been working through bringing these patients on to product.

The other part is we have a bespoke AI predictive modeling that our commercial team has been working through to find potentially new patients that have either been misdiagnosed or a suspicion index that allows for patients to walk through the process to get potentially diagnosed with NPC earlier. These tactics are starting to pay off and I think is giving me a lot of confidence that this market and the TAM is somewhere between that 300 to 900 patients. We're early, but I'm confident that we're making headway there.

The team [ fielded some ] market research at the end of the year that suggested that MIPLYFFA is a preferred NPC therapy trusted by most clinicians and showed improvement in balance, swallowing, cognition, speech and reducing falling. I think these are some of the feedback that we're getting. In the Q&A., maybe we can spend a little bit more time. We'll get Josh up, our Chief Commercial Officer, to work through that.

What's next for us is capturing this next wave of growth for MIPLYFFA. As I mentioned, the U.S. prevalence of approximately 900 patients in the U.S. living with NPC, and this 300 to 350 that are diagnosed and treated. This ability for us to continue to drive penetration of the undiagnosed patient population is what I think has given us a lot of confidence that the market is larger than what we originally see, the patients that are diagnosed.

In addition to that, the European prevalence is about 1,100 individuals living with NPC, but the diagnosis rates are much higher than that of the U.S. because they've had miglustat approved for well over a decade and have been able to educate and drive patients to therapy. We see both the European prevalence and the high number of patients who have been treated and the last 3 quarters of continued growth of newly-diagnosed patients putting us in a place where we feel like we can unlock the opportunity in the United States.

And then our expanded access programs with the geographic expansion that we've most recently discussed with -- if we look at the umbrella of the geographic expansion, it's both through our marketing authorization application in Europe and potentially the U.K. It's through the fact that we've got our EAP, Expanded Access Program, that is driving patients today and physicians are asking for the product through name patient basis.

And in addition to that, outside of Europe and the U.S., we just recently signed a distribution agreement with a partner in Europe that -- with a partner in Europe to support markets outside of Europe, outside of the U.K. And we're actually now shipping product to those patients under named patient basis. So broadening access to MIPLYFFA is the next wave of growth, the EU prevalence and getting into EU with an MAA, and continued driving of our U.S. launch is capturing that wave of growth for us.

I'm going to quickly run through some of our pipeline. I'm going to focus here just on vascular Ehlers-Danlos Syndrome, which as I mentioned is a severe autosomal dominant genetic connective tissue disorder. And it's the most severe form of Ehlers-Danlos Syndrome as a subtype. As I mentioned, it's a connective tissue disorder caused by COL3A1 gene mutations. It leads to a defect in type 3 collagen in both the vessel walls as well as hollow organs. And it's usually characterized by arterial aneurysms or hollow organ ruptures.

In the United States, about 7,500 individuals diagnosed with VEDS and that are genetic -- about 95% of the diagnoses are usually confirmed by the COL3A1 genetic testing. As I mentioned, devastating disease. Most -- or 25% of patients will experience an event, either the aneurysms or hollow organ ruptures by the age of 20. And about 90% of patients will experience an event by the age of 40. Unfortunately, arterial rupture is the most common cause of sudden death in these patients, with a median survival age of 51 years.

Celiprolol is a product that we're developing as a potential treatment for COL3A1 VEDS. And it's addressing VEDS by reducing the mechanical stress on collagen fibers as a selective adrenergic modulator. We actually have quite a bit of data that has been developed with celiprolol's use outside of the U.S. through a randomized and real-world data across the BBEST study in a few European cohorts. And what we saw is that the event rates, an arterial and/or hollow organ event, is decreased versus the untreated patients, and improved survival.

Now we're running a DiSCOVER trial, which is a Phase III trial. It's a decentralized pivotal trial being run under a special protocol assessment with the FDA, of which, as of the end of Q3, we've enrolled 44 of the 150 patients. As I mentioned before, it is an event-driven trial. And the interim analysis is that 28 events, of which if we hit the positive marker there, we will then move towards a -- sorry, then we don't have to move towards the final event rates, which is 46 events.

The one important part here around celiprolol is it is a -- although it has never been approved for VEDS anywhere in the world, it is the primary treatment for VEDS in Europe where the product was approved many decades ago.

Let me -- I know I'm running a little short on time here, let me quickly talk about the fact and how we're demonstrating financial discipline. As I mentioned, through the end of Q3, we have had net revenues of $26.1 million in Q3. And the large chunk of this is through our efforts in the U.S. with MIPLYFFA.

We do get pre-commercial revenues through our EAP program in France right now. That will be expanded with the execution of our distribution agreement that we announced over the holidays, that will bring us new revenue that are pre-commercial revenues in select territories. We get the license royalties from the AZSTARYS agreement and OLPRUVA.

Importantly though, our balance sheet is incredibly strong with $230.4 million, with just $61 million of debt.

In closing here, our growth drivers in 2026 and beyond are meaningful. The additional -- the continued execution of our U.S. launch unlocks the opportunity for us. As I mentioned, our conviction internally around the opportunity size for MIPLYFFA through our genetic testing collaborations and through the early undiagnosed patients that are being identified and put on therapy today has given us a lot of confidence in that. We're driving the additional clinical and scientific evidence that will allow for us to be able to further develop the foundation of therapy for us.

We have this possible patent term extension, which we believe is an unlocking event and a re-rating event for the stock. Every additional potential year that the IP would be extended beyond our current LOE with the orphan drug exclusivity is meaningful. And we believe that that should come in 2026, the final determination from the U.S. Patent Office.

And then we submitted our MAA and as part of our geographic expansion and continue to drive EAP patients in Europe and physicians with the experience in taking care of patients through named patient basis, but also broadening that outside of Europe and the U.S. I've mentioned this a few times on our earnings calls, our go-to-market strategy in Europe is still under evaluation. And we believe that as we continue to drive expanded access patients and utilization by key opinion leaders in Europe, we'll be primed to be able to make a decision whether we go it alone or we partner it. But today, we'll continue to drive the expansion through EAP where we can service patients.

And with that, I'm going to stop and see if we can open it up for some Q&A. Thank you for your time.

I'm going to also ask Josh Schafer to join us on stage, who's our Chief Commercial Officer.

Amazing. Thank you so much for the presentation. And first of all, congratulations on the success of MIPLYFFA.

To kind of kick things off for the Q&A, and if there's any questions, obviously, we can continue to take that from the audience, but what does the team attribute the success for MIPLYFFA today? And what do you all think will be the specific drivers for that continued success moving into 2026 and beyond?

What I'll do is I'll maybe start with the broad company, and I attribute the success that we've had so far to the focus that we put in to driving the things that we can control and execution that we've had and the way that we're innovating to be able to get product to patients. But maybe I'll ask Josh to talk a little bit about all the wonderful things we're doing on the commercial side.

Yes. Neil presented a slide which I think really speaks to what's going on with MIPLYFFA and how we've been able to drive that performance. Most notably is the clinical differentiation that we've been able to establish through publications, through building the awareness of that data. The commercial and medical teams have done a great job around building awareness of the disease and the need to treat. And our collaboration with genetic testing organizations has really driven the identification of formerly undiagnosed patients, which all led to really outstanding performance in '25.

Awesome. Now just to kind of continue on that and double-click on it, throughout the presentation, you mentioned that the awareness of the disease, diagnosis, the under and misdiagnosis of the disease that we've seen, outside of -- maybe through genetic testing, just double-clicking on it, could you just speak a bit more about how improvements could be made in diagnosis and how MIPLYFFA can get to those who obviously need it? And your thoughts on how -- and who you're working with. That would be great to hear a bit more detail about.

Yes. Well, first of all, this is a really small patient population, which prior to our approval in 2024, there were no approved treatments for Niemann-Pick. So many clinicians outside of those experts are just really unaware of it. Many of them read -- there was one line in med school that they read about it.

So we have really taken on the charge of helping to build the awareness, helping to identify these patients through a symptomatic presentation. And then if a patient presents with a certain array of symptoms, which were listed in terms of swallowing, speech, ambulation issues, these patients might likely benefit from a confirmatory genetic test. And we've partnered with a couple of companies who provide that test, and that's really helping to drive the awareness and identification of those patients.

Josh, maybe you can spend a little bit of time educating me and everybody else on the bespoke kind of AI work that we're doing with EMRs and claims data to be able to try and help people who otherwise may have not been diagnosed or been misdiagnosed previously, that now have the opportunity to get diagnosed.

Yes. This is a really, really cool work that I take no credit for, the team is doing its way above my complete understanding. But what we've done is we've gone through the claims data and we've identified patients who have been confirmed or who have been diagnosed with NPC. And we've looked at the array of symptoms that they have. We've looked at their treatment odyssey and misdiagnoses and clinicians that they've seen and a number of other parameters. And we've then looked at the larger claims database of patients who have those same symptoms, same travel through the health care system that were never diagnosed with NPC. And there is a surprisingly large number of patients who look like an NPC patient.

And that allows us as a sales team, as a commercial team to interact with those clinicians to say, "Hey, if you have a patient who looks like this, you might think about NPC as a disease that your patient might have. And we have a treatment option and we offer a way for you to confirm that diagnosis." So it's really -- and it's an evolving, of course, with all AI things, it's an evolving model. But as we get new information, it helps to inform how we go forward.

That's incredible. Kind of along those lines, as you've been talking to physicians and trying to educate them and also interacting with patients and payers, et cetera, what's been the feedback on MIPLYFFA? And how has that been? How -- what have you guys kind of been doing with that feedback? And would love to just hear a bit more details about that.

Yes. The research that was fielded at the end of last year is providing us with a lot of very actionable, timely feedback that allows us to be able to pivot. But Josh, maybe you can talk a little bit of some of the specifics because it's pretty good data. And it's important, it is anonymized. The work that we have done is not Zevra going out doing research. This is work that's been done in the community without us knowing that it's Zevra.

Yes. We went out to payers, patients and clinicians and did some independent market research and went through a list of questions around their view of the disease state and MIPLYFFA specifically. And the feedback was overwhelmingly positive, in which clinicians stated that they really view MIPLYFFA as the foundational treatment and the best option for patients in terms of halting the progression of the disease.

And caregivers went so far as to say, when their children or loved ones were on MIPLYFFA, it gave them confidence and they saw improvements in balance and ambulation, and really resounding feedback. But it also gave us the opportunity to understand our messages, what's working, what's not. And we're refining that according to that feedback.

No, that's amazing. And of course, always helpful to get that feedback. I think one of the things that stood out to me in the presentation was with -- given the success of MIPLYFFA, there's also so much more that the company is looking ahead to this year and beyond. Could you just maybe double-click on the MAA and EMA processes that are going on? And would love to hear a bit more about, I know you mentioned some time line, but any further details that could be helpful for everyone to know would be awesome.

Yes. So I'll take that one. So as I mentioned previously, we submitted our MAA at the end of July and through a standard MAA approach. But as a new application, I think that's an important perspective. We didn't actually refer back to our previously submitted application in our conversations with the regulators. It was really important that because of the magnitude of new data that we were going to be providing in the application around our pediatric substudy -- and for those of you that are listening, usually, you have a pediatric investigational plan that you do after your filing. We fortunately have the ability to have finalized that pediatric investigational plan as well as the study.

So that data has been submitted. Our longer-term OLE, the open-label extension data going out 5 years, we were able to submit as well. Along with we collect the data for a number of years in the United States through our expanded access program, about -- and then as we got approval, we were able to shut that program down and then convert those patients to commercial product. But we shut the database down at the end of March 2025. And as we locked that database, we're able to bring all that data into the filing as well. So the substance behind our application is much more robust than even what we had for the FDA.

Now moving forward, the European approaches is that you file and then they come to you with a 120-day list of questions, and they're clarifying questions. I can't talk about the questions or the process, but we're in the clock stop period today. And what I can say is that our team was prepared for every question that's come back, and there have been no surprises, which gives me a lot of confidence that now that we can actually provide the responses and further inform the dossier for our next step, which will be at the 150-day mark after this clock stop is over and we respond.

So I can't tell you what a specific timing is at this point. What I can tell you though is that we are in a position today of providing the most robust set of data ever collected at NPC to help drive and get expanded access to patients in Europe.

Awesome. It's very exciting to hear. And I mean, I think given that you talked a lot about how this will help you all expand into the EU a bit more. But beyond the U.S. and the EU, are there any thoughts that you might have on like overall global strategy and getting access to NPC patients worldwide?

Yes, our goal -- thank you for that question. Our goal is to maximize the impact we make for patients with NPC everywhere in the world. We are in a very interesting time when I think about the geopolitical space, the opportunities that we have to be able to expand access across the world. We are taking a very disciplined approach where we will provide compassionate use where we can, we will provide named patient basis access that may or may not have a pre-commercial revenue with it.

But we're also being very disciplined to protect our optionality to expand into Europe and not impact our pricing and not impact Most Favored Nation discussions that are ongoing, which I'm not sure anybody knows what it is today. But for us, we're taking a disciplined approach. How do we expand the product to as many patients as we can and provide access? But also being very disciplined not to shoot ourselves in the foot.

So today, through this distribution agreement that we signed over the holidays, it allows us to be able to service select territories. And for the first territory that we're going into under this agreement, we're actually able to have name patient basis reimbursement at U.S. WAC pricing. And this territory is outside of Europe, outside of the U.K., outside of the U.S. So that discipline of the identified patients that we've been working on with these distributors -- or this distributor for a number of months allows us now to service those patients but not impact our opportunity in Europe. Or the rest of the world for that matter.

Yes. No. And given the circumstances, it's always -- this is a tough path for companies to navigate, and the distribution agreement seems like it's a great step in the direction of figuring it out. I think for the next part of kind of talking more about the vision and next steps in the future, could you just, kind of going back a bit, could you just double-click a bit more on MIPLYFFA? You mentioned the potential patent protection, extending that. Are there any key updates that we should know about or the team is looking forward to present soon? And what are your thoughts about that?

This is a very big opportunity for us, and I mentioned it just in passing that it's a re-rating opportunity for this company. MIPLYFFA is clearly the key asset that we have in our organization today, with the largest amount of growth that we see, not just today in 2026, but in the next, today, 7 years that we have the orphan drug exclusivity.

As I mentioned, orphan drug exclusivity is through the regulatory pathways of the FDA. They provide you this through the orphan drug legislation. On top of that though, there's patent term extension. And today, we have an Orange Book listed patent that expires in 2029. So our orphan drug exclusivity is actually longer than that of our patent.

As part of our approval process, we can go back to the Patent Office and actually ask for an extension of our patent based on the amount of time it took for the product to get approved and through that process. We've worked with the FDA to identify the number of days that we're eligible for. We've now -- the FDA has now gone back to the Patent Office and said, "This we agree with the company," the sponsor in this case of those days. And the Patent Office opened up an open public commentary for about 60 days. That 60 days expired at the end of November, and without any comments.

So we expect the Patent Office to now rule as to how long we will get from our 2029 patent. The maximum is 5 years. We don't know what that will look like in terms of what the ultimate outcome is. But every month, every quarter, every year on top of what we have today of 2031, could go out to 2034, is a very meaningful impact to the company. So that's why we think, in 2026, this is a re-rating event for the company, and not yet being recognized in our stock.

No, that's definitely, yes, the impact of that will definitely be something we'll all be keeping our eye out for. Other than the milestones that were mentioned briefly in the presentation, are there any upcoming catalysts that investors or the audience should be aware of before we wrap up in a few minutes?

Yes. The keys to our sustainable growth over the next 5 years plus is execution on our U.S. opportunity. And as I mentioned, we have a lot more confidence that we are getting to the undiagnosed patient population this early in our launch, giving us a lot of confidence that this market opportunity is somewhere between 300 and 900. We just don't -- it's too early in our launch to know exactly what that looks like, but this has given us a lot of confidence.

The other one is in regards to our geographic expansion. We know that there are a large number of patients in Europe, as I mentioned, 1,100 patients from a prevalence perspective, and the majority of them have been on miglustat and where our product is used in combination with miglustat. So we see that geographic expansion as an important next step, while we also tap into the ex Europe, ex U.K., ex U.S. named patient basis opportunities to expand access for patients there as well.

And lastly, it's really about the Patent Office too. What we see there and what they come back with will be a great opportunity for us in terms of immediate re-rating. And it gives us time to go out and make sure we're impacting more patients that we can. Any month or a year that we can continue to invest in getting patients diagnosed and treated with a product like MIPLYFFA, it's going to be great for us all.

Awesome. Just quick last question. To address those goals and visions that you've just outlined, what would you consider the team's strongest tools to kind of impact that? You have a strong balance sheet, we've been seeing that. But anything else at the -- that you could consider an asset or tool that the company is leveraging to make sure those visions can come true?

Yes. We've spent a lot of time over the last 12 months as part of our strategic planning process refining the first strategic plan we had in 2024. And part of that has been around how are we going to show up as an organization. It started with the mission and vision and values that I discussed. And we're trying to redefine how to impact rare disease and how to get product to patients. I think that that focus that we have throughout our entire organization and the people that we've brought together, and we'll continue to build the culture of running through walls for patients and executing, is what's going to make us one of the most successful rare disease companies in the future.

Amazing. Those are all my questions. Any closing remarks from the team or anything else?

Thank you for being here. What a crowd. Appreciate you on a Thursday.

Good afternoon, and thank you for joining Zevra's Third Quarter 2025 Financial Results and Corporate Update Conference Call. Today's call is being recorded and will be available via the Investor Relations section of the company's website later today.

The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications. Please go ahead.

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our third quarter financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investors section of our website.

Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied in any forward-looking statements due to risks and uncertainties associated with Zevra's business.

Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and other filings with the SEC.

I'm pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; LaDuane Clifton, our Chief Financial Officer; and Josh Schafer, our Chief Commercial Officer. Our Chief Medical Officer, Adrian Quartel, will also be available today for the question-and-answer session.

Now, it's my pleasure to hand the call over to Neil.

Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. Zevra is building on a foundation for success, guided by our strategic priorities and fueled by the strong performance of MIPLYFFA, which is delivering meaningful benefits to patients with Niemann-Pick disease type C or NPC. This impact is translating into revenue generation and leading us towards operating stability that enables us to invest strategically to build a leading rare disease company.

MIPLYFFA is a foundational advancement in the management of NPC and the first treatment approved in the United States for this indication. NPC is a progressive and fatal disorder caused by mutations in the NPC1 or NPC2 gene, which disrupts intracellular lipid transportation. This leads to cholesterol and lipid buildup in cells, causing widespread dysfunction. NPC affects both children and adults with symptoms, including cognitive decline, speech and swallowing difficulties, motor impairments and premature mortality.

NPC is considered to be an ultra-rare disease with an estimated 900 individuals affected in the United States. And of those, only 300 to 350 have been diagnosed. We're excited to deliver on the promise of strong scientific data, which demonstrates that MIPLYFFA in combination with miglustat is the only disease-modifying therapy that halts the progression of NPC at 12 months and demonstrated a rapid onset of effect within 12 weeks of treatment initiation. Long-term data showed that MIPLYFFA was both safe and effective in halting the progression of the disease for more than 5 years. This compelling data is resonating with the community and driving continued strong performance.

I'm pleased to share that our third quarter net revenue reached $26.1 million, driven by $22.4 million in net revenue from MIPLYFFA. Additionally, there were 8 new prescription enrollment forms received in the quarter, bringing the total to 137 since MIPLYFFA became commercially available just a year ago. Building on the success of our launch, our commercialization strategy is focused on identifying diagnosed patients who may benefit from MIPLYFFA and on implementing targeted initiatives to improve detection and diagnosis for the many people who remain unaware they are living with this life-threatening disease. We have seen traction in both of these cohorts, and Josh will further elaborate later in the call.

A significant near-term growth milestone is to expand into the European market, where we estimate approximately 1,100 people are living with NPC. In Europe, there is an established precedent for treatment with miglustat. And with the existing clinical network, there is a favorable environment for a foundational treatment such as MIPLYFFA. As a reminder, we filed the Marketing Authorization Application or MAA at the end of July. That application has been validated and is under review by the European Medicines Agency. We are highly confident in our submission, which incorporates an expanded data set beyond what served as the basis for the FDA filing and U.S.A. approval. As a reminder, our filing submission included data on more than 270 NPC patients that have been treated with MIPLYFFA through the pivotal trial, open-label extension study, expanded access programs and pediatric substudy.

Our go-to-market strategy for Europe is under active development and will inform our decision to launch independently or through a strategic collaboration. Additionally, our Expanded Access Program in Europe and the U.K. is playing a critical role in building product experience and fostering strong relationships with physicians and clinics with 92 patients enrolled at the end of the third quarter. These engagements not only support awareness and education, but also lay the groundwork for a potential commercial launch. We've established a solid patent position for MIPLYFFA, and we've also requested a patent term extension with the U.S. Patent and Trademark Office, which could provide coverage beyond the term granted through our orphan drug exclusivity.

For OLPRUVA, there was one new prescription enrollment form in Q3 and covered lives reached 81%. Notwithstanding our best commercial efforts, OLPRUVA’s clinical differentiation has not penetrated a mature and well-served UCD market. As a data-driven organization committed to making judicious investments that drive meaningful benefit for patients, we have made the decision to scale back our sales and marketing efforts for OLPRUVA. While we evaluate the options for OLPRUVA, we will maintain market access and product availability for patients and continue providing support through our patient services program, AmplifyAssist. Going forward, we will provide updates if there's anything material to report.

Turning to our development pipeline. We are actively recruiting patients in the DiSCOVER trial, our ongoing Phase III trial to evaluate Celiprolol as a treatment for Vascular Ehlers-Danlos Syndrome, or VEDS. VEDS is a rare inherited disorder caused by mutations in the COL3A1 gene, which impairs Type 3 collagen production, essential for vessel and organ strength, leading to fragile tissues that put patients at high risk of ruptures in the arteries, intestines and uterus. In Q3, we enrolled an additional 5 patients in the DiSCOVER trial, bringing the total to 44 of 150 patients required for full enrollment.

Our efforts are focused on bolstering our relationships with clinics involved in the management of these patients' care while also expanding the network of genetic testing centers collaborating with us to identify individuals carrying the gene mutation. In parallel, we are conducting educational outreach to vascular specialists who frequently encounter these patients following rupture-related surgical interventions. As an event-driven study under a special protocol assessment with the FDA, we have an interim analysis built in after 28 qualifying events, and there has been one confirmed event through the end of Q3.

In summary, we continue to have strong momentum in Q3 and are excited about the many future growth opportunities for MIPLYFFA, including continued performance in the U.S., potential for patent term extension, significant upside potential through geographic expansion and the late-stage development asset with Celiprolol.

I'll now turn the call over to Josh for a deeper dive into our commercial progress with MIPLYFFA.

Thank you, Neil, and good afternoon. Physicians have expressed enthusiasm and interest in MIPLYFFA as the foundational treatment for NPC. As Neil mentioned, in the third quarter, we received 8 new prescription enrollment forms. This quarter marks the 1-year anniversary of the launch. And as we reflect on our progress, we are pleased with the overall trends. We have received enrollment forms from the leading NPC centers of excellence that our rare disease specialists have been targeting, demonstrating the impact of our field teams.

In addition, our sales efforts have expanded beyond the centers of excellence into community practices where many NPC patients are treated after initial diagnosis and treatment recommendations. Our sales and medical teams have been informing and educating clinicians about our differentiated mechanism of action, demonstrated clinical benefit as well as the recently published long-term data from our open-label extension trial. These compelling data provide real-world evidence that resonates deeply with physicians and patients alike.

We recently conducted independent market research with clinicians and NPC patients, and I'm pleased to share that MIPLYFFA was recognized as the preferred disease-modifying therapy for NPC. Clinicians reported confidence in prescribing MIPLYFFA for long-term use based on its efficacy and tolerability and patients and caregivers described noticing improvements in balance and swallowing, fewer falls and changes in cognition and speech.

We continue to focus on raising awareness of not only MIPLYFFA, but also of NPC to support the diagnosis and treatment of new patients. Our early investments in disease awareness initiatives, including our Learn NPC, Read Between the Signs campaign and our genetic testing resources are identifying patients who were previously undiagnosed. Through this testing program, physicians can order a genetic test for individuals suspected of having NPC. Anecdotally, we are hearing of physicians previously unfamiliar with NPC who have identified suspected patients, confirmed diagnosis through our resources and are now prescribing MIPLYFFA.

We have built bespoke AI-driven models to analyze electronic medical records and claims data to identify both formally diagnosed but untreated patients and those who have not yet been -- who have not yet received a diagnosis. These examples highlight the tangible impact of increased disease awareness, and they underscore the importance of continued education and outreach in ensuring patients receive timely diagnosis and treatment.

In the third quarter, we also maintained an impressive presence at local, regional and national scientific conferences and patient events. These included an oral presentation at the International Niemann-Pick Disease Alliance face-to-face meeting, several poster presentations at the Child Neurology Society Annual Meeting and 4 poster presentations at the International Congress of Inborn Errors of Metabolism. The presentations included new data demonstrating that patients who were randomized to receive miglustat only in our pivotal trial and then added MIPLYFFA in the open-label extension phase experienced a decline in annual disease progression. We continue to generate, publish and present peer-reviewed data to reinforce MIPLYFFA's use as a foundational treatment for NPC.

Finally, growing market access via reimbursement coverage remains top of mind for us. And in the third quarter, we increased the percent of covered lives to 66%, which is in line with what we would expect 1 year into the launch. We're achieving high overall reimbursement rates for MIPLYFFA, either through direct formulary coverage or through medical exception pathways. We seek to provide access to as many patients as possible. And to date, there has been broad utilization of our co-pay assistance and patient services.

Importantly, MIPLYFFA patients are showing continued adherence and staying on treatment. Patients conveniently receive their monthly refills through our AmplifyAssist program, which aims to deliver a first-class experience for patients and has helped to establish Zevra as a preferred partner within the NPC community. In short, our strong third quarter performance reflects MIPLYFFA’s differentiation and our focused commercial execution, and we are encouraged by the sustained momentum heading into 2026.

Now I will turn the call over to LaDuane.

Thank you, Josh, and good afternoon, everyone. In addition to the financial details included in today's call, we encourage you to refer to Zevra's quarterly report on Form 10-Q for more detailed information, which we intend to file later today.

In the third quarter of 2025, we reported net revenue of $26.1 million, which includes $22.4 million from MIPLYFFA, $2.4 million in net reimbursements from the French EAP for arimoclomol, $1.2 million from royalties and other reimbursements under the AZSTARYS license and $100,000 from OLPRUVA.

For our commercial products, we recognize revenue when shipments are received by the specialty pharmacy. Q3 MIPLYFFA net revenue was impacted by the redesign of Medicare Part D rebates, which led to recognition of a gross to net true-up from prior quarters of $1.2 million. The updated rates will become an ongoing component of gross to net estimates going forward.

Cost of product revenue for the third quarter, excluding noncash intangible asset amortization, was $1.2 million. Operating expense for the third quarter was $20.4 million, which was a decrease of $6.8 million compared to the same quarter a year ago.

R&D expense was $3.4 million for Q3 2025, which was a decrease of $7.5 million compared to Q3 2024, primarily due to reduced third-party costs following the completion of the KP1077 Phase II trial, combined with a decrease in personnel-related costs.

SG&A expense was approximately $16.9 million for Q3 2025, which was an increase of $700,000 compared to Q3 2024 due to additional investments in our commercial, medical and launch activities. Looking ahead to the impact of scaling back on promotional activities for OLPRUVA, these resources are expected to be reallocated to MIPLYFFA and to our patient services activities. Therefore, a meaningful change in selling expenses is not anticipated.

Net loss for Q3 2025 was $500,000, which led to loss per basic and diluted share of $0.01. This includes noncash stock compensation expense of $2.8 million, noncash fair value adjustment related to warrant and CVR liabilities of $5.5 million during Q3 and noncash intangible amortization expense of $300,000. For the same quarter in 2024, we reported a net loss of $33.2 million or $0.69 per share.

As of September 30, 2025, total cash, cash equivalents and investments were $230.4 million, which was an increase of $12.7 million compared to the end of Q2 2025. The increase in cash included $6.6 million in nonoperating cash received in exchange for the exercise of warrants and options during Q3 and $2.3 million in cash from interest income. Total debt was approximately [ $61 million ]. Based on the company's current operating forecast, we believe available financial resources are sufficient to execute on our strategic priorities independent from the capital markets.

While we continue to anticipate variability in our working capital requirements, which is largely dependent on the timing of receivables and payables, our disciplined approach to operating investments and capital allocation has significantly strengthened our financial position. Our commitment to maintaining cost efficiency and rigorous operational accountability has cultivated the flexibility and capacity to strategically invest in high-impact opportunities such as the MIPLYFFA U.S. launch and the Phase III study of Celiprolol while also preparing for the potential launch of MIPLYFFA in Europe. We are well positioned to pursue the opportunities we have to drive long-term value creation for our stakeholders and to accelerate our growth initiatives with confidence.

Now, I will turn the call back to Neil for his closing remarks.

Thank you, LaDuane. We're pleased by the continued success of MIPLYFFA and deeply encouraged by the support from both patients and physicians. Looking ahead, we believe we have a significant growth opportunity on the horizon with the potential approval and commercialization in Europe. We're committed to aligning our capital allocation with our strategic plan and to invest in opportunities that meaningfully impact patients while maintaining a sharp focus on creating sustainable growth. Thank you.

We'll now open the call for questions. Operator?

[Operator Instructions] Our first question will come from Kristen Kluska with Cantor.

The first one I have is, as we think about the last 2 quarters, there's been 15 new start forms. Curious if any of these have been patients that are newly diagnosed through perhaps some of your efforts? And also, as we think about the next few quarters up ahead, how you're thinking about the potential of finding additional patients that currently aren't diagnosed today?

Kristen, thanks for the questions. I'll get us kicked off a little bit here. You're absolutely right. The last couple of quarters, we've had about 13 new start forms and really pleased with where the team has been continuing to move forward. And actually, as I'm thinking about this, I think it's not 13, but 15, we had 8 and 7. So I just want to make sure we clarify that on the call. The reality, though, is that our team continues to move forward with the investments that we're moving. I'll ask Josh to talk a little bit more around the newly diagnosed components of where we are.

Kristen, thanks for the question. And yes, we're really excited not only with the performance we're seeing in terms of the number of enrollments coming in. But as you noted, many of them are coming in as newly diagnosed patients, and that number has increased from last quarter to this quarter. And that is largely through the efforts that we've articulated in the prepared remarks. But specifically, these are coming from the disease awareness campaign and our partnership with the genetic testing collaborators to be able to identify these new patients and make us aware of these newly diagnosed patients so then we're able to go into those offices and work with the physicians and clinicians to get them onto MIPLYFFA.

We've also done a lot in terms of helping to build disease awareness through our medical teams and building the evidence through conferences and publications. And we've also done quite a bit in terms of using advanced analytics and machine learning to be able to look into electronic medical records and claims data to find these undiagnosed patients. And so we are seeing a lot of that success.

And Kristen, let me follow up on that just a little bit. When we think about our 1 year in the market, which we're celebrating here right now, the total number is 137 prescription enrollment forms. That's approximately 40% of the diagnosed patient population that we've actually seen. And that's really a testament not just to the team and what they're doing, but also the strength of the long-term data that we've been able to execute on educating physicians and getting that awareness out there with patients and the like. So really pleased with where we are 12 months into the launch.

Okay. Appreciate that. And then when I think about your balance sheet, it's very robust. And this quarter, you were pretty much on the edge of breaking even. So how are you thinking about perhaps in the near to midterm, how you might take advantage of your robust balance sheet?

Yes. Thanks, Kristen. So we go through an annual cycle of planning for strategic session with our Board. And part of that, we thought -- we talk about capital allocation and understanding where is it that we can best be positioned to be able to move forward. I'll ask LaDuane to touch base on it, but this is all about us leaning in, executing on what we have, doing the best we can for both the commercial products as well as the development programs and then earning the right to do something else later on. LaDuane?

Yes. Thank you, Neil. And Kristen, as we execute against the MIPLYFFA launch, we certainly see that our operating results are beginning to reflect that success. And so we're appreciative and thankful for how that's coming through. Although I will tell you, although we did generate cash -- some cash this quarter, there remains some variability in our results as we continue to have dynamic working capital requirements between accounts receivable timing and accounts payable timing. So we haven't turned the corner yet, but definitely, things are pointing in the right direction.

And as Neil said, we are very much guided by our strategic plan. And I think executing right now on what's in front of us, the MIPLYFFA launch in the U.S., the expansion and potential approval in Europe, other ex-U.S. opportunities that we're exploring and then maintaining and continuing to support Celiprolol. These are the opportunities we have in front of us. And as we execute on those, we can continue to apply discipline and think about how we deploy capital across all the opportunities that may present themselves.

Our next question will come from Sami Corwin with William Blair.

Congrats on the progress. I guess since the majority of patients that are submitting the new enrollment forms or newly diagnosed patients, how are you kind of thinking about reaching the additional 200 or so patients that have already been identified? I guess when I'm kind of thinking about strategy, that kind of seems like it'd be low-hanging fruit. So how are you thinking about that? And then given you're a year into the launch, what percentage of patients are you seeing that are getting refills?

Great. So Sami, I want to make sure that I clarify. We didn't say the majority of the new patients that are coming in or hope we didn't say that the majority of the new patients that are coming into prescription forms are undiagnosed patients that are newly diagnosed patients. There are a component of them that are, but majority would be an overstatement. We have actually -- this is not the first quarter we've gotten new patients, though. We've actually had new patients in our last quarter as well. So that -- I just want to clarify that.

In regards to how we're thinking about the additional patients that are out there, I'll actually ask Josh to hit on that. And then 1 year in terms of refills, I'll ask Josh to hit on that as well.

Yes. So Neil, thank you for clarifying that majority comment. I was going to make the same. The majority of patients who are getting -- submitting enrollment forms are, in fact, patients who have previously been diagnosed and either on a current treatment or have been diagnosed and not received treatment. And we're continuing to find more and more patients like that every day. As we've noted on prior calls, we know where the majority of these patients are, and we continue to go into those offices and work with the clinicians to get those patients on to MIPLYFFA. In addition, we are very pleased that there are undiagnosed or previously undiagnosed patients who are now coming into the enrollment or now receiving enrollment forms as well.

With regards to the percent of refills, I would note that we are really, really pleased that the majority of patients are actually who are coming in, we're able to find a pathway to getting reimbursement. We have 66% of covered lives today. And even those who are not covered through formulary, we're able to get paid coverage through a medical exception pathway. And almost all of those patients are getting refills on a month-to-month basis.

Got it. And one more, if I may. What is the average age or weight of the patients currently on therapy? And has that kind of differed with the -- or differed compared to the patients who are on the Expanded Access Program versus those that have come on to drug after?

Yes. So I'll take that one, Sami. So when -- if you recall, our clinical trial program was primarily skewed towards children. Our Expanded Access Program actually brought in more adults, and it was about a 50-50 mix between children and adults. As we now actually get into the real world, the real world experience has continued to actually allow us to actually have that [ 50-50 percent ] split in regards to kids and adults.

In regards to the weight, what we did when we actually announced our pricing for the program is we used an average weight that was based on our 83 Expanded Access Program patients that were in there as well. So we don't provide the guidance specific to weight, but I do believe that it's still at a 50-50 today.

Our next question will come from Eddie Hickman with Guggenheim Securities.

Congrats on the progress this quarter. Just a few for me. It's really nice impressive increase in coverage, I think, growing from 52% to 66%. So what have been the key drivers to see that much increase this quarter? And how fast and how high do you anticipate that coverage level going? And then when you -- now that you have a year under your belt, can you talk about how the patient enrollment form split is between the centers of excellence and the non-centers of excellence?

Yes. I'll ask Josh to take both of those. It’s right up his alley.

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Yes. So we're really pleased with the progress that we're making in terms of the percent of covered lives. I think it really speaks to the strength of the data and the fact that MIPLYFFA is the only disease-modifying treatment out there with the data that shows that it halts the progression of disease at 12 months. And that data has been really compelling to payers.

We're continuing to see and anticipate that, that coverage will increase. And we feel like we are right where we should be 12 months into launch, and we're going to continue to work to increase that coverage and to ensure as many patients as possible can receive MIPLYFFA.

We're also really pleased that we've been able to target and receive enrollment forms from almost all of the centers of excellence that our sales representatives or rare disease specialists are targeting. But more importantly, what we're seeing now is that the new enrollments coming in are coming from community-based physicians as well. And the dynamic is maybe a patient will go to one of these centers of excellence, get an initial diagnosis and a referral and then go back to his or her local GP or internal medicine specialists who is then managing the care going forward.

So what we're seeing is a real breadth and increase in the overall number of prescribers as well, and we're really pleased with that.

Appreciate that color. And then one final one, if I may. For Europe, when do we expect the next update on that? And when just thinking about maybe just the French EAP, like should we assume a similar conversion as the U.S. EAP once that's approved there? Or help us think about the dynamic of how that EAP will factor into paid drug if you got approved there?

So Eddie, we are in the valid -- as we talked about in the prepared remarks, we are right now in the post-validation phase, 120-day clock that will allow us to be able to get our first inclination as to where we are. But I think it is important to remind everybody that we are coming off of the back of an FDA approval and an AdCom and a positive vote and a very solid launch into the U.S. market. It's the largest, most compelling data package in NPC. We've added, as we talked about in our prepared remarks, the additional data on top of what we submitted to the FDA. So we feel really good and confident about the MAA submission. Once we get that 120-day information, it will provide us with an opportunity to know a lot more. I don't know when we will then come back to you and the Street to understand what that looks like because it's a list of questions. It's not an approval. For us to be able to understand how to be able to then talk about that more broadly, I think it will be more closer to the 150 days. So give us some time on that and then see if we come through.

You talked about the French EAP conversion. We do feel really good about the opportunity, but it will be based on reimbursement on the conversion rates within Europe. Once we get the product approved, each country will go country by country around how we reimburse -- navigate reimbursement within those countries. And those countries that come quickest will convert fastest. And today, as I mentioned on the call, we have 92 patients now in our -- it's really, we call it our global EAP. It's primarily Europe now. And those patients are a very small number of countries. So we feel like we've got the ability to continue to drive patients in the EAP -- well, not drive, fulfill patients in the EAP that we'll be able to convert them at the same rate once we get reimbursement in the countries.

Our next question will come from Sumant Kulkarni with Canaccord.

On MIPLYFFA in the U.S., we appreciate the 40% share that you achieved pretty quickly, but do you think we are at steady state with respect to addition of patient enrollment forms for a quarter? And what are the key variables that might cause a step change in that number, especially given that the sales force will only have MIPLYFFA to focus on given the scale back on OLPRUVA?

Yes. So we've seen 13 in the first quarter, 7 in the second quarter, 8 now in this third quarter. I think it's way too early for us to call this a steady state. We are seeing the results of our efforts to find new patients and to diagnose new patients. And as mentioned, several of the new enrollments that we got in the quarter were from newly diagnosed patients. So I expect that we're going to continue to see more of that taking place and the efforts of our sales team to continue to identify currently diagnosed patients, to also find these undiagnosed patients. I think all of that is really telling a good story looking forward.

I'd also mention that we completed recently some market research, which we completed with payers, clinicians as well as patients. And clinicians stated that they felt very confident in prescribing MIPLYFFA for the long term based on its safety and its efficacy, and they recognized it as really the preferred disease-modifying therapy for NPC. And patients stated that they saw median improvements in their balance, their swallowing, fewer falls and change in cognition. And I think all of that really speaks well for what future quarters might bring.

Got it. On MIPLYFFA in Europe, what are your initial thoughts on what price might be like relative to the U.S. or the EAP in France? And finally, there's been -- I guess, the sleep market is waking up with buyouts orexin data. What are your latest thoughts on KP1077 and the strategic options there?

Yes. Let me take a couple of those. In regards to Europe -- in terms of European pricing, Sumant, we've done in parallel to our marketing authorization application, a lot of work in regards to understanding the market dynamics in Europe, what the pricing potential is in Europe. As you just stated around our EAP in France, we do get pre-commercial revenue from that, which has also been part of the calculation. We won't know based on -- until we understand what the label looks like, what a final price in Europe is.

What we do really appreciate around Europe is that of the 1,100 patients from a prevalence perspective, there are the majority of those patients with miglustat being approved for well over a decade that have actually been diagnosed and have been on some type of a therapy, that provides us with an opportunity to be able to impact the European market at a faster rate than that of what we even have seen in the U.S. because the diagnosis rates in the U.S. have been lower than that of in Europe. So that's something that, for us, we believe the penetration will be good. Will it be the same price as the U.S.? That's to be determined at this point.

Got it.

You also asked another question, Sumant, around 1077. Yes. No. So to comment on 1077, we remain open to strategic alternatives. We are having conversations, but I can't really comment more on that right now.

[Operator Instructions] Our next question will come from Brandon Folkes with H.C. Wainwright.

Congratulations on the quarter. Maybe just following on from a few questions before. How are you thinking about additional commercial investments behind MIPLYFFA in the U.S. in 2026 versus the current commercial infrastructure? And given you're seeing some success, right, in the disease awareness campaign driving previously undiagnosed, right? I think it's the success to have any of those patients kind of being enrolled at this stage. But how are you weighing up maybe potential additional investments behind disease awareness and increased diagnosis in 2026?

So Brandon, I'm going to ask Josh to comment on some of the specifics around this. But if you recall, we have been investing now into a commercial infrastructure to be able to not only invest in one product, but in multiple products in these centers of excellence and providing value to these centers that we can then provide the education, the patient services, the medical support, the clinical data that's necessary to be able to pull products through and provide patients with the best experience that they can once they get prescriptions. So I think that our investment is rightsized to be able to move forward and execute on this product. And as we have mentioned in the past, getting OLPRUVA up to speed too in terms of the awareness and clinical differentiation. Josh, do you want to talk a little bit about 2026?

Yes, sure. And just to kind of underscore what Neil said, our top priority from a commercial and medical standpoint is to identify patients who can benefit from MIPLYFFA. So there really is no single tactic that we're talking about. It is a cross-functional across the entire organization. This is a top priority for us. And so we continue to invest in new publication strategies and new data generation, continuing to look at ways that we can identify patients through claims data and electronic health records. So we're continuing to evaluate that, and our budget is really based on that as a priority. We've been doing that in 2025. We'll continue to do that in '26 and onward. So I don't see any market change in our investments to do that. We're going to continue to evaluate the -- what works, what doesn't work and pivot as we need to because it really is an absolute priority for us.

Our next question will come from Jonathan Aschoff with ROTH Capital.

Congrats on the quarter. And I was just curious, what's the rough cost you expect to have to sell out to launch in the EU? And is there anything noteworthy to say about MIPLYFFA inventory levels end of the second quarter versus end of the third?

Yes. Thanks, Jonathan. I'll take that in regards to the European opportunity. We've laid out a number of different approaches in terms of the research we've done, the pricing we've done, the approaches we may take. We've been having capabilities presentations from local distributors to some type of a hybrid approach that we might take or to just put it in the hands of folks who can actually deliver for patients better than we could.

I mentioned previously that we had 92 patients already in Europe in our Expanded Access Program and continuing to grow that on a quarter-over-quarter basis. Our goal is to be able to either execute on the opportunity in Europe ourselves or put it in the hands of somebody who can do it better, [Technical Difficulty] shareholders as well. What that cost is, is to really depend upon the approach that we take. So I can't give you a number today. But what I can tell you is that we've done all the work that's necessary for either going it alone or put it in the hands of another party that can service patients better and with better economics as well as the ramp that it would take to be able to get MIPLYFFA in the hands of patients in Europe. So stay tuned on what that might look like as we get through our labeling discussions and the MAA.

I'll ask LaDuane to talk about your inventory question on a quarter-over-quarter basis.

Yes. Thank you. The MIPLYFFA inventory, we monitor carefully, and we utilize internal metrics like days on hand to make sure that what's in the channel is consistent and is sufficient to service patients. So there is no large fluctuations quarter-to-quarter since we use those metrics to guide us and make sure that, that's not driving any kind of trends.

One other detail I want to make sure is clear, though. When we think about revenue, we also have the impact of our gross to net. And while we don't normally give the details of that, we did note that there was a true-up adjustment from the recent changes redesigned to the Medicare Part D rebates. And that did have some impact as we noted. About $1.2 million was recognized in Q3, therefore, reducing net sales by that amount, which was from prior periods.

So just calling out that there's 2 dynamics there to be clear. Inventory in the channel is consistent at the levels appropriate that we've maintained throughout the launch period. And then this gross to net had a unique impact in Q3, and we've adjusted our rates based on that to go forward from there.

Lastly, Neil, just remind me how many of those 92 patients are French or in France?

Yes. Approximately 30 patients is what has been consistent over a number of years in the French EAP program. It's remained the same.

This concludes the Q&A portion of today's call. I'll now turn it back over to Neil for closing remarks.

Thank you all for joining us today. Sorry, operator, are you still there? It looks like we have one more person in the queue.

We'll next go to Jason Butler with Citizens.

I guess just wondering if you could comment qualitatively on the number of patients that have received -- have an enrollment form but haven't received reimbursed drug yet. And I guess what I'm asking is directionally quarter-over-quarter, has that remained stable? Or are you seeing that number decrease or any comment there? And then also the time from receiving an enrollment form to getting reimbursed drug. Is that now stable? Or is that changing at all?

Yes. We are still in the active phase of launch, Jason, but it's a really good question that I'll ask Josh to hone in on. The pull-through of patients to paid drug, I am incredibly impressed with the team. And then also in regards to as we get new enrollments in and then are able to move through that reimbursement process, team is doing an outstanding job. So Josh?

Yes. Jason, thanks for that question. And again, it is something that we're incredibly proud of what we're seeing. The number of patients who are able to receive a paid drug is exceptionally high. We do have patients who are going through some period of free drug as we're investigating their benefits. They might go on for a 30-day period. Those patients are oftentimes unable to get into paid patients. So at any given time, we might have some patients in the free goods program who then become paid in the following month. So it's a little bit difficult to give you any precise numbers there. But what I can tell you is the vast majority of them do end up getting paid.

And we are seeing -- your second question was around the time from an enrollment coming in to then a paid dispense. And we have seen that time shrunk dramatically since launch. And I think that's due to, again, the execution of our team. It's also due to the payers really beginning to appreciate the strength of MIPLYFFA data, and the benefit that it brings to patients. And so that time has really gone from what was, in some cases, months, now down to weeks. And we have, in fact, seen some patients get turned around in 24 hours. And so we're really pleased with that.

Got it. And then just a quick follow-up for LaDuane on the gross to net point. Is this reestimation or true-up something that we should expect to happen on a seasonal basis, an annual basis? Or just how should we think about the stability of the gross to net moving forward?

Yes. So this specific adjustment came from the redesign of the Medicare Part D, which came out of recent sort of administration changes, Inflation Reduction Act, et cetera. And so this one is sort of, I think, behind us. But -- and let's be clear, this was a change in the rebate in terms of catastrophic coverage. So MIPLYFFA being one of the higher price as a rare disease drug, it does get through to catastrophic coverage under Medicare sooner. So 2 changes. They made that threshold to get to catastrophic sooner, and they also increased that rebate from 9% up to 20%. So that's a change now that's in the system, and we've adjusted our estimates going forward.

But the other detail about gross to net and one of the reasons we don't always dig into the details, except for in this case where it had a meaningful impact in this quarter is there could be other adjustments to gross to net going forward. Payer mix is something that early days in the launch. We know where we are today, but that could shift. We could continue to see changes in other dynamics within that. And so gross to net changes probably are something we will live with as a commercial company. But for right now, I think this specific item is now trued up and would be in the computations.

I am showing no further questions at this time.

Thank you, operator, and thanks, everybody, for joining us today. We look forward to discussing our year-end and Q4 data with you in the coming year.

Thank you. This brings us to the end of today's meeting. We appreciate your time and patience. You may now disconnect. Thank you.