Catalyst Pharmaceuticals, Inc. Aktie

Okay. Good afternoon, everyone. Thank you for taking the time to join us here. For our next presentation, we're excited to have Catalyst Pharmaceuticals. And joining me from the company to my left is Rich Daly, who's the Chief Executive Officer of the company. For those of you who don't know me, I'm Glen Santangelo. I'm one of the Life Sciences Analysts at Barclays. I cover spec pharma and animal health. We do not cover Catalyst Pharmaceuticals, but it's been interesting to learn a lot about it in the past several weeks. So really looking forward to the presentation. So thank you, Rich, for joining us. Very glad to have you here.

Thanks, Glen.

Maybe a good place for us to start for those who don't know the company, maybe we should just spend a minute or 2 talking about your 3 primary commercial products are in different stages of their life cycle. Maybe just sort of give a high-level overview of the company for a minute or 2, and then we can sort of dive right in.

No problem. So Catalyst Pharmaceuticals is a company focused on rare diseases. And in our rare disease portfolio, it's actually ultra-rare. So we have a muscle activating drug called FIRDAPSE which works in Lambert-Eaton myasthenic syndrome. And then we also have a drug for Duchenne Muscular Dystrophy. It's a steroid, we believe a differentiated steroid called the AGAMREE. And FIRDAPSE has been on the market for about 8 years and consistently grown 15% to 20% every year. So it's actually defined the law of decreasing increases over time, which is just a tremendous story.

And then in March of '24, we launched the AGAMREE for Duchenne's and I love the opportunity to talk about this in a little bit, but it looks like it's a differentiated steroid and has a really interesting clinical profile and some data just came out at MDA this week in Orlando that further supports that out of Europe. So we're really excited about it.

And finally, we have FYCOMPA, which is a drug for epilepsy. We bought that drug from Eisai a couple of years back, and the drug has performed really well, but we experienced generic incursion in about midyear, but the drug has continued to perform really, really well, although it is declining in revenue as expected.

Okay. Excellent. All right. So maybe why don't we start with some recent results given that we just kind of came off fourth quarter earnings. I mean, 2025, you wrapped up a great year, 20% revenue growth, I think you sort of forecasting even faster growth in 2026, and we'll get to that. We'll dissect the pieces of that in a minute, but maybe you just want to give us a quick overview, 2025 and the momentum you had into 4Q. And maybe was there anything looking back on 2025 that maybe surprised you, maybe either positively or negatively relative to maybe what you would have thought as the year sort of progressed?

Sure, sure. So we entered the year with a lot of expectation for continued success for the company and for the brand, and we saw that. It was a really interesting year. So as I said, FIRDAPSE continues to grow 15% to 20%. It's a very solid performer. So we're really excited about that. And we implemented a couple of things, Glen, in the year on the specialty pharmacy side. So as you know, in the rare disease space, we use specialty pharmacies, which actually helps patients get on the drug sooner, get on the right dose, titrate as appropriate and then remain on it. And we did that in June, 2 things in June and September. And we're seeing the results of that. So patients' desire or leaving the brand has declined. So the performance of the brand has propped up on that. That's on the FIRDAPSE side.

And we also initiated a number of programs for cancer-associated LEMS. So LEMS is LEMS, but some of it's not associated with cancer and some of it is. The non-cancer associated is called idiopathic and then there's cancer-associated LEMS. We work with the National Lab to get testing up on a panel. So there's a very simple test called the VGCC test, Voltage-Gated Calcium Channel test, and that's now on National Labs. And we also worked with the NCCN, National Comprehensive Cancer Network to actually get FIRDAPSE into the guidelines for cancer-associated LEMS. And now we're working with the GPOs and the GPOs control -- there are 3 GPOs that control about 80% to 90% of all community-based oncology practices, and they follow pretty strictly the NCCN guidelines. We're really excited about that.

On the AGAMREE side, AGAMREE has launched really, really well, and it's sourcing patients from both the generic prednisone that's on the market and EMFLAZA or generic EMFLAZA. About 45%, 45%, 10%, the remaining 10% come from naive patients. The one thing that occurred and the brand weathered this really, really well last year was some of the challenges, the safety challenges on ELEVIDYS. So we knew when ELEVIDYS got its label expanded in the middle of the year that there would be some challenges because patients needed immunosuppressive dose. And so that created sort of a line where there was a delay in patients getting converted to from prednisone or ELEVIDYS -- I'm sorry, prednisone or EMFLAZA to AGAMREE, but the brand weathered that really, really well and came out of the year in a very strong position.

And just as I said FYCOMPA experienced generic incursion. But because of some of the work that our Chief Commercial Officer, Jeff Del Carmen did in keeping promotion on the brand all the way through the end of the year, the brand continues to perform well, although it is declining as expected.

All right. So why don't we dig into each of these products individually? Why don't we start with FIRDAPSE. Very strong guidance for 2026. Your $435 million to $450 million in revenues implies 21% to 26% growth, obviously, a big number. You started to touch on some of those key drivers. But maybe could you help us think about sort of the idiopathic LEMS versus maybe the market share gains you hope to assess on the cancer side, which you were just sort of speaking about.

On the idiopathic side, the #1 misdiagnosis is generalized myasthenia gravis. And one of the things we're really excited about for the patients is that all of this effort on DTC for generalized myasthenia gravis, we believe, will benefit us down the line. So as these patients come in, they'll be diagnosed with gMG. A really interesting fact here is that the average physician sees one LEMS patient in their entire career. So it's that rare. So when they see a LEMS patient, they automatically think muscle weakness and the extremities and all that must be gMG. So they'll put them on a gMG therapy. And with all this awareness going on, we think it's going to drive more patients in to be diagnosed.

Most of them will be misdiagnosed with gMG, but then those therapies likely won't work. And so they'll be looking for the next thing because actually accelerate they're coming to FIRDAPSE. The average diagnostic journey for LEMS patient is between 4 and 6 years. So you can accelerate that, you can get the patient on. And these patients on the idiopathic side live a normal lifespan. So they're diagnosed typically in their 40s, 50s, but then they'll live a normal lifespan.

On the oncology side, the patients are generally -- LEMS is generally associated with small cell lung cancer. And we know that there is no really -- no good outcome there. The average patient without LEMS lives about 9 months if they have small cell lung cancer. However, the average patient with LEMS appears to live about twice as long. It has nothing to do with the product per se, but LEMS is an early indicator of small cell lung cancer. So a patient that gets LEMS generally gets diagnosed earlier in the disease process and thus, they live longer.

So the way this works out, in any typical year, there are about 150 idiopathic patients that could be diagnosed, and there are 900 that could be diagnosed on the cancer side. And so working with the large GPOs creates a really great opportunity to help serve those patients. So here's the takeaway. As successful as the brand has been, our penetration on the idiopathic side is about 30% and the penetration on the cancer-associated LEMS for FIRDAPSE is less than 10. So there's tremendous upside in this for the patients and obviously, for the company as well.

But you made the case that the new NCCN, the Comprehensive Cancer Network guidelines may have an impact in terms of how these patients are diagnosed and treated. Do you think that will ultimately increase sort of or lessen the impact from the increased diagnoses, if you will?

Yes. We do think that. So on the idiopathic side, and we have to keep pointing to these -- we think of this as 1 molecule, but 2 brands or 2 distinct markets because they're treated in different areas. So when we think about the opportunity, Oncology Care, I spent 15 years in Oncology. Oncology care has become very, very complex. NCCN guidelines become very important because of the complexity. And so there are care pathways in each of these GPO organizations that say they want to follow the NCCN guidelines. That's always such a focus for us. So we think that can drive behavior amongst the oncologists, yes.

And are you changing sort of your firm's commercial behavior in terms of education and promotion and doing different things to sort of drive these guidelines home and increase the diagnosis rate?

So the idiopathic side is very much a prospecting market. There's high concentration in the oncology side. So we work with the GPOs. They have education programs. They have other opportunities to help the physicians really get in line with the guidelines because these are the preeminent guidelines that are out there, and they want to follow them. So yes, we've changed it. We actually have dedicated -- a very limited dedicated force in the field to work with the GPOs at the very highest levels of the GPOs and really drive that education, drive that adoption of the care pathways.

Can we talk about the intellectual property on this product? I think you had some sort of settlement recently with Teva that may be -- that locked in 2035.

February 2035.

With that agreement with them, but I still think there's another litigant out there, Hetero.

There were 3 first filers. We settled with 2 of the first 3. We settled with Lupin and we settled with Teva. Teva was the first one to settle. And we see Teva, as many people do, is probably among the most sophisticated generic producers in the world. And so we feel really good about that settlement. We have still one remaining, Hetero. Our trial was scheduled for March 23 to start. We thought it would be about a 5-day trial. However, the basic understanding we have of constitutional law is that criminal trials will supersede any other trial and it appears the judge may have a conflict. I want to really emphasize that, may have a conflict with our trial dates because he has a criminal case, he has to hear as well.

However, criminal cases can have an opportunity to settle, obviously, if there's some sort of arrangement with the defendant and the state. So there may be an opportunity to stay on track. We're still tracking that. It's actually in our public docket that...

I'm sorry, when was the Teva settlement? And when are the scheduled court dates for Hetero?

The Teva settlement was in January of last year.

January, okay.

And Lupin settled about midyear. And then we're still -- obviously, we're open to conversations with our colleagues at Hetero. But if we have to go to trial, we certainly will go and defend our intellectual property. We're very confident. And I think the settlements support that confidence.

Okay. Maybe can we shift gears to AGAMREE? 68% growth in the fourth quarter. And I think you've been pretty impressed with kind of what you've been able to accomplish to date. When you look at the guidance of $140 million to $150 million, that obviously implies a more modest 20% to 28% growth, maybe not surprised given the law of large numbers. But maybe could you talk about some of the underlying drivers of that growth in the back half of the year and help us assess the durability of those elevated trends in 2026?

So it's a great question, Glen. So when we think about the LEMS market and the lack of concentration, the DMD market, the Duchenne's market is the polar opposite. There are about 100 centers that treat 90-plus percent of the DMD patients. You can imagine, early diagnosis, it's very clear what the patient has. 45 of those centers actually have 80% of the treatment through the use of steroids. And if you use a steroid, patient who uses a steroid early in diagnosis, their average ability to walk increases by 2 to 2.5 years. Steroids is foundation therapy. So it's the thing you're going to start before you start anything else. And then you're going to keep that drug on board because of the extension of the ambulation.

So the drivers of this are, again, commercial team under Jeff Del Carmen has done a great job of really penetrating those centers of excellence. We have 100% of the top 45 and about 90 or so percent of the top 100 have used the drug. And now we've gone wide. Now we need to go deep. And so we're using some of the opportunity to talk about the differentiation with the potential differentiation. Again, it's not in our label, but what we saw this week at Muscular Dystrophy Association, we saw the critical issues for a steroid are behavior. Patients on steroids have behavioral issues. They have stunted growth if they're on long term. Their bones are generally weaker. They have higher fractures, they have cataracts and they have cardiovascular disease. Eventually, DMD patients succumb to cardiovascular disease in general. The data that came out this week is very interesting. It was a study, the GUARDIAN study versus standard steroids -- standard of care steroids, and it showed that there are potentially some benefits to AGAMREE that...

Using it as a first-line treatment instead of the corticosteroids.

Well, it is a corticosteroid, just to be really clear. But using it as -- how does it compare on fractures, how does it compare on height and how does it compare on cataracts. So very interesting data, and I encourage you to take a look at it. It shows the data are really compelling.

And you said 80% of the diagnosed cases are in these 100 centers of excellence?

About 90% are in the 100, and the 100 centers are about -- 80% is in the 45 and about 90% is in the 100, yes.

How do your salespeople sort of cover those? I mean, let's make it a little bit easier.

We -- it does. It makes it a lot easier. We have only 12 sales representatives, 12 regional account managers, and they're responsible for those 100 centers. And because of the concentration of the business, we can actually cover those 100 centers and actually go up to about 130 centers with just 12 individuals.

And just to be clear, I mean, the growth that we're seeing, is that from people that run maybe those first-line steroids that are switching over or people starting with AGAMREE as sort of the first-line treatment?

So from the launch...

Or a combination of both?

Actually, it's a little bit of both. From the launch, we source -- we initially thought we were going to source patients from EMFLAZA, which had -- it's hard to tell because there's no publicly reported data on this market. So we thought it was about 35% share. So when the product launched and from the day of the product launch to today, it's been pretty consistent. We source about 45% of the patients from prednisone, 45% from EMFLAZA and 10% state that they're naive to therapy. So we're sourcing from the entire market instead of just 35%. So we are really thrilled to be participating in 100% of the market.

What was the name of that study that you were just referenced?

GUARDIAN, the GUARDIAN study. And just some additional information, it was announced maybe about a month or 2 ago, and the data just came out of...

And what about the SUMMIT study? Because thought the SUMMIT study was also doing something similar sort of evaluating the efficacy benefits over the traditional standard treatment of care.

That's exactly right. And so we look at the -- we're going to put about 250 patients, and these are patients not subjects. This is an open-label trial. So about 250 patients into the clinical trial. It's versus standard of care. And so we're looking at the opportunity to look at behavior, bone health, stature, fractures, cataracts and cardiovascular. So when we started the study, obviously, we didn't have the data from the GUARDIAN trial. But now that we see the data from the GUARDIAN trial, our confidence in the SUMMIT study actually has increased. And so we're really thrilled about it.

And how does that influence your negotiations with managed care and potentially payer coverage and reimbursement?

So this is a really, really salient point. We see coverage for both FIRDAPSE and AGAMREE at about 90% approval. So we're really thrilled with the opportunity to work with the caregivers and the providers to actually get the patients on, but we're not seeing very much pushback from payers.

Okay. Can we shift gears again to FYCOMPA. Haven't spend much time just sort of given where we are post the LOE, but we see the decelerating sales, you guided to, I think, $40 million to $45 million, and that's down from $115 million, right, which is maybe not all that surprising, but is there any potential opportunity for upside relative to that? Or are you doing anything on the commercial side to try to extend the life in any way or?

No. So typically, we all know that what we call a retail product, so FYCOMPA is not a rare -- epilepsy is not a rare disease. So it generally goes through the retail pharmacy. We see about 90% of prescriptions on the brand go away in 3 months, maybe even less time. Epilepsy is different. It's sticky. Patients want to stay on it longer, and we're seeing that. We're experiencing that. And this is -- this $40 million to $45 million is right in line with the performance since the generic incursion about midyear and it continues, and we're really confident. But upside, we've taken all promotion off. And so it's really mostly website and social media that's what we're doing.

Okay. Can we talk about business development and capital allocation for a few minutes. The company has over $700 million in cash. And I think on a recent call, you said you're now willing to look at clinical stage assets in addition to sort of proof of concept or different stages of development and versus just commercial-ready assets is the point I'm trying to make. And so maybe just update us on your latest thinking there. What type of opportunities you're seeing in the market? Should we expect you to start to use a good chunk of that $700 million of cash in 2026?

So we would love to put the money to work. We think that's the best use of cash in the acquisition of products. So our target -- typical target is a product in the rare space. Again, we're agnostic to therapeutic area because we believe the infrastructure we have to support the patient, get the patient on, keep the patient on if it's appropriate, is robust. And if not industry trended among the industry standard.

And so we're looking at opportunities that peak out at about $500 million, and we think that keeps us below a certain level where we're competing in a pool where we can win. So that's what we're looking at. And we're seeing a lot of opportunities out there. And the major filters we go through is, number one, has to be differentiated product. Number two, we have to be able to make money because we want things that are closer to the market, even if they are in Phase III. And then we have to align with management if it's licensing on the vision for the product. Those are the main filters we go through. And then as we get into the data, we see there's lots of opportunity out there.

And you recently enacted a share repurchase authorization to $200 million. And I think you guys were active in 4Q. You bought some stock. And so how should we -- and that authorization runs through December of 2026. And so relative to the $700 million, do you think there's an opportunity here to buy back stock and do business development at the same time and maintain some level of flexibility throughout the year?

So we have no debt, no funded debt, and we are generating cash every quarter. So when you think about a program that has about $200 million in potential spend for share repurchase, we don't think this affects our ability to do business development at all. We think we can move forward using the cash for if we need to take on debt.

Okay. We got a couple of few minutes, so I want to ask a couple of financial questions. Turning -- when we think about the guidance, $615 million to $645 million, that's 4.5% to 9.5% revenue growth. Obviously, it's a smaller number than last year, but that's explained by FYCOMPA.

Absolutely. That's right.

Yes. So I mean, could you maybe unpack that guidance a little bit for people and just talk about FIRDAPSE and AGAMREE, the growth expectations offset by FYCOMPA, anything else to sort of read into that number?

We think that -- so on AGAMREE, we can start with that. We think the choppiness of the AGAMREE situation because of what happened on gene therapy in 2025 is we're getting past that now. So we think there's opportunity to grow there. We love this GUARDIAN data. Obviously, we can't promote it, but this is a very, very tight community. The parents are generally the caregivers. The patients are very active in their therapy, and it is a concentrated market.

On the FIRDAPSE side, again, we look at the opportunity to accelerate the growth of the brand because of the idiopathic side, the gMG promotion that's out there that surrounds us and also the opportunity to work with these oncology practices to improve patient care. When you have penetration of only 10%, something is missing. And the opportunity is to utilize a very simple blood test to actually get the patient identified and then treated appropriately.

Below the revenue line, could you talk about your spend guidance, right? Because I'm sure you're trying to balance profitability here with investing in these growth assets, but it doesn't seem like you have a need to generate more near-term cash just given how much dry powder and capacity you have on your balance sheet. But how do you think about that spend guidance? And profitability expectations for 2026?

So we haven't given specifics on guidance on SG&A, but relatively flat. I mean we don't have to do much more. We're pretty excited about that. This will be the first full year, though. So you see FYCOMPA going away. This will be the first full year for cancer-associated LEMS focus in our budget. So there's sort of a trade out there. And we obviously look at that, and we think that's positive because of the growth that will generate. On the R&D side, we gave a range mostly because we want to look at SUMMIT and life cycle management opportunities, specifically for AGAMREE.

One of the things I love to say when we're talking to investors is the drug works. It's a great steroid. And it could be applicable to other opportunities in rare where you have consistent use of steroids. So we're going to explore that life cycle management. So there's probably a slight tick up there.

Okay. We're just about out of time. So I want to figure out a way to sort of bring this all together and give you an opportunity to deliver the last word. But when I sort of look at the stock, it's had a tremendous sort of 5-year run with maybe the last 12 months, it's been a little bit more range bound. And so when you sort of take a step back, what do you think sort of explains maybe the stock being range bound more so over the last 12 months versus the 4 years sort of prior to that? And when you have investors here, is there anything you think that -- I don't want to say people are missing or maybe should think about differently or you think could be a greater area of focus for them? Like is there anything else that you want to -- any other message you want to deliver to the investors now when you think about bringing it all together?

Sure. Great question. So in the last 12 -- last 2 years, we've outperformed the NASDAQ, which we're pretty proud of. I mean, nice job. And in the last 5 years, significant outperformance, great. I think the thing that we really want to focus on when we talk to investors is the life cycle management opportunities and how to grow AGAMREE beyond.

FIRDAPSE, it's like the mailman. It just delivers. It's great. It's a wonderful product, and it will continue to grow. And we think there's opportunity in there as well. So really focusing on the balance between idiopathic and cancer LEMS, but then talking about maximizing and optimizing our opportunities in DMD, but also looking beyond DMD for general use of a steroid, perhaps an improved steroid across the board in rare diseases.

Okay. All right. Well we'll leave it there. We're already out of time. Rich Daly, thank you very much, CEO of Catalyst Pharmaceuticals.

Thank you.

Good morning, and thank you for standing by. My name is John, and I will be your conference operator today. At this time, I would like to welcome everyone to the Catalyst Pharmaceuticals Fourth Quarter and Full Year 2025 Financial Results Conference Call. [Operator Instructions]

I would now like to turn the conference over to Mike Kalb, Chief Financial Officer.

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's fourth quarter and full year 2025 financial results and business highlights.

Rich Daly, President and CEO, will lead the call today; and Jeff Del Carmen, our Chief Commercial Officer and I will also present. Additionally, other members of our management team will be available for the Q&A.

Before we begin, I would like to remind you that in our remarks this morning and in the Q&A session, we will make statements about expected future results, which may be forward-looking statements for purposes of federal securities laws. These statements reflect our current expectations, estimates and projections and do not guarantee future performance.

They involve risks, uncertainties and assumptions that are difficult to predict and may not prove to be accurate. Actual results may vary from the expectations stated in our forward-looking statements. These forward-looking statements should be considered only in conjunction with the detailed information contained in our SEC filings, including the risk factors described in our 2025 annual report on Form 10-K filed yesterday, February 25, 2026, with the SEC.

At this time, I'll turn the call over to Rich. Rich?

Thanks, Mike. Good morning, everyone, and thank you for joining us today. I'd like to begin with a review of 2025, which was another fantastic year for Catalyst before moving on to the plans we have set for 2026.

2025 was defined by notable growth as evidenced by another year of record revenues, execution of our strategy to maximize the value of our best-in-class commercial portfolio and at the center of all we do, personalized support for patients living with rare diseases.

For the full year 2025, total revenues grew by 19.8% year-over-year to $589 million, exceeding our previous guidance, which was the upper end of our range of $565 million to $585 million and highlighting our ability to capitalize on market opportunities while maintaining operational excellence.

Full year net product revenue for 2025 reached $588.8 million, an exceptional 20.3% increase over 2024. This is driven by a number of factors, most notably continued patient identification and market penetration. And as demonstrated by our 2026 guidance of total revenue between $615 million and $645 million, we are confident in the continued growth trajectory of our differentiated products.

Let's begin with the 2026 forecast down by product, starting with our promoted products, FIRDAPSE and AGAMREE. FIRDAPSE guidance for 2026 is $435 million to $450 million, reflecting an increase of 21.4% to 25.6%. AGAMREE guidance of $140 million to $150 million, forecasting a 19.6% to 28.1% growth. And finally, FYCOMPA, $40 million to $45 million, which effective at the beginning of 2026 is no longer promoted as a result of generic competition that entered the market in 2025.

Now let's take a closer look at our 2025 performance. Revenue for our flagship product, FIRDAPSE, was $358.4 million, an increase of 17% for the full year and 18% when comparing quarter 4 2025 to quarter 4 2024. FIRDAPSE remains the only evidence-based FDA-approved therapy for Lambert-Eaton myasthenic syndrome, or LEMS, a debilitating nerve muscle communication disorder that results in progressive weakness and fatigue.

We are making significant headway in the 2 distinct markets for the product, idiopathic LEMS and cancer-associated LEMS, and we believe there is still significant opportunity for growth in both of these submarkets. Combined, we view the LEMS addressable market opportunity to be in excess of $1 billion. Jeff will cover the brand performance, driven by exciting initiatives that we believe will help our team deliver continued growth for FIRDAPSE.

As you know, in 2025, we finalized settlement with 2 of the 3 first filers. One suit remains against Hetero USA, and a trial has been set to start on March 23, 2026, which is prior to the expiration of the automatic 30-month stay on May 26, 2026. We remain confident in our ability to protect our IP.

Moving to AGAMREE. Our differentiated corticosteroid medication approved for use in the treatment of Duchenne Muscular Dystrophy, or DMD, a rare and life-threatening neuromuscular disorder. AGAMREE delivered 154.3% year-over-year growth with 2025 revenues of $117.1 million. Our launch strategy targeting centers of excellence penetration delivered outstanding results. With this success, we have now pivoted our efforts to going deeper in each of these core institutions.

Our goal is to ensure the greatest possible use of AGAMREE an effective and differentiated steroid in what we believe has a greater than $1 billion addressable market. We plan to tap into the full potential of AGAMREE through our ongoing SUMMIT study, a 5-year follow-up study evaluating approximately 250 DMD patients once enrollment is completed. By increasing the full body of data that assesses the potential long-term benefit over current standard of care, we believe AGAMREE can be further differentiated, allowing us to build more awareness and drive further growth.

With regard to maximizing the full value of AGAMREE, we are presently conducting a Phase I study to evaluate dose equivalence between AGAMREE and other steroids and potential immunosuppressive activity as well. We are also currently assessing potential indications beyond DMD, where AGAMREE may serve a broader array of patients with rare diseases. We look forward to updating you further on our expansion initiatives with AGAMREE as the year unfolds.

Lastly, FYCOMPA. Despite its loss of exclusivity in May of 2025, the product delivered net revenue of $113.3 million in the year, outperforming our expectations. Due to generic competition, we are forecasting sales of FYCOMPA in 2026 of between $40 million and $45 million, which reflects our expectation that FYCOMPA will remain a solid revenue producer for us.

Beyond our portfolio optimization initiatives, we are pursuing our evolved and focused business development strategy aimed at identifying the right opportunities to supplement our strong organic growth. Our business development engine conducted over 100 assessments in 2025. Notably, about 90% of those were inbound, underscoring our reputation in the industry as a proven leader that delivers value through launching, supporting and growing our promoted assets.

With our industry-leading rare disease expertise, best-in-class commercial capabilities, established plug-and-play infrastructure and trusted status within the rare disease community, we are confident in our ability to drive continued long-term repeatable success through business development.

As we assess the broader landscape and levers at our disposal to create value, we are focused on remaining nimble and acting opportunistically to ensure we are well-positioned to identify, assess and onboard rare disease products that will grow our portfolio and positively impact the rare disease community.

To be clear, we will maintain the same guiding principles that have enabled our prior success, remaining disease and modality agnostic while prioritizing on-market and near-market differentiated rare disease products.

In addition, as we reported during our JPMorgan presentation earlier this year, we have now expanded our search to include therapies in late-stage development with positive proof of concept, a differentiated profile and a well-characterized regulatory path. We will also continue to focus on assets with peak sales of up to $500 million, which is where we believe we can be most competitive and best suited to integrate with our existing infrastructure.

With that, I'll turn the call over to Jeff, who will provide additional insights into our commercial performance. Jeff?

Thanks, Rich. We are very pleased with our exceptional performance in 2025, marked by full year combined total revenues of $589 million, surpassing the upper end of our updated guidance of $565 million to $585 million. This outstanding achievement was driven by FIRDAPSE reaching a record high of $358.4 million, the continued successful commercialization of AGAMREE and the strong contribution from FYCOMPA despite the entry of generic competition.

Let's start by reviewing our advancements with FIRDAPSE, the only evidence-based FDA-approved treatment for Lambert-Eaton in myasthenic syndrome. In the fourth quarter of 2025, net revenues amounted to $97.6 million, showcasing a remarkable Q4 2025 versus Q4 2024 growth of 18.3%. Furthermore, FIRDAPSE's full year 2025 net revenues showed strong 17.1% growth year-over-year. FIRDAPSE's performance this year reflects deliberate disciplined commercial execution across the patient journey.

First, by expanding and optimizing our lead-generating channels, we increased our data leads of identified LEMS patients in active diagnostic stages by 40% in the fourth quarter. These patients consistently represent approximately 50% of new starts each quarter, reinforcing the importance of our sustained investment in early patient identification.

Second, we significantly improved our conversion efficiency through tighter coordination across field teams, commercial analytics and marketing, we increased the rate at which qualified leads transition to treatment. As a result, new patient enrollments for FIRDAPSE exceeded our 2025 forecast, demonstrating the impact of more focused execution.

In the second half of 2025, we also saw increased VGCC testing by more than 1/3 versus the first half of 2025, reflecting targeted initiatives to accelerate diagnosis among LEMS patients. By shortening time to diagnosis, our awareness activities are expanding the treated population and improving the overall patient journey.

Finally, in June, we launched a pharmacy outreach program designed to support newly enrolled patients in achieving their optimal therapeutic dose. This initiative contributed to a significant reduction in new patient discontinuations, strengthening early persistence and reinforcing long-term value.

Overall, FIRDAPSE's performance underscores the effectiveness of our end-to-end commercial strategy, expanding the top of the funnel, improving conversion, accelerating diagnosis and enhancing patient retention to drive sustainable growth. Our next phase of growth for FIRDAPSE will come from both idiopathic LEMS and cancer-associated LEMS. We will continue to leverage the increased data leads to accelerate new patient acquisition while continuing to deploy initiatives to accelerate the diagnostic journey for LEMS patients.

As for cancer-associated LEMS, our primary focus in the first half of 2026 is and will continue to be pursuing relationships with leading oncology networks to integrate the updated NCCN guidelines into their care pathways, which we believe will lead to more addressable patients in the second half of 2026. It's important to note that we have already seen significant uptake in the number of new positive VGCC tests ordered by oncologists in the second half of 2025.

Turning to AGAMREE. AGAMREE continues to build strong commercial momentum as a differentiated therapy for the treatment of patients living with DMD. For full year 2025, the first full year of AGAMREE being on the market in the U.S., we delivered net product revenue of $117.1 million, representing 154.3% year-over-year growth. In the fourth quarter alone, net product revenue reached $35.3 million, up 67.5% compared to the fourth quarter of 2024, clear evidence of accelerating demand and effective execution. 

Adoption across top DMD centers of excellence continues to expand. To date, 100% of the top DMD centers of excellence, which represent about 80% of all DMD patients have enrolled at least 1 patient on AGAMREE and 270 unique health care providers have submitted enrollment forms. This reflects broad and deepening engagement across the treatment community as well as growing confidence in AGAMRE's differentiated clinical profile.

Importantly, we saw a meaningful reduction in discontinuations and cancellations in the second half of 2025. This improvement was driven primarily by increased provider familiarity and experience with AGAMREE as well as fewer disruptions from DMD market events. We believe that this trend reinforces the durability of demand as the market matures.

Since launch, approximately 45% of patients have transitioned from prednisone and 42% from EMFLAZA, underscoring AGAMREE's relevance across established treatment paradigms and its ability to compete effectively within multiple segments of care. In addition, the median age of new enrollees has dropped 1 year in the most recent quarters compared to when we launched in 2024.

Reimbursement performance remains strong with success rates above 85%, consistent with our expectations. Our commercial organization continues to execute with precision, prioritizing targeted provider education, optimizing field force impact and deepening payer engagement to support sustained uptake and long-term market expansion.

FYCOMPA delivered full year 2025 net product revenue of $113.3 million, exceeding the upper end of our guidance. As previously noted, we expect continued revenue erosion from increased generic competition to impact FYCOMPA performance moving forward. As of December 31, we discontinued personal promotion and assistance programs for FYCOMPA. However, we forecast that 2026 net product revenues from this product will continue to be meaningful. 

In closing, our commercial organization continues to operate with rigor and accountability, translating strategy into consistent portfolio performance while laying the groundwork for our next wave of growth for FIRDAPSE and AGAMREE. Our diversified portfolio, combined with differentiated commercial capabilities and disciplined execution gives us confidence that we will be able to achieve our 2026 revenue guidance. 

Our focus is unwavering, elevating commercial excellence across markets, expanding and accelerating patient access and unlocking the full value of our portfolio to drive durable growth and sustained shareholder returns. I want to recognize and thank our entire commercial team for their commitment to execution, performance and most importantly, the patients we serve. Their dedication is fundamental to our continued success.

At this time, I would like to turn the call back over to Mike.

Thank you, Jeff. Our fourth quarter and full year 2025 financial results demonstrated another strong year, driven by our solid financial performance, financial discipline and strong execution.

Our total revenues for 2025 were $589.0 million, an approximate 19.8% increase when compared to total revenues of $491.7 million for 2024. 2024 included approximately $2.4 million of license and other revenue, which consisted principally of a milestone payment earned from our sublicensee in Japan, receiving regulatory approval to commercialize FIRDAPSE for the treatment of patients with LEMS in Japan compared to license and other revenue in 2025 of approximately $182,000. 

For the fourth quarter of 2025, total revenues were $152.6 million, representing a 7.6% year-over-year increase. FIRDAPSE's fourth quarter of 2025 net product revenue increased 18.3% over the fourth quarter of 2024 and 5.9% compared to Q3 2025. AGAMREE's fourth quarter 2025 product revenue net increased 67.5% over the fourth quarter of 2024 and approximately 9% over Q3 2025. 

For 2026, we are forecasting FIRDAPSE net product revenue to be between $435 million and $450 million, which takes into account an increase in gross to net driven by the IRA impact on our Medicare Part D net product revenue. We expect that the IRA impact will continue to increase annually.

We are forecasting AGAMREE 2026 net product revenue to be between $140 million and $150 million. We are forecasting FYCOMPA 2026 net product revenue to still remain meaningful at between $40 million and $45 million. The expected decrease is a result of the entry of generic versions of FYCOMPA during 2025. Net income before income taxes for 2025 was $283.5 million, a 31.1% increase compared to $216.3 million for 2024. 

We reported GAAP net income for 2025 of $214.3 million or $1.68 per diluted share. GAAP net income increased by 30.8% compared to GAAP net income for 2024 of $163.9 million or $1.31 per diluted share. Non-GAAP net income for 2025 was $346.2 million or $2.72 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMRE and Ruzurgi of $37.5 million stock-based compensation expense of $24.8 million, income tax provision of $69.2 million and depreciation of $0.4 million. 

Non-GAAP net income for 2025 was $346.2 million or $2.72 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMRE and Ruzurgi of $37.4 million stock-based compensation expense of $22.8 million, income tax provision of $52.4 million and depreciation of $0.4 million.

Our effective tax rate for 2025 was 24.4% compared to 24.2% for 2024. The effective tax rate is affected by many factors, including the number of stock options exercised in any given period which we expect will be relatively consistent for 2026, but will likely fluctuate from quarter-to-quarter.

Cost of sales expense was approximately $87.3 million in 2025 compared to $68.8 million in 2024 and consisted principally of royalties. As a reminder, AGAMRE royalties paid to the ultimate product licensor equals 7% of net sales up to $250 million with additional increases as net sales increase.

Additionally, through December 31, 2025, we were also required to pay 5% on net sales up to $100 million to our direct licensor. Further, we are also required to pay our direct licensor royalties of 7% of net sales in excess of $100 million and up to $200 million with additional increases as net sales increase. The company is also required to make a $12.5 million milestone payment when AGAMREE's net product revenue reached $100 million, which was achieved during the fourth quarter of 2025.

This milestone payment obligation was capitalized during the fourth quarter of 2025 and is being amortized over the estimated remaining useful life of the asset. Further details of our royalty obligations for AGAMRE as well as FIRDAPSE and FYCOMPA are disclosed in our 2025 Form 10-K. Beginning in July 2026, as per our contractual arrangement with Eisai, we will be required to pay FYCOMPA royalties equal to 6% of net product revenue to the product licensor for FYCOMPA.

Research and development expenses were $12.7 million in 2025 compared to $12.6 million in 2024. Our R&D spending during 2025 was comprised mainly of costs to support our ongoing AGAMRE studies. Relative to the normal course of business, absent another acquisition, we are forecasting research and development costs in 2026 to be between $17.5 million and $22.5 million.

Selling, general and administrative or SG&A expenses for 2025 totaled $193.8 million as compared to $177.7 million for 2024, primarily attributable to an increase in employee compensation related to annual merit increases and an increase in headcount.

Further, general and administrative expenses increased due to consulting fees related to multiple business initiatives, including business development activities, which were offset due to decreases in contributions to 501(c)(3) organizations supporting patient assistance programs.

While we are no longer actively marketing FYCOMPA, we anticipate that 2026 SG&A expenses will increase slightly compared to 2025 due to the costs associated with our continued efforts focused on increasing the awareness of FIRDAPSE for the potential treatment of cancer-associated LEMS.

As reported, we ended 2025 with cash and cash equivalents of $709.2 million compared to $517.6 million at December 31, 2024. The increase in cash of $191.6 million was largely driven by $208.7 million in cash generated from operating activities, partially offset by $17 million of cash used in financing activities, which includes the repurchase of $25.3 million of common stock during the fourth quarter of 2025.

We believe that our life cycle management and our business development activities will not be adversely affected by our share repurchases under our share repurchase plan. We believe our current funds, along with our expected continued generation of cash from operations, continue to provide us the financial flexibility to fund our existing R&D programs, meet our potential contractual obligations and support our strategic initiatives, business development and portfolio expansion efforts, leading to long-term growth and value creation.

More detailed information and analysis of our 2025 financial performance may be found in our annual report on Form 10-K, which was filed with the Securities and Exchange Commission yesterday, February 25, and can be found on the Investor Relations page of our website.

At this time, I will turn the call back over to Rich. Rich?

Thanks, Mike. As you heard on today's call, Catalyst is entering 2026 from a position of strength and with significant momentum. Our strategic priorities remain clear, and we are primed to drive continued growth.

On FIRDAPSE we'll focus on executing our dual market expansion strategies, prioritizing patient identification efforts for idiopathic LEMS and ongoing education to promote the updated NCCN guidelines for cancer-associated LEMS.

For AGAMRE, we plan to progress the multiple initiatives underway to maximize the potential of this differentiated asset, including facilitating earlier disease detection and deepening our market penetration to drive commercial expansion as well as advance the ongoing SUMMIT study and other life cycle management activities.

Lastly, we will continue to pursue strategic and thoughtful business development with an eye toward exciting and low-risk opportunities where we believe we can unlock meaningful value. We are proud of our progress in 2025 and plan to deliver continued success in the year ahead.

I want to end by thanking our employees, partners and shareholders for their support and dedication. I'll now turn the call back over to the operator for questions.

[Operator Instructions] Our first question comes from the line of Samantha Semenkow with Citi.

This is Ben on for Sam. To start, can you speak to the drivers that underpin the growth implied within your 2026 guidance for FIRDAPSE and AGAMREE?

Thanks for the question. I'll turn it over to Jeff to start the answer.

Absolutely. So, Ben, when we look at FIRDAPSE first, we are extremely excited about the opportunity that still remains. You look at both sides of FIRDAPSE that we've specified and that's idiopathic LEMS, where we feel we're only about 30% penetrated at this point. And then you have the cancer-associated LEMS where we're well under 10% penetrated. So again, significant opportunity there.

As I mentioned on the call, we have a pool of patients there that we've stated 500 in the past and that we've seen significant growth from specifically our data leads in the fourth quarter. And so now that number is greater than 600. So based on those identified patients that are somewhere in their LEMS diagnostic journey, we believe that there's plenty of opportunity here in the short term to help those patients convert on to FIRDAPSE treatment.

We've also identified some pharmacy intervention programs that have helped us reduce the discontinuations of patients in the first 4 months of treatment where we saw some higher-than-expected discontinuations. So based on those intervention programs, we've seen these patients able to reach their optimal therapeutic dose within the first 4 months. And at that point, we've seen a reduction in the discontinuation of those patients by 12%. So that's very significant, too.

So, from the cancer associated side, we've seen a 21% increase in all VGCC testing year-over-year in '25 versus '24. With that, patient identification also is critical. So, we know that number will, in the long term, turn into patients' potential leads for us to help. And then we also expect that screening to take place in the first half of the year. But then in the second half of the year, we expect more arrangements with group practices that will help us convert those leads into patients on therapy. So that's FIRDAPSE.

Jeff, just on the FIRDAPSE side, you talked about the number of leads that we have. Can you talk a little bit about the improvement in quality of those leads?

Absolutely. So, we've become much more efficient in helping these patients go from a diagnosis of LEMS on to treatment when appropriate. We use AI, we use machine learning, and we have 5 different data sources that we utilize to qualify the leads and ensure they are unique and identify which of these patient leads are the highest priority and have the most urgency to get on treatment. So, we point our sales force into that direction where to go. So, we score these leads for them. And that's been extremely helpful in helping these patients convert on to treatment sooner.

For AGAMREE, we're very pleased with the performance last year. And based on the low side of our guidance, the 20% growth this year. We expect in at least a 20% growth in 2026. I mentioned broad adoption and across the top 45 or the top COEs that make up 80% of all DMD patients. So, we expect to not only continue that, but to deepen that penetration within those sites.

We've seen many physicians now. The -- with the experience that they have, we've seen discontinuations and cancellations that have also decreased over the last 2 to 3 quarters. So that's also a positive sign of greater experience and strong adoption and acceptance of AGAMREE.

Our next question comes from the line of Joon Lee with Truist Securities.

Congrats on the quarter. This is Asim on for Joon. So just kind of on the last one, I mean, your FIRDAPSE guide for '26 is above the historical 15% to 20% that we've typically seen. So, I just want to understand of all the factors that you mentioned in the answer to the previous question, what's the biggest driver of that increase? And is that how we should be thinking of FIRDAPSE growth beyond '26 in 2027? And then just on BD, I mean, would it be fair to assume we'll see a deal this year?

Jeff, do you want to build on the previous answer to that?

Sure. Asim, the thing that really gives us the most confidence is really in the short term, the greater than 600 pool of patients, the patients that are somewhere in their diagnostic journey to LEMS, but we know they're LEMS patients not yet on treatment. So over 50% of our new enrollments comes from that pool of patients. So that gives us significant confidence.

The other big reason for confidence is the 21% growth in VGCC test year-on-year. We're actually seeing a 9% growth quarter-on-quarter of VGCC test. So that volume, the velocity of new tests that are being done also will help us identify and help these patients sooner and shorten their diagnostic journey. So that's one of the big things.

And our ability to be more efficient, like I mentioned, helping these patients that are diagnosed get on to treatment faster. And then on the second half of the year, that's when we talked about incremental patients coming from the cancer-associated LEMS opportunity. So that's when we truly expect both idiopathic and CA-LEMS patients to increase here in the second half of this year.

Before I take the BD question, Asim, just to build on Jeff's answer, too, think about the timing of some of the things we put in place. So, this will be a full year of the dedicated sales forces. We changed that in April of last year. So we expect there to be some dedication provide some value.

But also, Jeff, can you speak to the timing of the pharmacy program because that, again, was not a full year program either, and we're seeing the nice results there, too.

Thanks, Rich. We initiated the pharmacy intervention program in June of last year. And we saw a significant decrease in patients that were not able to get to the -- their optimal therapeutic dose. We saw a reduction in that. And like I mentioned, we saw a 12% decrease in discontinuations of those patients after we initiated that program.

So again, like Rich mentioned, we only have basically half a year of that program in place. And we feel that in the long-term, that will help patients get to their optimal therapeutic dose and when appropriate, stay on treatment.

I'll take question -- thanks for the questions here today. So we're going to continue along the line that we've pursued, which is a diligent and thoughtful approach to looking at opportunities and as I think we've talked about before, we really look at a couple of elements in this. We're obviously looking for a differentiated product profile rather and something that really can improve patient care.

The second element for us is we have to be aligned on the vision with our potential partner or licensor on not only how the product will launch, but what might be life cycle management opportunities. So we think that's really important. And then, again, focusing on that near-term accretive opportunity is really important to us as well. Our BD team has done a phenomenal job of exercising these elements and looking for the ideal opportunity.

The one other thing we have to take into account as we assess opportunity is especially as we go deeper into the pipeline, working our way back into post proof-of-concept opportunities with a relatively clear regulatory path is the regulatory environment today. And so we are, again, diligent and thoughtful in our approach.

As to whether or not we can say we'll do a deal in the year, obviously, we want to improve the portfolio that we have to improve care for patients. And so we'll continue our diligent efforts here and bring in products that we believe we can add true value to and the products that will also add value to the lives of the patients we serve.

Our next question comes from the line of Pavan Patel with Bank of America.

Congrats on the commercial execution this quarter. I thought it was pretty strong. So I will focus on AGAMREE commercial. And my two questions. So the first is on the detail on the median age of new AGAMREE enrollees dropping by a year. I thought that was interesting.

So maybe if you can speak to whether you're seeing a meaningful shift where physicians are using AGAMREE as a first-line steroid for newly diagnosed boys rather than just switching older patients who are already experiencing toxicities from either prednisone or EMFLAZA? And how does that capturing that younger demographic impact assumptions around longer-term patient durability?

And then the second question is with regards to the reimbursement. I know you mentioned that it's tracking above 85%. So maybe if you can just speak to what's the pushback that you're seeing in the remaining 15%. Is that just largely a step edit requirement of try prednisone first? And do you expect that 85% rate to pick up as we move through the quarters in 2026?

Sure. Great questions, and I'll take the latter first. And when we think about the AGAMREE reimbursement landscape, we're very pleased with that greater than 85% approval rates. And to your point, yes, absolutely, there are some step edits. But over time, those steps, those patients are able to get on to treatment.

What we do is we provide bridge treatment or free drug for patients while they're going through those steps. And until they're able to get approved, then we convert those patients under reimbursed patients over time. So we believe that's very, very strong. And in fact, we're closer to 90% as far as reimbursement rates go. When we look at the average age, the decrease by 1 year, so the average age in the last 6 months has come down to closer to 11-ish versus the 12 that was before. So that's very significant.

And you had mentioned it, why it's significant is because younger patients, they have greater adherence to therapy. And really, they're more likely to experience the positive tangible benefits for longer. So it's a great thing for patients but we're seeing that play out. And you asked what does that mean that patients are coming over without doing a step or going to prednisone or generic EMFLAZA or EMFLAZA. And about 10% of our patients actually come directly without ever having experienced a steroid before. So it is the first steroid for the DMD treatment. So we are seeing that.

Jeff, to build on Pavan's question, when we think about the change in age, obviously, the dosing is start at the top end of the dose and work down. So these patients are coming in, they're younger. One would anticipate maybe a different dose? Are we experiencing that?

No, actually, we're seeing the same dose.

So I think it's really positive for longevity, persistency on the drug and then obviously, for the dosing for the patient to get the benefit.

Absolutely.

Our next question comes from the line of Leland Gershell with Oppenheimer.

Congrats on the quarter. And this is Jason on here for Leland Gershell. Question on AGAMREE. One and how does the SUMMIT open-label expansion affect the forecast for AGAMREE going forward? And to what degree are the assumptions built into the 2026 guidance? And maybe one more point. How should we think about additional indications to support AGAMREE growth going forward?

So before -- I have Will join in, I just want to touch on the fact that the focus here for SUMMIT is enrolling patients, getting the patients into the trial. And with that, I'll just turn it over to Will to talk about what some of the expectations are. Will?

Yes. Thank you, Rich. Thank you, Jason, for the question. We are -- continue to be excited about the SUMMIT trial. We continue to bring in new sites and to enroll additional patients towards our goal of getting this to a trial where we have a significant enough patients where we can really pull out some robust data analysis from this trial.

We're also excited about it because of Santhera's announcement from last November as well as their upcoming MDA conference poster presentations and abstract presentations on their Guardian trial as well as evaluation of other open-label data that they have that look at really the same -- many of the same parameters that we're looking at in SUMMIT, where they actually show some important positive top line data, including comparisons to large natural history data sets that show normal growth in the AGAMREE-treated patients compared to stunted growth in the corticosteroid treated population, a decreased rate of vertebral fractures, decreased rate of cataracts, no cases of glaucoma, and these are all important endpoints in our SUMMIT trial.

We are also additionally looking at progression of cardiac effect, et cetera. So this is a trial that we are purposely driving to evaluate long-term potential benefit in these really important glucocorticoid side effects.

And for, I think, the latter part of your question, Jason, I'll pass it back to Rich in regard to how we look at it as a potential impact on performance of the medication.

Thanks, Will. This is a great question, Jason. So thank you. When we look at these data that we see for Santhera, again, it's very encouraging for SUMMIT. But we have to keep in mind that none of the endpoints that we see in the data from Santhera are in our label at this point in time. So presently, we don't have a great impact built in. I don't think we have any impact because we can't promote it.

So -- but again, we're highly encouraged by these data. We're really excited to continue the SUMMIT trial to see if we can build on the data set. And obviously, physicians can use the product as they deem appropriate, but we need a robust data set, and the Santhera data is a great start for us.

Our next question comes from the line of Luke Herrmann with Baird.

Looks like a nice FIRDAPSE guide. Can you just help provide any additional color on the extent to which you expect the growth for FIRDAPSE to be sort of backloaded? Or does the traction you're seeing with idiopathic sort of smooth things out in the first half? And I have a follow-up.

So yes, we do expect strong enrollments from the idiopathic LEMS here in the first half to help as we build the screening for cancer-associated LEMS. Like I mentioned, we do expect incremental patients for cancer-associated LEMS in the second half of the year.

I think when you build on this, when you think about how this is -- the patients are diagnosed in the journey they go through, Jeff did a really nice job of explaining how we're accelerating that when it's appropriate. And we'll continue to see that. But there is a cadence to this that we've -- I think we've seen over the last 6 or 7 years.

And on the cancer side, we're working to advance that, but we would expect there to be an incremental opportunity in the second half, especially when we see the VGCC tests increasing, I think that's a really good sign, but it does take time to get those patients into the treatment queue.

Great. And then just a follow-up on business development. I guess, in light of the favorable trajectory for XBI recently, do you think this could make BD activities more challenging? Or have you not seen much of a change to the breadth or quality of inbounds at this point?

We're seeing no change to the quality of the inbounds. We like the opportunities we're currently evaluating. And it comes down to the 3 or 4 points I looked at before when we get into due diligence, making sure that we have good alignment opportunity to offer differentiated and improved care and then obviously return -- get a return as quickly as possible, as reasonably possible.

Our next question comes from the line of Sudan Loganathan from Stephens Inc.

This is Kesav on for Sudan. Congrats on wrapping up 2025. So I understand that VGCC testing initiatives are primarily expected to materialize during the second half of this year. But could you all provide some color on the magnitude testing volume has possibly already had on FIRDAPSE sales?

And then my second question is, could you talk about how the Phase 1a readout in this quarter on establishing translational dosing is expected to impact your plans for AGAMREE going forward?

Jeff, why don't you take the first one and then Will shall take the second.

Like I had mentioned, there is significant growth year-on-year, 21% in the year-on-year for the VGCC test. So that's always a positive sign. We know it's tough to tease out what -- how much of that is the cancer-associated LEMS or test ordered by an oncologist versus a neurologist. So I would say primarily, a lot of that increase is still stemming from the idiopathic LEMS side. So there's plenty of opportunity still remaining to continue to increase the VGCC testing within oncologists. So that presents a significant opportunity still for us moving forward.

Jeff, as you look at the VGCC testing and that is an indicator of conversion to therapy, where would that rank as an indicator of conversion?

It's of our patients coming in each month, it's about 50% to 60% of our new enrollments come from these new leads.

So that's increasing?

Absolutely.

Will, do you want to take the next question? And I'm sorry, could you just repeat the question so we have clarity on it?

Yes, for sure. So like for the Phase 1a readout in this quarter, first quarter of 2026 on establishing that translational dose for AGRAMEE, -- could you kind of just provide some color on like how that readout might impact your plans going forward for AGRAMEE or if there was going to be any substantial impact?

Yes. And happy to answer that question. Thank you for the question. One quick point of clarification is that we've announced that we will have analyses of that data within the first half of 2026, so not this quarter, just for that clarification.

And effectively, what you're referring to is our evaluation in this study of biomarkers of inflammation as well as immunosuppressive biomarkers on multiple doses of AGAMREE. As to how that might impact business development or I should say, life cycle management opportunities for AGAMREE is essentially if we see, say, strong immunosuppressive effects, it might direct us towards certain life cycle management opportunities.

And if we see minimal immunosuppressive effects, again, that might direct us to other life cycle management opportunities. The life cycle management evaluations broadly for AGAMREE are important for us and active, and we're excited about a number of the potential additional target indications that we're evaluating currently.

And to build on that from a business impact, when Jeff mentioned on the call that we're seeing high retention rates, so there could be a potential business impact to getting the right patient on, but we are seeing a reduction in patients who are not staying on therapy, so a higher retention rate.

And remember, about 45% of the patients switch from prednisone, 45% switch from an EMFLAZA, whether it's branded or generic. And so, the physicians are getting more and more comfortable over time. And this could just improve that with a little bit more direction should we be able to get it into the label, but that's yet to be seen. But just to build on that, we're happy with the progress we're making on retention as well.

And at this time, we have no further questions. That concludes our question-and-answer session and today's conference call. We would like to thank you for your participation. You may now disconnect your lines. Have a pleasant day.

Good afternoon, everyone, and welcome to the 44th Annual JPMorgan Healthcare Conference here in San Francisco. My name is Alex Kramer, and I'm an investment banking associate on our health care investment banking team. It is my pleasure to welcome our next presenting company, Catalyst Pharma.

Today's presentation will be given by Rich Daly, President and CEO of the company. Following Rich's prepared remarks, there will be some time for Q&A. We ask that you hold any questions you have until that time.

And with that, it's my pleasure to turn the mic over to Rich.

Thank you, Alex. And I just want to say thank you to JPMorgan for the invitation. It's a pleasure to be here with you and meet the investors and the bankers and the community that's interested in rare diseases. So thank you very much.

Obviously, safe harbor statements, I think you're all familiar with that. So who is Catalyst Pharmaceuticals? Catalyst is a commercial stage biopharmaceutical company focused on rare diseases. And we look to in-license, purchase and then develop, if necessary, products for the use in rare diseases. We're really proud of our legacy, and we're really proud of how we do business. We're going to spend some time talking about that today.

So when we look at the opportunity in rare diseases, really, we have 2 molecules that we use. One is FIRDAPSE for Lambert-Eaton myasthenic syndrome. It's the only evidence-based product that's approved for use in Lambert-Eaton myasthenic syndrome or LEMS. You'll be hearing about LEMS a little bit. And then AGAMREE. AGAMREE is a differentiated steroid for use in Duchenne's muscular dystrophy. And some very interesting data has come out of Europe recently, which we're really excited about, which supports our opportunities here and supports the improved care of patients.

So we are forging new paths. That's our goal, is to forge new paths to support those individuals living with rare diseases. And we really have a growth through patient-first approach. We really look at the approach, how we can best serve the patient, and we'll talk specifically about that actually on the next slide. We have bespoke patient programs that ensure that patients not only can get on therapy, stay on therapy and optimize their dose.

And we have a 90% compliance rate with our medicines because of our approach through a specialized pharmacy, hub and 3PL, third-party logistics provider. We're a recognized partner for value. We do a significant amount of business development and 90% of our acquisitions that we looked at in 2025 were inbound. In other words, people were looking at us saying, this is a company that performs, this is a company that delivers, and we are really proud of that statistic. And since the commercialization of the company, we've had a 37% CAGR. And again, looking at building on that as we go forward. And for AGAMREE, which has been on the market since March of 2024, we have a 224% growth of that product through September 30, 2025.

And one of the things we're really proud of, and I think caused a little bit of consternation in some of the conversations we have is we're a profitable company. We're a commercial company in rare and we're profitable. And that's rare. And we're really proud of our ability to manage our business really effectively. We have more than $700 million in cash, no funded debt, and we have the ability as a buy-and-build organization to acquire -- to spend more than $1 billion in acquisitions. And we'll talk specifically what we're targeting when it comes to the acquisitions or licensing or partnerships.

So how do we serve the patient community? Well, we look at it as a threefold opportunity. One, we have industry-leading hub, our patient services hub, best-in-class patient support and then we also look at a high-touch specialty pharmacy. This is our program called Catalyst Pathways. And when we look at the crossover, the Venn diagram that you see on the screen, it becomes really important. We look at free bridge medication. So from the time a patient is diagnosed and enters our system, we give them free bridge medication. If the patient can't get insurance coverage, we guarantee them insurance coverage through -- in appropriate programs by working with our field-based assets that report to the company and working through the pharmacy. We have personalized insurance navigation through the hub and through our -- what I call our patient advocacy liaisons. And then we have broad co-pay programs for those patients who are potentially in government services, and we actually direct them through the hub to better services for -- better insurance coverage rather.

This high-touch specialty pharmacy, I talked before about the 90% retention rate in our patients. We have -- as a pharmacist, a health care practitioner, they can actually reach out to the patient and help the patient appropriately titrate the drug, stay on the drug and address any questions and actually work with physicians. And we'll talk specifically about how they do that with the LEMS physicians, the neurologists. Obviously, we have health care practitioner education and then direct-to-patient outreach, where we actually will call those patients and ensure that they actually get the prescription they need and then can titrate appropriately.

One of the things we're really proud of is our patient advocacy and the patient support that we provide. We have these patient access liaisons that we have in the field that work with patients through permission marketing. So they actually get permission from the patients and that help the patients understand the disease because it's so frustrating for rare patients to actually understand their disease. Typically, for us and our patients, especially on Lambert-Eaton myasthenic syndrome, the diagnostic journey is more than 4 years from first symptom to actually understanding their disease and getting on therapy.

We have the patient ambassador program. These are patients that are actually on the drugs that we have and they'll talk peer-to-peer with the patients who are considering it. Again, arm's length transaction, we stay away from it. The patient is actually free to speak as they deem appropriate. And then we also partner with independent patient advocacy groups to be sure that they have the support that they need to get the information out about our therapies.

So we have a clear and focused acquisition strategy to supplement our organic growth. So we have 2 arms of it, organic and inorganic. The inorganic growth is the business development that we seek. And so we look at rare disease products across diverse areas. We are therapeutically agnostic and modality agnostic because we believe that the infrastructure that the company has to support the patient and the physician and the provider -- I'm sorry, provider and the caregiver, namely the parent, we think those are applicable to all therapeutic areas. We are looking for immediately accretive or nearly immediately accretive opportunities because we want to continue to build our P&L, build the strength of the company so that we can actually generate more cash to get access to more patients -- or more products rather to help more patients. And we are also expanding our mandate to look at products that are in development. So by working with companies that actually have products that are past proof of concept, we can then take a look and see if those products would fit our portfolio.

We seek opportunities with a peak sales of up to $500 million. And the reason for this is the way we describe it is that once you get above the $500 million threshold, different people show up at the auction, different companies. And while we have a really strong balance sheet and a really strong income line, we want to be sure that we are one of the strongest folks at the table when we do, in fact, go for it. And we are looking for U.S. and ex U.S. worldwide rights for our products, the products that we are investigating.

So a little bit more detail. A proven business development engine. So we've acquired all of our products, obviously, and developed FIRDAPSE and then had a partner, a licensor in Santhera for AGAMREE. So we have established infrastructure that is a plug-and-play infrastructure. We can put those products in, get them out of the market and actually get them to patients rapidly and support those patients.

Demonstrated success in launching. I talked about the statistics around AGAMREE, wonderful launch, tremendous opportunity to improve the care of patients with Duchenne, but also the sustained growth of FIRDAPSE. We've had 15% to 20% growth for almost the entire life of the product. And we all know about decreasing increases over time. You would expect products to slow in their growth. FIRDAPSE on a bigger base continues to grow at 15% to 20% per quarter. It's astounding. And it mostly is because of the commercial and medical outreach we do in identifying patients, and we'll go a little bit deeper into that.

As I talked about already, end-to-end patient access programs, we want to make sure that patient has the support they need to make the best decision they can for themselves and along with their health care provider. And then adept at extending value. So we look at opportunities for extending the value, so typical life cycle management, looking to build on the success we've had with the products we already have access to.

And as I mentioned before, financial flexibility. With $700 million, no funded debt and dropping roughly about $60 million to the bottom line every quarter, we have a lot of flexibility. So very, very strong P&L and a well -- really well-managed company.

So these are the products that we have, talked about it. FIRDAPSE is the only evidence-based treatment for the -- therapy for the treatment of LEMS for Lambert-Eaton myasthenic syndrome. And we believe that this market has an addressable market in excess of $1 billion. So we're really excited about this one. AGAMREE, equally excited, a newer product for us, as I mentioned, novel corticosteroid. Again, the addressable market here is $1 billion. The fundamental difference in these 2 markets is for FIRDAPSE, we are the only player, and there's an opportunity for us to really excel here. And then for AGAMREE, we are 1 of 3 players in this market space. But again, we believe we have a different story and a differentiated story to tell.

FYCOMPA is for treatment of epilepsy, extended patient preference driving durable revenue performance following generic entrants. We had generic entrants come into the space in June of last year and the performance of the product has been outstanding. Typically, there's a stickiness that's generally associated with epilepsy products. There is another product that went generic a month before us, and we have outperformed that product by 2x with generic incursion. So really strong performance.

So let's break down our markets. So we talk about Lambert-Eaton myasthenic syndrome, debilitating nerve communication issue, nerve muscle communication opportunity for patients to improve. We believe there are between 3,600 and 5,400 patients here. And all of the information I'm going to share with you is at the lower end. We always assume the most conservative base. So we're going to talk about 3,600 patients. 50% of these patients have Lambert-Eaton myasthenic syndrome associated with a cancer, typically small cell lung cancer. And we have a pool of about 500 patients that we've identified on their diagnostic journey. We do this through market intel, competitive intel, machine learning and artificial intelligence. And 50% of our patients every month, every quarter come from that pool of patients. This is a prospecting market. So having to stay out in front of these patients and understand where they're coming, this is what's generating this continuous growth of 15% to 20%.

So breaking these 2 markets down. This is really a story of 1 molecule, 2 separate markets, and we have equal focus on both markets. Idiopathic or ILEMS is not -- is Lambert-Eaton myasthenic syndrome not associated with cancer. And this is typically treated in the neurologist office. Estimated prevalence as we said, 50-50 split between these 2, so about 1,800. New diagnosis per year. So this is the potential new diagnoses. In ILEMS or idiopathic LEMS is about 150, and cancer-associated LEMS is about 900 per year. The life expectancy of these 2 populations is quite different. So you would expect a cancer patient with small cell lung cancer, a devastating diagnosis to not live as long. So there's interesting opportunities here to actually work through all the oncology practices that are out. We believe the accessible market population is about 80%. So patients who are newly diagnosed might not have significant physical issues and might not demand therapy and patients who are well advanced in their disease process might be too far along. So we're, again, taking a conservative view of this market opportunity.

The success we've had so far and the 15% to 20% growth with such a small population of patients, really, you're looking at market penetration on ILEMS of only 30%. So there is significant upside here. And on the cancer-associated side, we've made tremendous leaps in the last year on our ability to reach this patient population, and we expect this to be a real growth driver for us and a value driver.

So how are we going to attack these markets? So let's talk about idiopathic LEMS for a second. You'll see the same structure here. We have 4 basic steps that we're trying to accomplish in growing this market and helping patients who are suffering and frustrated, help them get the best therapy they possibly can. So we talked about the ongoing patient discovery and looking for those patients and sourcing those patients. So we have that opportunity. Myasthenia gravis is #2. It is the #1 misdiagnosis for this disease. And with all of the noise that's going on in the marketplace around FcRns and the direct-to-consumer advertising, we think this is a tremendous opportunity. More patients will be diagnosed with myasthenia gravis and therefore, misdiagnosed.

And so the next step, we're looking to increase the rates of diagnosis by getting reflex testing done at national labs. So if you come up negative on your myasthenia gravis diagnosis for a blood test, you should automatically get a reflex test for a VGCC test, which is determined -- helps determine if the patient actually does have LEMS. And so now our focus is on -- we've checked off the first 2 boxes. We're focused on box #3 and then growing the awareness of testing throughout the community. So being sure that there is actually reflex testing that is going on. So that's the ILEMS portion of our business.

On the cancer-associated LEMS portion of our business, we have a similar structure as well. We got streamlined testing what Jeff Del Carmen, our Chief Commercial Officer, calls frictionless testing. Previous to our arrangement with a national lab, a patient would -- if they were -- the physician thought they were going to be diagnosed or wanted them to have a blood test, they would have to go to a distant site, have the blood drawn, come back with the results for the physician. Now we have frictionless testing, the blood is drawn in the office and picked up. This applies actually to both markets, which has really been quite helpful on the idiopathic side.

National Comprehensive Care Network, or NCCN, updated their guidelines in July to include not only how to diagnose LEMS in a cancer setting, but also that FIRDAPSE is the only evidence-based medicine for the treatment of LEMS. So boxes 1 and 2 are checked off. And now we're working with large, high-control oncology practices to take the NCCN guidelines and translate them into local clinical practice so that they are following their own care pathways in their practices and then forging a relationship with these practices to do ongoing education as well. And we're starting to see a significant uptake here. The testing of VGCC test, the trend in it is increasing 9% per month -- or per quarter, rather, for the last 2 years. So we are gaining traction, and we're really excited about this opportunity.

Translating now to Duchenne's muscular dystrophy and AGAMREE, I think everybody here knows the story, rare life-threatening neuromuscular disorder. There are between 11,000 and 13,000 boys and young men who have this disease. 95% of them are typically diagnosed. So hold this in juxtaposition to the LEMS market where patients are not diagnosed, they are frustrated. They have a long diagnostic journey. Here, the diagnostic journey is quite quick. And we also see opportunities for newborn testing as well, which should accelerate that.

The challenge is that 90% of patients have been on a corticosteroid at some point, but today only 70% are. This speaks to a gap in the offering that DMD -- or the corticosteroids that they could provide to patients. So there's a wide market gap here. So AGAMREE is a differentiated, uniquely positioned to -- a differentiated profile, uniquely positioned to address the unmet clinical need. A patient that takes a steroid from diagnosis and takes it continuously will walk for 2.5 to 3 years longer than a patient that does not take steroids. The challenge with steroids is their adverse events. We want patients to take steroids, but we also want them to be healthy. We'll talk about that a little bit more.

So it's -- AGAMREE is clinically proven to work. It's equally efficacious to prednisone. It has a potential for significant reduction in steroid-induced adverse events. And we're seeing this play out in the marketplace and the feedback we get from physicians. And the recent addition to the DMD testing to the recommended uniform screening panel by U.S. HHS or Department of Health and Human Services, could drive earlier diagnosis of these patients, which we think is absolutely outstanding. So we're really excited about this because, again, getting them on a steroid that could be safer for them, it could create real benefit.

So how do we achieve AGAMREE's full potential? So we have a multifaceted approach. We have a strong clinical profile with outstanding acceptance. 90% of the top 100 centers of excellence have used, at least -- have diagnosed and treated at least 1 patient. So we have good penetration here. And we needed to leverage this to further drive HCP awareness. So we have breadth and now we need to go for depth. So 85% conversion from branded and generics. So there's prednisone and deflazacort, 2 products that are out in the market. And we're getting 90% to -- 90% of our conversion from those -- our patients are coming as conversions from those 2 products. 10% are naive.

When we first launched the product, we assumed we would go for patients with deflazacort, which was a branded product just prior to our launch. It turns out we're playing in 100% of the market, which is tremendous upside for us. And then we have to realize the full potential. We're conducting a Phase I study to look at the translational dose between prednisone, EMFLAZA and AGAMREE. And we're also looking at the immunosuppressive potential for it because other diseases outside of Duchenne's, it would be great to have a steroid that you take long term in the rare space without immunosuppression. And there's some really interesting data, as I said, that's just recently come out of Europe.

So again, looking at the opportunity to evaluate the long-term safety benefits, and we're doing the SUMMIT study, open-label 5-year study with up to 250 DMD patients across approximately 25 centers. Real-world evidence, we're looking for a number of parameters. One, behavioral -- improved behavior. Being on a steroid is challenging for the behavioral aspect, especially for young boys and young men. Stature and growth, long-term steroids, traditional steroids actually suppress growth. They also hurt bone formation. So you have shorter young men, which is a really big issue in the space for young boys. And then bone health, you could have increased fractures. And then cardiovascular health is the long-term issue, the true long-term issue for this, and this is what most patients with DMD actually die of is cardiovascular issues. But there's also cataracts, too. And so we're looking for SUMMIT to help us uncover how this might work out for patients, be better for patients.

The last product we have is FYCOMPA. And as we mentioned, differentiated epilepsy product, but it actually faced generic incursion first half of last year, late in the first half. And then actually, it's oral solution actually experienced generic incursion in December, but continues to outperform and really do quite well given the dynamics you see in epilepsy.

So here's the performance of the company, as you can see, 17.4% growth third quarter on third quarter and then a 25.6% growth. So the company continues to do a phenomenal job in execution from a medical and commercial standpoint. And our products with -- obviously, we're going to expect to see FYCOMPA decline, but we're looking at both products actually taking up the mantle and growing faster and resulting in better growth for the company.

So we get asked all the time about guidance and what are you going to do? And we want to just give a high-level update on how the company has done. So for 2025, we actually updated guidance late in the year, and we are looking at achieving the upper end of the guidance here. So we're really excited about that. And then going product by product with FIRDAPSE. We have a very tight range that we held to at our most recent update, and we are going to be within the range, which, again, great performance here.

And then AGAMREE, given all the choppiness in this market, you see with ELEVIDYS and other things that are going on, and we don't compete with ELEVIDYS, but obviously, the parents were very concerned about what was going on. So we were looking to make a statement that, "Hey, this is a good and safe product." It became very challenging for us. And really here, we're looking at or slightly exceeding the upper end of guidance. So again, very exciting for us.

And then FYCOMPA, I talked about upper end of guidance here, and we raised the guidance on FYCOMPA late in the year.

As I mentioned several times, because my colleague is very proud of this as are all of we, but we have $700 million in cash and no funded debt, which gives us the opportunity to really be quite aggressive in the business development space and in investing in the existing products and the life cycle management of the products.

The achievements, I'll just go through really quickly. FIRDAPSE NCCN guidelines, we talked about, settled patent litigation with Teva and Lupin for FIRDAPSE. FIRDAPSE launched in Japan. We have a sublicensee in Japan, DyDo. They launched. AGAMREE, sustained continued demand. The product is performing very, very well. We advanced the SUMMIT study, which is critical to the success of differentiating the product in the market. And then we have a sublicensee in Canada, Kye Pharmaceuticals. This is the first product approved in Canada for the treatment of Duchenne's muscular dystrophy.

And then on the '26 priorities, pursue immediately or nearly immediately accretive acquisitions, and again, look for that opportunity to work with products that have established proof of concept. FIRDAPSE continue to roll out the NCCN guidelines to those large, high-control oncology practices and advance patient identification on the idiopathic side of LEMS. Again, we're 50% of new patients start -- greater than 50% of new patient starts. And then protect our FIRDAPSE IP. We have one last defendant, Hetero, and we're looking to defend it. And AGAMREE, drive deeper. As I said, we've gone broad. Now we need to go deep and evaluate life cycle management opportunities to begin to actually move backward into the development of drugs and develop our capabilities to develop. And then leverage DMD RUSP, which is the recommendation for infantile or newborn testing. And then continue to advance the SUMMIT study.

So with that, I'll take questions.

So I'm going to have a few questions and then we'll open it up to the audience. I know we have some in the audience as well. So no worries. So I'm just going to ask a few and then.

So I guess just first on FIRDAPSE, can you provide some more detail around the 2 markets that you described, both the cancer-associated and idiopathic LEMS? I think you said $100 billion -- or $1 billion opportunity. So can you just talk about how you think about the size of those sort of -- it's 100 -- it's $1 billion combined, but what is the split between the two?

So roughly, they're about the same size. If you were to have -- if we were to achieve the market, actually, the cancer-associated LEMS side is slightly bigger, but they almost split 50-50, but just slightly bigger. So yes.

And then I guess staying on that, and you mentioned at the end, just with Hetero being the last litigant remaining, can you just talk about your confidence in the IP there and if that goes to trial?

Sure. So I'll give you a time frame. We have a hearing with the magistrate later this month. And then the trial is set to go in late March and the 30-month stay is set to end in May. However, if the judge doesn't have enough time between the trial and the extension or the 30-month stay ending, he can actually extend it if he wants to. So that's the time frame.

Our confidence is really, really high. We had 4 filers. One was not a first filer. So we have 3 first filers. Teva was the first to settle. And Teva is a very sophisticated company. They saw the IP. They had the opportunity, and they decided to settle. And we would -- under the agreement we have with them, they could come in, in February of 2035. So we have 9 years of life left with already having 7 years of life in the product. So we're really excited about that. Lupin settled for the same time frame. And again, we're really confident in our intellectual property estate around FIRDAPSE.

And then just one more for me. So just on BD, I know you talked about it at the outset. You mentioned looking at some clinical stage opportunities. Can you just give us a little bit more detail around those expanded parameters that you've put in place? And how you think this approach will impact your business development activity at assessments?

Yes. So we have to look at the capital markets and what's happening with the capital markets. So in the last 2 years, the capital markets, as we all know, have been very, very tight. So a company like ours that can actually partner with or license from another company, we have really strong opportunity because of the strength of our balance sheet and our ability to execute. As the markets get a little bit looser and loosen up, we see that this could present a challenge for us. So about a year ago, we started working on how do we get back into the pipeline, how do we move so that we can actually have future-oriented milestones we can talk to with investors and patients. And so we see that opportunity with both FIRDAPSE and AGAMREE, and we believe that bringing those assets in and developing those capabilities and developing those products give us an opportunity to have a much more future-oriented story.

So we widened the aperture. We believe that our skills and our capabilities are applicable, as I said earlier, to any therapeutic area because about what any patient group would want is about 90% the same, therapeutic area to therapeutic care in the rare space. They want a lot of support. They need that drug delivered on time. They want to talk to other patients and our infrastructure is there, and we believe we have an industry-leading infrastructure to do that.

Audience question.

Very interesting comments. I have a question about the difference across the 3 products in pricing, what your assumptions were when you were developing those products? I noticed the first one, you said has no competition. It seemed like the revenue guidance was roughly 3x that of the other segments. I assume that's reflected in there because they're all about $1 billion total addressable market. But I'm curious, what did you learn along the way for the life cycles of these different drugs when you were relying on your own assumptions and then when you got to the payer discussions, particularly because it's a very different game in rare disease than it is elsewhere.

Absolutely. Great points. Been in the industry for 30 years, 35 years, and I think it's really important to pay attention to what the payers tell you. And so as an example, with AGAMREE, we knew that deflazacort -- we are going to have generic entry of the deflazacort in the DMD market prior to our launch. So we spoke with 17 decision-makers about what should we do and how should we do it? And we took their guidance and we see almost no pushback. You have typical step therapy that's there, but I think listening to the payers and understanding the payer perspective here and when we do business development, we actually go out and we talk to payers as we're thinking about what products we might bring into the organization.

So I think the lessons learned are the payers -- we want to make this as easy for the patients as possible. We don't want to create a barrier for patient utilization. We think we all win. The difference in their sales right now is you have a launch curve for AGAMREE, which is still going up. And FIRDAPSE is doing quite well, but had a 7-year -- 6-year head start, 5- to 6-year head start. So you're seeing just a basic history and the relative size of the 2 products. To be really clear, in DMD, I'm not sure that with 3 other players in the space, I'm not sure that a game can achieve the same level of success financially that you would have for a solo player in the marketplace. But again, we want to differentiate the product, show that it has potential to be the best and then continue that. Thank you.

Maybe I missed this in your presentation, but you are acquiring already approved drugs. Is that right? Or are you developing them in clinical trials yourself?

So currently, we developed FIRDAPSE ourselves. We did that. We did the development of the product. Today, our strategy is to acquire immediately accretive or nearly immediately accretive. So think of products that are on the market or products that are actually in development. So as an example, AGAMREE, we acquired the license to AGAMREE while it was post the FDA -- NDA filing. So we're willing to actually now move backward and take on appropriate risk, we believe.

So why is a company willing to sell it to you? Are they -- do they think that they can't develop the market or they don't have the capital to do it? That's -- I really am interested in why -- how you're acquiring these products and when you acquire them, number one? And the second question is there are a lot of new technologies coming along for DMD, epilepsy and so forth that are pretty exciting. How are you going to compete with those?

Sure. So first question, why would somebody want to partner with us right? Well, they may sell it to us. So for instance, we bought FYCOMPA.

Are you buying 100% of the drug? Or you...

We did. For FYCOMPA, we did. And then the other products, we actually licensed from somebody else. So when you look at it, it may be a change in strategic direction. So I think with FIRDAPSE, the reason why we got that was the company had a change in strategic direction. That happens all the time. With AGAMREE, AGAMREE is developed by a Swiss company with not a very strong presence, had a commercial presence, very small in the U.S. But when they took -- they look at what would it take versus what kind of cash they have, they may say the better issue -- the better opportunity for us is to actually license this product out. So think about companies that have enough cash to maybe launch but not sustain the launch because launch is typically 2 to 3 years or they don't have a footprint in the U.S., and they want to get into the U.S. and establish something in the U.S. So it could be a starter for them to just generate revenue for the company. So that's typically what we see.

And then your second question?

On the new technologies coming along for DMD, epilepsy and so fort.

So we look at CNS and our -- one of the reasons why we increased the aperture was because all of the growth is coming in movement disorders. And those opportunities are much bigger than $500 million peak. So that's why we're looking at other areas. When we talk about DMD, to your question, steroids are the foundation therapy. You can see gene therapy coming in. You can see other therapies coming in, but that patient is going to be on a steroid, we hope a better steroid and we look for that opportunity. Epilepsy is not a rare condition. We got access to FYCOMPA as a way to improve our cash flow so that we could buy products, build our balance sheet and go forward. We've got great talent. We've got great people with that acquisition as well, and that was an acquisition. But our goal is -- we don't see ourselves in epilepsy in the future. It's very crowded. 90% is generic. High Science sales, we love the people we got with it because they're highly qualified, but it's not a long-term play for us.

And then I guess just one to stick on the BD point a little bit. In your presentation, it said 100-plus BD assessments in 2025. I guess is that saying you looked at 100-plus potential BD opportunities? And then a question related to that would be, I guess, what did you see as the main reasons why you might not have pursued a particular opportunity?

Sure. So Preethi and I talk about this all the time. Preethi is our Head of Business Development and Strategy. We look at 3 high-level filters first. The first filter is, is the product differentiated and is it -- is there a high unmet need? The second one is we have to be able to earn money. Otherwise, there's no point in servicing patients, right? So we want to be sure that it's a profitable venture for us. And the third element, which I think is typically undervalued in alignments or associations with other companies licensing or acquisition is culture and fit. And so we did evaluate at some level, $135 million, some went much deeper.

Sometimes when you're talking to a company, they'll say, "We believe that the product is differentiated, and it's an unmet medical need." You get into the data room and it's maybe not as good as proposed. Or in this -- to the second point about being able to be profitable on that product, sometimes people have very interesting valuations about what they think they should get for their product. And so we have to be thoughtful and say, "That may be good for somebody else, but we can't go down that road." And then the third one is you get very far down the line, and you learn a lot about your partner and you say, "I realize we're at the alter, but can't really marry you. I'm opting out." So -- and that does happen from time to time. It's very rare because you don't get that far. You'd typically learn a lot about somebody before you get that far.

So it really just hinges on those 3 pillars you talked about are the 3 sort of biggest screening mechanisms and then from there, it's sort of...

Right. And then we go down and we talk to payers and we say, "Okay, is it realistic to do this? What's going to happen with the reimbursement schema and always going diving into the deep and looking at the FDA minutes or FDA communications because we may have a view of our product, but the FDA may have a different view, and we want to know that before we do the deal. So there's a lot -- it goes -- a lot more goes into it.

Thank you very much. Thanks for your time.

Ladies and gentlemen, the program is about to begin. At this time, it is my pleasure to turn the program over to your host, Jason Gerberry.

Good day, everybody, and thanks for joining us at the BofA virtual CNS conference. My name is Jason Gerberry I'm one of the biotech analysts here, and I'm joined by Pavan Patel, who's one of my colleagues who helps with Catalyst Pharma. Catalyst Pharma is our next company presenter and -- Rich Daly, CEO. So Rich, thanks so much for joining us and giving us the latest update on Catalyst.

Jason, thanks for having us. I really appreciate it and looking forward to the conversation. So thank you.

So maybe just starting with kind of the overarching BD in licensing, that's obviously kind of core to what you do in building out the business. And you had sort of a stopgap product with FYCOMPA, but your 2 main assets for kind of the longer-term are AGAMREE and FIRDAPSE. And so how important is it for you to get a resolution with FIRDAPSE IP before you deploy capital in a more significant way? Maybe just talk about the importance of doing a deal in 2026 as a priority. Maybe we can start there and then delve into additional questions.

Sure. So we feel like with the acquisition or the arrangement with the AGAMREE and Santhera. Santhera is the originator of the product. We feel like we've had ample time to incorporate the product and digest the product and move the product forward. We're always on the look for opportunities. As you mentioned, we're a buy-and-build company. And so we're looking for those products that are immediately accretive or nearly immediately accretive. They're in the rare space, differentiated unmet clinical need and a ceiling of about $500 million in sales. So what we're trying to do in that scenario is eliminate the clinical risk, minimize the regulatory risk.

We did take some regulatory risk with AGAMREE and then absorb the commercial risk because we think that's a hallmark and the strength of the company. So we want to get a deal done. We want the right deal, however. And I think you saw in our press release for Q3 earnings, we -- since the beginning of the year, we assessed more than 100 opportunities.

And one of the things that's fundamentally changing in the marketplace is the capital markets. So typically, an opportunity for us would be a company that has enough money to launch, but not sustain. And so with our infrastructure already built, we can come in and really take over, or an ex-U.S. company, Asia, Europe, that is looking for an opportunity to plant the flag in the U.S. that doesn't want to take the commercial risk.

But with the capital markets opening up, again, we may be in a situation where we have to broaden the scope of what we're looking at and perhaps go back just a little bit into the pipeline into the Phase III, which is proof of concept is assessed and is positive and looking forward. And we've been talking about this for 2 years.

So that's the nature of what we're doing. We've been looking at those opportunities and assessing them as well as the immediately accretive or nearly immediately accretive opportunities.

You mentioned -- and maybe I haven't heard this part before, but a ceiling of $500 million, that would be a peak revenue is sort of like what you're screening for?

Yes. So when we think about what can move the needle for us and what can move the needle for competitors who might be larger. You stay around the $500 million, and that doesn't really necessarily move the needle for some of these companies. So it's a different set of companies that show up at the auction. So when we're there, and we have about $689 million in cash and no funded debt, we're a powerful bidder. We don't want to get into a war of attrition with a much larger player. And as good as our bank is and our balance sheet is and our ability to take on debt, we're not going to compete where we can't win. And some of those larger opportunities just aren't for us right now. That will change over time.

Yes. Well I know there's been like analogies to your business model to Horizon Therapeutics who had a model like this. And there was probably a time when people thought TEPEZZA would be a $500 million peak drug, right? There is an opportunity for FIRDAPSE to eclipse $500 million in peak revenue, right? So is it that maybe $500 million is sort of a conservative base case when you look at assets, but they have the upside potential to exceed that revenue threshold just.

Yes. Great question and great commentary. Yes, so we look at it and we want to -- we think about how we might be able to accelerate that opportunity. So when you look at the opportunities to grow products, really, you're looking at your company, rather, in the life cycle management, take the products that are in line, commercial, how do you get patients on earlier keep them on longer or optimize their dose. Then there's expansion -- and we do that for both AGAMREE and FIRDAPSE. And then there's expansion within the claim.

So you look at FIRDAPSE as an example, that expansion for LEMS applies both idiopathic and cancer-associated LEMS. And we have a real focus on maintaining our idiopathic business, but also growing. And then obviously, there's label expansion, too. And we're going to look at that for AGAMREE as well.

We think there's opportunity. This is a steroid that works. There are data that's just come out of Europe from Santhera, the GUARDIAN data that look really, really promising and support our work on the SUMMIT trial, which we can talk about. But as the steroid works and it has a favorable profile for long-term steroid use versus standard of care, we're going to look for other rare disease opportunities to apply that molecule.

And so that's one of the key initiatives we've got in growing the company. So you put the whole thing together, the last one for the company is business development and looking to actually accelerate growth of the company through incremental products.

When do you think you'll have a handle on that aspect of other rare opportunities for AGAMREE? I imagine with patent and the data exclusivity and factors like that, you can't wait forever to identify those and more visibly kind of outline those?

Right. So the orphan status for AGAMREE will come to an end in 2029. But because of patent restoration, we believe we go out to 2034, so we have time. However, that doesn't mean we're going slow. So we're right now doing a Phase I study and the Phase I study is looking at 2 elements: The first element is what's the dose equivalence between prednisone and EMFLAZA and AGAMREE. So we can give better guidance to practitioners should they ask. And the other one is, what's the immunosuppressive dose?

And when you think about the immunosuppressive dose, we look at that from a gene therapy standpoint because steroids are used onboard with gene therapy and you need to immunosuppress those patients prior to giving them gene therapy. But we can't actually lose.

So if we find the immunosuppressant dose, there's an opportunity to apply it to gene therapy and then ratchet it down to those doses that are effective and don't cause immunosuppression for those other non-DMD opportunities. If we can't find the immunosuppressive dose, it's an ideal product for long-term use in rare conditions. And so we're really excited. We'll be seeing those data after the first of the year. And once we see the data, we'll be making the decision about where we go. So I would say first half of the year, we'll have a decision.

Okay. And then going back to my initial question, what I didn't hear was that FIRDAPSE IP resolution was like a swing factor at all on the BD prioritization and timing of making a decision?

Coming to a positive resolution on the FIRDAPSE IP is obviously a high priority for us. We're interested in doing it. We -- there were three 1st filers. We've settled with 2 of the 3. There was fourth filer, not a first filer. We settled with that company as well. And there's one descendant left. And when you look at the situation with Teva settling [indiscernible]. Teva is obviously a sophisticated player in this space and -- in the generic space. And so with this settling, gives us further confidence even though our confidence was very, very high in our IP.

To your question about whether we're waiting for that, the answer is no. We're looking for that opportunity. We have great cash flow right now. We want to continue to build the company, and we want to look at those opportunities that can really deliver for patients and for the company.

I haven't checked recently, but do you have a trial date teed up for that one outstanding?

We do. So the -- it's Hetero and that the trial date is scheduled for March and the 30-month exclusive -- or the 30 months pause actually ends in May. So we believe that gets the judge enough time if we get to trial. But again, we're going to do the right thing for the company and for the patients and for their shareholders.

In terms of deal type size structure? Is the priority still sort of an exclusive license, maybe something that's manageable on the upfront size of dollars out such that you cannot have to take on leverage? Or is the aperture just, hey, we're going to go with the best opportunity in front of us and if that requires us to take on leverage, we'll do it.

So there's an opportunity to take on leverage. We would want to be sure that it's a slam dunk. So when you look at -- we have almost $700 million. So what could we do on the upfront plus the cash generation we have right now on a quarterly basis. So we would want to really be sure that if we were to take on leverage that this is more than likely an on-market product with a long horizon. You mentioned the FYCOMPA opportunity which was a great opportunity for us because it really diversified our income statement, gave us additional cash and enable us to do the Santhera or AGAMREE deal. If we're going to do something where we think on leverage, it has to be really spot on in -- pretty much a PTS of 100% on the commercial side. So on market with long legs.

And then I think you guys had made some comments about looking a little bit more broadly in orphan space beyond CNS. I guess I would have looked at your business model and said, when you're looking at all these different rare orphan indications, like these deals are probably not predicated on SG&A synergies because sales forces are pretty small. So I guess like the only relevance to me of that statement is just like hey, maybe we have competencies in-house for looking at stuff in the CNS space. But now we feel like perhaps we have greater competency in house to look, I don't know, in another therapeutic area specifically that rare orphan.

Sure. So from a competency standpoint, so as an example, FIRDAPSE and AGAMREE are the fourth and fifth products I've worked on in the rare space in my career. So we feel like we have great competency across the board, not just with me, obviously, but with everybody. And you look at the diverse backgrounds from a therapeutic area standpoint, we've got great skills in-house, and we can do that. I think when it comes to what's going on and how you support the business and how you support the patient, it gets back to that first statement I made about life cycle management for a product, get the patient on earlier, keep the patient on longer and optimize their dose. That infrastructure sits behind the sales organization. Obviously, the specialty pharmacy, the patient hub and the third-party logistic provider or the shipper of the drug.

So when we look at that, we believe we're really set up. And that part of the business is applicable to any orphan or rare condition. So we believe that with the expertise we have in-house, plus the expertise in supporting the patient and the provider and the payer, we think we can move well beyond CNS.

In your BD team, team, can you remind me how many people you have looking at transactions? And do you have the, I guess, the scope internally to look at assets coming out of China? Or is the China biotech engine really less focused on some of these nichier orphan drug applications that kind of fall within your sweet spot because it does seem to me like there's a lot of focus on meet better sort of treatments in big, large established categories? So I just wonder how that maybe fits within your scope of transactions that are interesting to you.

So I've worked at a number of companies that are structured a lot like Catalyst is structured. So very focused, not a huge organization. One of the things we do is we take our internal organization of 5 or 6 people who are totally committed to the business development end of it and we leverage external providers who can bring more to the table because as you know, it's episodic, even though we've evaluated 100 or more than 100 since the beginning of the year, the peaks and troughs are there as well. So we want to bring those folks in when we get really far along and get expertise, supplement the expertise that we have. China is not out of our daily wick. We like the opportunity. Any company that's not based here in the U.S. and looking to plant a flag, as I said earlier, we're interested in talking to those folks. So Asia, Europe, et cetera. So we're continuing to, and we do have the bandwidth to do it.

Okay. Well, maybe we'll shift gears to AGAMREE, which has been an important new launch for you guys. And maybe just the latest in terms of how you're sourcing patients between EMFLAZA and prednisone? Has it changed or evolved? Has it exceeded expectation? Where the patient sources are coming from?

Sure. When we initially launched -- or prior to the product, initially launched the product, we expected that we would source mostly from EMFLAZA. What we're finding out is there's an incredible dissatisfaction with the standard of care out there. So we continue to source -- I'm just going to give you the rough numbers and it [ facilitates ] to a percentage point or 2, depending on the month you're in, but 45% from EMFLAZA, 45% from prednisone and about 10% are naive.

So that continues from the launch up till now, which we think is an incredible sign that the product has great potential going forward. And there is a generic EMFLAZA on the market, and we're not affected by that. We have approvals in the 90 -- almost 90% range. So the opportunity is really to look for those new patients or keep patients on if you have a superior product profile where patients will benefit. So the sourcing continues to be exactly what it was at launch, which speaks well to the product.

In just rough terms, this market, right, because it is capped by size factors. But I think EMFLAZA achieved peak penetration of around 1/3 of the market, if I recall.

It's hard to tell because nobody reports data here because of our proprietary pharmacies. That's what we believe it was about 1/3 of the market, yes.

Okay. So call it 250-ish sales at getting 1/3 of the market. So said a different way, it could be a $750 million market for steroids assuming gene therapy, adoption didn't somehow diminish the utilization of steroids. Is that a way to think about the ceiling of this marketplace.

I think it's -- there's a couple of ways to think about it. Number one, 95% of patients have been on a steroid and only 70% are on today, and that speaks to the dissatisfaction with the therapy, even for patients who are not ambulatory, the ability to more successfully operate their chair because of improved dexterity or maintained dexterity or the ability to operate a mouse or keyboard is in their window. Both of these are the window on the world.

So we think there's an opportunity with a better steroid to actually grow the market albeit it's capped at 11,000 to 13,000 some estimates as high as 15,000 in the U.S. patients. So when you take the conservative estimates at our price, which is roughly equivalent to the price of branded EMFLAZA, we believe that the addressable market is actually above $1 billion. So if we can prove and you look back at the GUARDIAN trial, which I referenced earlier and the SUMMIT -- SUMMIT trial, which we're doing in the U.S., we can prove that those benefits do exist. We think we can get more than our -- than an unfair share of the market.

How important really is SUMMIT to getting to some of these more bullish market outlooks in your mind versus ex SUMMIT, like, I don't know, like do you feel like you still would be on a trajectory to having EMFLAZA like kind of peak sales profile?

It's a great question. And when we look at the GUARDIAN trial, it validates our faith in the SUMMIT trial. So here you have 110 patients over 5 years looking at -- versus standard of care. And you see less vertebral fractures in AGAMREE patients. You see stature, normal stature. So long-term steroids and adolescents can inhibit and you see fewer cataracts. So if you have an older patient population, clearly, they're not going to face growth issues, but you have cataract issues, bone health, et cetera. So I think SUMMIT's pretty important, and the guardian results give us a lot of faith that this is the right path to take. So to answer your question, yes, it's pretty important.

So those updates that we get from Summit are going to be periodic, right, over time and you do more follow-up and so the first update will be some time. Can you remind me is that next year?

We don't -- we haven't said yes. So we're still enrolling. We're looking for 250 patients across about 30 or 35 sites. And we really -- we need to have those patients on for a significant enough period of time to see the benefit of the bone or see the benefit of the cataracts. So we believe there might be other benefits as well, such as behavior. That will see pretty quickly if it's there. So we're looking for that -- so we'll give updates as we continue to successfully enroll patients. And I want to focus that these are, in fact, patients, not subjects. These are patients who are on commercial drug.

So they get a significant number of enhanced testing, and they got a significant benefit from participating. So we think it's a pretty important trial and focus a lot of effort there.

Okay. And then how has the payer coverage sort of evolved? Do you feel like going into 2026 that this single generic step edit was going to be the more broadly applied kind of coverage policy type?

I'm sorry, could you repeat the question, Jason? I'm sorry, I had a hard time hearing you.

All right. Just thinking about sort of the payer coverage, right, going into 2026, do you think that, that's going to be primarily this single generic step that's been, I guess, in my mind, the predominant type of policy is step through one generic only, not two?

Yes. So right now, the majority of patients are on a generic -- they're on generic prednisone. And those that are on EMFLAZA are not going to go back. It doesn't make sense for an insurance company to say go back to generic EMFLAZA, if it didn't work. So we think they'll -- there are 2 elements to this. Number one is that we're getting -- almost as I said, 85%, almost 90% of the prescriptions approved. And when we first launched, it was probably about 30 days from the time we submitted the prescription, so it was approved. So we give those patients bridge drug, right, to make sure that they get on therapy as fast as possible. Now it's down to less than 2 weeks. So I would see that continuing to evolve as more payers get more comfortable with the profile of the product and the use of the product.

Okay. And then is there any -- you can speak to more real time in terms of interplay with gene therapy rollout, how that's impacting steroid utilization, if at all?

Yes. So I would say that the market itself -- so remember that steroids are a foundational therapy. It's the drug you get on first before you get on anything else. Patients that are on steroid therapy walk 2, 2.5, sometimes 3 years longer than patients who don't go on do steroids. So we're not competing with gene therapy, but the choppiness of this water because of all the things that have happened, the performance of the product in the face of that choppiness, I think, is outstanding. So we're looking forward to settle out. We would love for -- as everybody would for there to be no more challenges with gene therapy and then sort of get on a steady state.

And so we've had, what we call, a queuing effect. So patients that are on a steroid are less likely to change if they're waiting for gene therapy. So it gets -- almost creates a line, if you will. The challenge with this scenario in the marketplace is that we saw the label expand for gene therapy, then we saw it contract. And so there's just a lot of confusion. And as that settles down, it gives us a stronger share of voice in the market because we can actually talk about the benefits of therapy with AGAMREE. So we're hoping it stays steady state.

How are you guys thinking about preannouncing around the time of JPMorgan started the new year for your business, do you feel like you have enough kind of visibility into these components of the business that this would make sense for you guys to sort of preannounce revenue for your business lines?

So we may do it at a high level. I think what we want to do is we want to take a look at the markets and be sure that we have a really good understanding. Obviously, at this point in the year, your forecasting is complete, but you just want to be as appropriately cautious as possible. So last year, we announced that our earnings call for the end of the year earnings call and that's our plan for right now. So we would want to make sure that we have a really good understanding of how the markets are trending when we do, in fact, give guidance.

Okay. Anything else that we haven't hit on with respect to this topic. I don't know if you've actually even sort of shared any of the orphan indication opportunities that are potentially of interest to you or potentially any sort of directional sizing indicators for what those expansionary opportunities could be as AGAMREE follow-on indications, if you can't be that specific at the moment on like maybe what you're looking at?

Yes. Just at a high level, we would look for those rare conditions that require long-term steroid use and have the largest patient populations where a change in steroids is meaningful to the patient or to the provider, hopefully, to both. But we're not in a position right now. We'd really like to see those data from the Phase I to have a better understanding in that way, I think we can go forward with immunity.

I mean, with the pricing paradigm that you have with this, I imagine that does the utilization need to be in sensitive populations like pediatric or I'd imagine like there are very large market opportunities where steroids are used, right? But trying to narrow it down. Is some of this population sensitivity, you probably want to stay in rare orphan, right? You want a sensitive population that you're going in the pricing structure. So I'm just kind of curious if I'm thinking about that the right way?

You are. You are. So rare orphan is where we want to focus and where the patient can gain the best benefit. So as we discussed earlier in this call, older patients won't have the benefit. They don't have the benefit for stature. So if there's an opportunity to provide a pediatric patient with that opportunity to grow like their peers, that's a great opportunity for us.

Older patients are going to be worried about bone strength. You just reduced the vertebral fractures for long-term steroid patient. That's critically important cataracts, that's critically important, too. So the ancillary costs that you would experience or maybe avoid if the drug actually does have the profile, we believe it has, I think could create tremendous value in these rare and orphan markets.

Okay. Makes sense. Maybe shifting gears to FIRDAPSE and I actually have just a couple of mechanical questions. As we think about just the different scenarios around IP litigation, I just want to make sure I got this right. So you have a sort of tail terminal patent that covers certain aspects of the composition, whether they do or do not have sort of this impurity that you've identified in your patent. And I believe what I understand historically, it's very difficult, did not have that impurity in the product, right? And that is something that's patented. So is that sort of the anchor patent as you kind of look at this? And what underpins the confidence kind of going into whenever a patent litigation could...

So we're not going to get to too many specifics. So we have 2 patent families here. And for somebody to actually win, they would have to win on every single patent. So any patent they violate or if the defendant is found to be in violation of it, that's a win for us. So our patent estate is robust. And so we believe our patents are robust, and we're going to defend them to the very end. Obviously, we want to do what's right for the business and get back to the business of the business, but not at the price of sacrificing future value. So -- and we believe that the Teva and the Lupin agreements are very fine agreements for all parties. And so we like that structure.

Understood. And you have a BioMarin royalty. That rolls this year if I understand correctly?

So as we talked about in our Q3 -- quarter -- third quarter 10-Q, the BioMarin royalty on FIRDAPSE ends in January of '26, And based on the midpoint guidance, the vast majority of which is U.S., our royalty obligation is about $30 million to $35 million. And all this is in our Q, I believe, footnote 13. And so that reduction in royalty goes straight to pretax income and will contribute to operating leverage. So that's a big win for the company.

Okay. Pavan has got some questions.

Yes. I guess just in terms of like the uptake in FIRDAPSE lens patients, I think you've previously estimated that there are upwards of 900 tumor lens patients, and only 3%, 4% of those are ever diagnosed. So this is the growth segment of the LEMS patient population. Maybe if you can update us on the traction that you're seeing in those thoracic oncology centers and whether the diagnostic code implementation has improved materially to aid in the identification of those patients.

Sure. So when we think about the cancer associated LEMS part of it, remember, there are 2 parts of the business, and it's split about 50-50 patients. There's the idiopathic or non-cancer LEMS and the cancer LEMS. And so the traction we're experiencing right now really goes back to -- goes back actually a couple of years when we were making the change on the NCCN guidelines and recommending change. But the success we've had in this year of streamlining the VGCC test. So there's a test that's very indicative of whether or not a patient would actually have LEMS.

So we got that testing centralized and now there's blood pickup in the office where the patient is -- previously the patient ought to go out, come in. So the traction we're getting is building on these first steps. Then we got the NCCN guidelines in July, which is a huge win for patients and a huge win for the company. And now we're working with these large high-control oncology practices to embed the change in NCCN guideline into the care pathway.

This is a really critical step. So looking again, high volumes, high control. And then from there, working with these accounts to then help train the physician that when they see a small cell lung cancer patient, they should, in fact, do a VGCC test first. They may partner with the neurologist for a definitive diagnosis or they may feel comfortable doing it themselves. The recommendation is to work with a neurologist. The NCCN guideline recommendation is to work with the neurologist. So the traction we're getting is the receptivity right now from the oncology practices that are saying, "yes, this looks very interesting."

We don't anticipate significant impact for the cancer LEMS in the first half of next year because there are many of these practices. It takes a significant amount of effort to get in there and actually help them understand the change in NCCN guidelines and then what they have to do for the change in their care pathways. We have dedicated resources in this field that are looking at this and working with these accounts. And I think the traction right now is quite positive.

And can you remind us like what's the pool of patients that you've identified for treatment with FIRDAPSE And how is the composition of that pool evolved over time in terms of the split of idiopathic versus oncology LEMS patients.

So the pool we keep is about 500, and we use data and machine learning to look for those patients who have gone through a certain series of events in their diagnostic journey. And then we hyper-prioritize to those who were furthest along. And so we're seeing about 50% of our patients in any quarter come from that pool, whether they're idiopathic or they're cancer. Your question is really difficult to answer because on the split, it's typically about -- at any point in time, it's typically about 80-20 with 80% being idiopathic.

That's why we think there's such a nice upside here. The reason why it's difficult to answer when they come in is because LEMS is sometimes the first indication that a patient has small cell lung cancer. So they come in with idiopathic lens, but over time, they end up with the diagnosis of small cell lung cancer.

So it's not as clean as we would really like. But we are seeing, as an example, the work that we've done in increasing VGCC testing of 9% per quarter quarter-on-quarter, which is really phenomenal. We think that's really exciting. And we're not really that embedded into the care pathways yet. So we think there's a lot of upside here.

Yes. And then I guess like another layer of this is the diagnosis of LEMS with myasthenia gravis. And I think you've noted that about half of your patients have come from this pipeline of pool of patient opportunity. So maybe if you can talk about how the rate of converting these misdiagnosed MG patients to LEMS diagnosis has accelerated over the last 12 months? And how much work is there left to be done here?

So this side of the business, the idiopathic side of the FIRDAPSE business is what we would call a prospecting business. You have to find these patients. They're hard to find. The average physician sees one in their entire career. So it's challenging. So we're following a similar path that we talked about on the oncology side. So obviously, we have this pool of patients we track and through machine learning, we hyper-prioritize. And then there are RAMs, our regional account managers, going and prioritize again.

Looking at the opportunity for growth, we think myasthenia gravis and all the great therapies that are out there for myasthenia gravis are excellent. And actually, this is a great investment that we actually don't have to make because there's a lot of DTC out there.

And many of these patients will end up with what is called a seronegative myasthenia gravis test. And so we're doing the same thing with the national labs that we did on the cancer side, which is trying to get them to embed reflex testing. So if a patient comes up with seronegative for myasthenia gravis, so there's no -- there are no antibodies there. Reflex test them for VGCC, and we're getting a lot of traction there as well.

So we believe that this will become potentially, if successful, less of a prospecting market and more concentrated in those high myasthenia gravis accounts. So that's what we're looking for. And then obviously, to grow the awareness through education -- targeted education with those accounts. And that's our plan on both sides of the business. We think both have great growth potential and so we're going to work both ends of the market.

Yes. I guess hitting on FYCOMPA really quickly. That's sort of an asset that's undergoing generic competition. So maybe if you can talk about the slope of that erosion. I know it's been a little less than initially expected. So is the brand stickiness holding up in 4Q as well? And how you expect that through 2026 onward?

Sure. So it's a little early to tell about the fourth quarter. And let's just go back over and talk about the timeline here. So on the solid dose, there's 2 formulations of FYCOMPA. One is solid dose and the other is suspension -- oral suspension. What we've seen with the solid dose is there were 2 first filers in this market, and they could have come in, in late May. Only one came in and came in, in late June.

So we got a nice maintenance of our business there. And the erosion, because only one is there, and the player, again, it goes back to Teva is the most sophisticated player in this market. Teva came in with a really good price, market minus. So they looked at the market and said we're coming in with a 17% discount.

There were other products that went generic products that went generic in the epilepsy space around the same time that we did, but they had multiple players coming in. We did not experience that. So there's a lift from that. There is the stickiness that you always see with epilepsy patients. They want to have a drug that they can be sure is going to protect them from seizure. But we are also -- we know that last week, 2 additional generics, solid dose, were approved. They are not yet on the market as far as we can tell.

So they're not taking away from our ex-factory sales. And here we are on the 8th of December. So we see if we can finish the year strong, and we took up guidance on this product. However, the oral suspension, we expect a single first filer to come in or to be approved in the middle of December. And prior to the incursion of generics back in June, oral suspension was 15% of our business. So we're seeing a shift, and we expect to see a shift. So product has done really, really well so far. But we all know, successive brands is inversely proportional to the number of generic players and now we're going to be experiencing more generic players. What we're looking for is to be able to cover that loss of sales with growth on our other products. And that's what we're really pointing toward.

And then in terms of like a higher level question with capital allocation. So the Board recently authorized a $200 million share repurchase program. So should investors be thinking about this as a signal that maybe M&A valuations are too high? Or is this just a signal that the balance sheet is really strong and then you have flexibility to do both buybacks and business development deals as you see fit at a given time? And how should we think about how you're thinking about the balance of capital towards buyback versus BD in a given year?

Yes. So excellent question. So we don't like our share price. And my joke is if you ever met a CEO did, you got the wrong CEO. So -- and we think we have the opportunity to add -- contribute value back to shareholders without hurting our opportunities in business development. So on a non-GAAP -- on a GAAP basis, we did 52 and change in the third quarter. And so we believe we can continue to do that. And this share repurchase is over 15 months. So we have a loaded -- we are loaded. We are ready to go on business development.

This share repurchase will not affect our ability. Again, we have no funded debt. So if there was an opportunity -- as Jason and I were talking about earlier, if there's an opportunity to leverage on a great deal we would take that opportunity. So we think this is a good way to return some value to shareholders while executing our business.

And then if I can go back to AGAMREE with the label expansion opportunities like, I think this is something that's come up a bunch of times because it has an upside lever to a product right now in terms of The Street's forecast? And just trying to narrow down like what you think are the most appealing or attractive opportunities. Like can you help us think through the framework that you're using for that -- those rare conditions.

So when we think about the opportunity, we want those rare conditions. So it's got to be rare. We want it to be a long term and where there's a high need for steroid? If's it's episodic, it's less interesting to us, if it's continuous. And we have that profile that you see in the GUARDIAN trial where bone strength is there or growth or stature, avoids of cataracts, et cetera, better behavior, we like those opportunities. So again, just focusing on the opportunity to create the greatest value for patients as quickly as possible. And we'll have a better handle on that after the new year to be able to pick the right target.

And then in terms of like the IP outcome that could come post a potential bench trial that's scheduled in March 2026 for FIRDAPSE, like how should we be thinking about the bull-bear scenario here, like assuming a worst-case scenario -- or FIRDAPSE goes LOE earlier than we expected, like how would -- how should investors be thinking about such a scenario and the risk profile of the company in such a scenario?

We really believe in our IP, and we're going to do everything we can to defend it. We think, again, settling with 3 out of the 4 defendants so far, I think it demonstrates a solid position for us. One of the most interesting things about the FIRDAPSE market is we have already faced competition that was significantly price reduced in Ruzurgi. And Ruzurgi out there at half the price of FIRDAPSE. And the interesting thing is when we talked -- we just talked about the value of what's behind the front of the house. So the value is getting the patient on to the drug, keeping them on and optimizing the dose for their benefit, those systems, those -- that infrastructure is so valuable to a company like ours, it can make a huge difference. And Ruzurgi coming out at half the price, we actually didn't feel any significant dent to our business.

So we think that we have a pretty good system that can defend us going forward. But again, we have strong belief in our IP and we look forward to resolving that in the most appropriate way possible for the benefit of the company and patients.

Rich, I have a couple of follow-ups. So first, just on, like your rare orphan portfolio, how should we think about pricing power going forward? I know that these are expensive medications. And is there a certain cap 2% to 3%, given, I don't know, with plans, these can be needle moving? Or are they so small? Maybe there's a little more latitude with pricing power. And I know that there's some Medicaid exposure here, right, so there's price penalties. So just maybe help us think through the framework there?

So we look at the opportunity if there is an opportunity to increase price to cover our increased cost or anything like that, we look at that and we measure against what the penalties, Jason, you talked about. And we're not going to do anything egregious that you can look at a company that's trying to drive the top line, but ends up with less on the bottom line. And that's actually quite possible in these markets where you can actually take a price increase that's quite high and end up making less money. We don't like that. So we don't like that, so we don't do that.

So we're always going to look for that balance of what we think is the right thing to do based on the services that we provide, the value that the drug provides and the market conditions, whether it's PPI urban or whatever is going on. So we're always going to judge that in a multifactorial way, but the pricing power of old is just not there anymore 10, 15 years ago.

Yes. And then with cancer LEMS, do you -- my understanding in past discussions with you guys, this is not like a buy-and-bill opportunity with these community oncs such that like this could be a money-making endeavor for community oncologists, which could spur faster utilization. I know that some people have asked me about that, but I feel like in the past, you guys have said you don't expect this to be a buy-and-bill product with these oncologists.

So it could be actually. So oncologists -- medical oncologists cover the cost of their practice through the reimbursement around medication, whether it's injected, infused or oral. Many of these larger practices do, in fact, have in-house pharmacies that can distribute. And quite frankly, if these patients are coming in on every 3-week or 4-week basis for their cancer therapy, having that care team centralized in one spot becomes critically important for the outcome.

One of the things important for the outcome. One of the things we didn't talk about is we think there's about 900 -- initially 900 new patients on the cancer LEMS side. Those cancer LEMS patients, they have small -- typically small cell lung cancer. A small cell lung cancer patient lives about 8 or 9 months. Nobody knows why, but a patient with small cell lung cancer LEMS lives 17 months.

And in our discussions with oncologists, they've said, well, I assume the patient only live 8 months or 9 months. Now I'm seeing them 1.5 years, I think about them differently. And I want the quality of life to be higher. And you know with LEMS, Lambert-Eaton myasthenic syndrome. As the disease progresses, the patients lose the ability to walk, ambulate or certain control of extremities.

So if they can improve the physician can improve the quality of life of the patient, that's something that quality of life of the patient, that's something they're interested in. And we do believe that some of the accounts will actually use this -- the opportunity to distribute FIRDAPSE in-house as a way to offset costs of their practice, overhead, malpractice insurance, et cetera.

Yes. Okay. That's helpful to know. I appreciate your time, Rich, as always, and this is really helpful. Thanks for the participation.

Thank you so much. I really appreciate the opportunity, and your time. Thank you.

Thank you.

Thanks very much. Take care.

Today, it's my pleasure to be hosting Catalyst Pharmaceuticals for a fireside chat at Citi's Global Healthcare Conference. I'm joined today by CEO, Rich Daly; CFO, Mike Kalb; and Chief Commercial Officer, Jeff Del Carmen. Thank you so much for being here today, Rich, Mike and Steve.

To start, maybe, Rich, you could just like level set for us for those that might not be familiar that are listening in, can you just give us an overview of Catalyst and each of your product candidates?

Sam, thanks for the opportunity to be here. We really appreciate it. Happy to go over the products and be joined by my colleagues here and to talk about the scope and span of the business. So Catalyst -- the focus of Catalyst is orphan and rare diseases. We have 3 products. We have FIRDAPSE, which has been on the market since 2019, and it's for Lambert-Eaton myasthenic syndrome, both idiopathic and cancer associated, and we'll talk about that in a little bit.

We also have a drug -- our second drug was FYCOMPA for epilepsy. While not a rare disease epilepsy, actually, the acquisition of FYCOMPA actually served the company quite well, and we can go into some detail around that. The solid dose of FYCOMPA actually lost its patent protection back in May, late May, and we've seen some incursion into the business, but the products has overperformed our expectations, and we're really happy about that.

The last product is AGAMREE for the treatment of Duchenne muscular dystrophy. And it's a steroid, but we believe it has potential to actually benefit patients in a way that other steroids or standard of care steroids can't. And some recent data that's come out from our colleagues in Europe really demonstrate the potential benefits of that, and we look forward to talking about that.

We are a buy-and-build company. We don't do basic research, although we did do the research that supported the approval of FIRDAPSE. We are looking to acquire products that are significantly derisked, so products that are on the market or near the market with a decent runway of opportunity for continued commercialization. That's our focus right now, but -- and we'll talk about this, I'm sure, during the course of this discussion. We are considering our options as we move back into the pipeline, further into the pipeline for continued development. So products that might be a little bit more on the regulatory risk side, or might be in late-stage development proof of concept is well understood. And we believe our very strong cash position, balance sheet and the cash flows that we have can continue to support our strategy of buying products, but also working our way back into the portfolio and then also supporting our products and life cycle management as well.

Thank you, Rich. That was a really good overview. Lots to go off of there. Maybe just let's just talk about the overall strategy for the commercial engine first. As you think about the next 12 months, how do you expect that to evolve in terms of your priorities as you commercialize your 3 products?

I think first and foremost, one of the things we want to do is we want to make sure that we cover the gap for FYCOMPA. So as I said, the product is overperforming, and we're thrilled with that. But we do know that one product has been approved for the -- as a generic. And we know that 2 others were recently approved, but not yet on the market. So we expect to lose price and volume there, and we've said that consistently as we talk about FYCOMPA. But again, we're overperforming. So our focus has been to minimize that impact while optimizing and maximizing the impact on -- for the patients with Lambert-Eaton myasthenic syndrome with FIRDAPSE and those with Duchenne's muscular dystrophy with AGAMREE. So we really have taken, I think, an appropriately aggressive approach to the market.

Let's talk about FIRDAPSE with LEMS. The market is divided into 2 parts, idiopathic or non-cancer LEMS and then cancer-associated LEMS. And with myasthenia gravis and the number of products that are -- have been approved in gMG, we're seeing on the idiopathic side, a really great opportunity to tail -- get a tailwind from that. We did -- and Jeff can talk about that more in detail.

On the cancer-associated LEMS, we've -- Jeff and the team have done a great job in getting frictionless testing done and for the patients because there is a test for VGCC test. And then changing the NCCN guidelines. So this is a really important thing for us as we take our next step. So we have a 4-step process, the treatment -- I'm sorry, the testing with VGCC and the National Labs frictionless testing, changing the NCCN guidelines and then working on improving care pathways for patients and then really looking at the opportunity to educate the physicians within the high-volume, high-control oncology practices.

The last part is looking at AGAMREE. AGAMREE is an incredible product. We really, really like it. We think there's potential beyond DMD in rare conditions that require steroid. So we're conducting a study right now to see if we can determine the immunosuppressive dose of the drug. Regardless of how that turns out, it's positive for us. So if we can't find the immunosuppressive drug, this is a really good thing for patients who are on long-term steroids in other rare conditions. If we do find it, then we obviously can figure out the best way to work with those patients, but also work in gene therapy as well because patients who are going to go gene therapy require immunosuppression. So we almost can't lose in this opportunity. We should see those data early in the year -- later this year or early in the year next year.

And that's from the SUMMIT study that you're.

No. So this is a Phase I study. Great question. I'm sorry I didn't make that clear. It's from a Phase I study where we're doing 2 parts. First, we're looking for the dose equivalency between -- I'm sorry, Emflaza, prednisone and AGAMREE. So when a patient moves, 45% of our patients come from prednisone, 45% of our patients in the DMD market come from Emflaza. But there's no information on the label about the appropriate dose to move them to. So we think that's really important.

The second thing in this Phase I study is looking at this immunosuppression I talked about and figuring out how best the product can be used beyond DMD. And we'll be looking at that once these data come out and making a determination about the life cycle path for the product because we think it has potential given some of the profiles we've seen some work that's from work that's been done in Europe. So on the SUMMIT study, that's a 5-year 250 patient. And I mean patient, not subject because it is commercial drug, we'll be tracking and looking for the benefits of AGAMREE over standard of care steroids.

And the benefits that we believe are out there are better behavior, less aggression, potentially better growth or stature, better bone health, fewer cataracts and then also potential for cardiovascular benefit. Cardiovascular benefit is the #1 cause of death for patients with DMD. Recently, our European colleagues at Santhera came out with a high-level summary of the GUARDIAN study. We can talk about that a little bit more. They haven't published much of the data yet. They're waiting for some other opportunities in professional meetings, which 111 patients over 5 years, and it showed a benefit on stature. So normal stature for patients on AGAMREE, bone health, excellent bone health and then fewer cataracts. So this, we believe, is a really good indication that our faith in SUMMIT is well placed. And so we're excited to see more of that data come out at some of these scientific meetings that are coming up.

Got it. Okay. That's helpful clarification. So for the Phase I, when you say additional indications outside of DMD, are we staying in the neuromuscular space? Or could we expand beyond that? I mean there are plenty of diseases where steroids are still make a foundation of the treatment paradigm. So how large is the spread for AGAMREE potentially in your view?

It's too early to tell the spread, but we're looking at anything in the rare space. So as long as it's rare, as you know, Sam, from the conversations we've had and the statements we've made, we're therapeutically area agnostic because we believe in the rare space, the infrastructure that we have where we support the patient, help the patient get on drug and importantly, stay on drug and then optimize their therapy, we believe that infrastructure is incredibly strong and can be -- is applicable to anything in the rare space. So if it were to go beyond CNS, we'd be comfortable with that.

Got it. Okay. And so then when you think about AGAMREE and its penetration into the market, you have some of this data that you just mentioned from Santhera. You have the SUMMIT study that you'll be able to rely on when that data starts to roll out. I guess how should we anticipate the launch to grow over time? Like what does that curve look like potentially?

Okay. I'm going to turn it to Jeff, so he can give you some background and some insight. Jeff?

Sure. So Sam, as we've discussed, we're very pleased with the launch so far. And we've seen broad adoption. There are 100 COEs, give or take a few, for DMD. And we have about 95% penetration within those 95 -- or those 100 COEs. By penetration, I mean at least one enrollment coming from each of those COEs. And then the top 45 COEs make up about 80% of the steroid business for DMD, and we have 100% penetration in those accounts. So we've seen broad adoption.

Now what we're trying to do is get deeper adoption within each account. So that's where we focus. And that's what we anticipate seeing over the coming year plus down the road. And that's how we expect the launch to go is to continue to deepen that penetration.

Right. And in terms of the data that we'll see out of SUMMIT over time is the behavioral piece, I think you've said in the past is likely to be one of the first data readouts. I guess when you have that in hand, how does that help to just drive the launch further in terms of the breadth you're looking for or the depth, excuse me?

So I'll take a first crack at that. So when we see that data for the first time, it won't be in our label. So from a commercial viewpoint, we won't be able to do anything with it because we are a compliant company. But obviously, those things are -- that information gets out through medical meetings and through publications. And we're going to look to leverage our voice in the market outside of the commercial space to be sure that physicians have the best information and can make the best decisions along with their patients and the caregivers in the DMD space. But there is potential for a label change, but obviously, it's never guaranteed. And so we're going to look to make sure that there's awareness about that, but again, in a totally compliant manner.

We believe that the behavioral data will be helpful. However, that's very easily seen today by physicians that are utilizing it because you can see it within a week and how patients are -- the boys are reacting in school and how they're behaving. So those types of things. It's the other data coming from the SUMMIT that will be extremely helpful if positive.

I see. Okay. And how should we think about just the overall peak market opportunity for AGAMREE -- now that you've had almost 2 years launched coming up, I believe. I guess what have you learned? And what should we think about the peak opportunity of this drug?

So we believe this is -- at today's prices, we believe this is about a $1.4 billion opportunity in the total addressable market. And that takes into account 95% of patients have received a steroid, but only 70% are currently getting a steroid. So without any incremental growth in the treated population, the total addressable market is about $1.2 billion. All else being equal, if we're just as good as the other players, we'll get 25%.

If we can show and demonstrate superiority in certain elements, whether it's behavior, stature, bone health, cataracts, cardiovascular over time, one would believe we should get an unfair share because we would have data that demonstrates that the product is better. So a 25% share, $300 million, but we haven't given any long-term forecast. We're just talking hypothetically. But if we can continue to demonstrate what we saw in GUARDIAN, we think there's significant potential here because these patients are struggling and they're looking for different options.

Jeff, I don't know if you want to make any comments.

Nothing to add. Well said.

Got it. No, that's super helpful context. And then I think you mentioned this as well, Rich, about the potential immunosuppression for a gene therapy as well. I'm wondering like where does that stand? And where could we maybe see an impact from that?

So it depends if we find it, right?

Sure.

So there's a potential we won't. And if that happens, then we'll obviously not be a part of that market. If it's there and we have some of the other benefits that the physicians and patients and caregivers can see, then I think we can participate in that part of the market. But it will be a long time before that's actually in our label. So again, it's -- this is a very, very tight community, the DMD community, the patients, the caregivers, usually their parents, typically the mom and the physicians are very, very tight.

So again, we can act in a compliant manner and get the word out. And then obviously, it's up to the physician in conjunction with the patient and the caregiver to make the decision on what they want to do. But again, I want to just focus -- I really believe that regardless of how that turns out, we can go in either direction. And so we're pretty excited about those data coming out.

Yes, absolutely. I mean it sounds like the base business that you have right now is quite strong, and there's a number of tailwinds that we could see materialize over the next, call it, 12 to 24 months potentially, at least data-wise, maybe if not future label change.

Agreed, yes.

Yes. No, that's a really good position for AGAMREE. Okay. So why don't we go ahead and switch over to FIRDAPSE then? I mean we started -- you gave us a really good overview of that one as well. Why don't we start with the oncology segment, if you don't mind. I'm wondering all of your efforts, I think you laid out what they are quite clearly already. But how have those been received by physicians? How is that education progressing, the efforts that you're making?

Great. Jeff, do you want to take that?

So the education is coming from multiple fronts, and we're focused obviously on thoracic oncologists, but really the community-based oncologists. So we are going to conferences. We have 2 MSLs fully dedicated to cancer-associated LEMS. We just launched an unbranded website, cancerassociatedlems.com that's providing this information. We have educational efforts within these high-volume, high-control GPOs out there so that when physicians have a newly diagnosed small cell lung cancer patient, they receive education from the company, from their GPO that kind of provides updates from the NCCN guidelines on what their approach should be. So those types of things, and it's being very, very well received.

Rich, I don't know if you want to talk about one of the meetings you just had.

So changing the care pathways, it's the old saw about HMOs. If you've seen one HMO, you've seen HMO. Changing the care pathways across this continuum of accounts is on a one-off basis. So we're focused on the high volume, high control, just like you would with the managed care, where the physicians within the structure of the company, the oncologists are judged by how closely they follow the care pathway.

Recently, we've been reaching out. I worked in oncology for 15 years. And so we reach out to folks we know and say, look, the NCCN guidelines have changed, have you updated? And I had a conversation with the leader of an oncology account of about 50 oncologists. And he said, okay, we'll make that change. And about 10 minutes later in the call, I said, I just want to remind you, you need to make that change. And his response was, I made it while we're on the call, move on, which was great.

That's the exception, though. We don't expect that to happen because these are very large organizations with varying levels of control and size within their own ones. So McKesson is the largest. And so we plan to work top down through McKesson and bottom up through the plans as well through the -- not the plans, but the accounts themselves and get the education out there. And then some of the best investment we can make when they change that care pathway is to be working on education so that the physician knows if I have a small cell lung cancer patient and the test panel comes up, check VGCC because you want to know.

And as we know from our experience, often the first sign of small cell lung cancer, not often, sometimes the first sign of small cell lung cancer is Lambert-Eaton myasthenic syndrome. So we want to be watching that as well. And the physicians have shown a great deal of comfort in being able to not only diagnose, but also actually treat the patient in the oncology setting. And that makes sense. They'll be back in on a regular basis. So again, we're seeing lots of progress. But we don't expect any impact, any significant impact before probably midyear of next year because it just takes time to get through all of these. And we're talking about 3% of 15,000 or 30,000 patients who have small cell lung cancer. And because of the life expectancy of those patients, there are at any time about 900 patients that are out there. So we think this is -- has significant potential for growth for the company.

A couple of things to add. This is a significant opportunity, like Rich mentioned. And 90% of the cancer-associated LEMS patients are undiagnosed today. So that's why all our focus right now is educating physicians on this and screening so that we can help these patients get diagnosed and help these patients out. So that's one of the reasons why we're so focused on screening.

No, that makes a lot of sense and the opportunity is there for sure. When you think about that inflection point, I think you said mid-'26, you're obviously working to change a bunch of care pathways. Does that all -- I guess, do you get enough momentum at that point? Is that when you expect the education to hit sort of like, I guess, an upward curve where the diagnosis rate should start increasing? And is that just like a critical mass of these care pathways changed in your favor?

So yes. When you think about our business, so Jeff said, and then we have to keep these numbers very clear, less than 10% of these patients have been diagnosed. Now let's talk about our patient pool, the number of the patients that are being treated with FIRDAPSE, less than -- around 20% of our patient pool. So getting behavior changes takes time and continued effort on education. So we're seeing a lot of positive traction for the care pathway change, but then we need to invest, and we've planned it out for next year in the education by making sure that physicians are aware.

Once -- what we've seen, not broadly, but when we talk to physicians, they say, these patients live on average about 9 months. A patient with Lambert-Eaton myasthenic syndrome and cancer lives about 17 months. And that's a lights on moment for the oncologist because they think, wow, this patient is actually going to live about twice as long as I thought. So quality of life really does matter. What can I do to help that patient? So getting that word out, getting the education just takes time and effort, and we are well positioned to do that as we go forward.

Yes. That makes sense. And I guess just can you talk a little bit about that strategy, about how many of the accounts -- you mentioned McKesson being one of the large ones. Are there any others that are on your list? I'm curious if you have like a couple of waves of targets. And then just how are you thinking about, I guess, that as it progresses? Like how do you progress through wave 1, wave 2, wave 3, if they exist?

So I think Florida Oncology is probably the largest oncology practice in the U.S., single practice. Then you have Tennessee Oncology, Texas Oncology. And all of those are part of one of the GPOs. So again, working the top down and the bottom up with the field resources that Jeff has talked about from the MSLs to the national account directors. Again, I think to your point and to the word you used before, momentum, there will come a point where we'll hit that momentum and there'll be more tests done. So the strategy is really to approach it from both sides of the business and to try and help grow. Jeff?

Yes. Just to add to it, about the 3 largest GPOs make up about 80%, have control over a top 80% of the community oncologists. So it's to our benefit if we focus on those big GPOs. And so that's how we anticipate getting traction sooner. And as soon as we can get traction within U.S. oncology with Florida Oncology, then we go to Texas Oncology, then you can start building momentum. But that's the fastest way to be able to get share of voice with thoracic oncologists.

Is there -- or would you consider sharing sort of, I guess, testing rates and how they have increased over time as like a KPI that maybe we could track for the oncology segment?

Sure. So it's very hard to tease out oncology -- oncologists that have initiated a test or a neurologist that has initiated a test. But what we have seen is a significant increase in the number of VGCC antibody tests that have been done. over the last 1.5 years, quarter-on-quarter, there's at least a 9% growth each quarter. So we're seeing that growth come along. It coincides with the amount of education that we're putting out there, too. So we strongly believe our efforts are making a difference, and we expect that to grow continuously as we move on.

Got it. That's super helpful. And then, just talking about the idiopathic opportunity, Rich, you said that there's still a large amount of opportunity there. Maybe just talk about how you're going to further penetrate that segment over time and what dynamics we should be aware of for that?

Sure. I'll turn it to Jeff, but it's pretty similar in approach to a systematic stepwise approach to the market and leveraging the increased noise around general myasthenia gravis because that's the most frequently misdiagnosed condition for somebody who has idiopathic Lambert-Eaton myasthenic syndrome. Jeff?

We're very excited about the opportunity that still remains with the idiopathic side. When we look at it, I mentioned that we always have about 500 or so these pool of patients, we call them pipeline patients that are somewhere in their diagnostic journey to LEMS, but we know -- strongly believe they're LEMS patients. So about half of our enrollments each month come from these pool of patients, so we know them. So what we need to do is continually find new patient leads to backfill ones that are converted on therapy. And we have been doing this for years. And we always find new sources to backfill. So not only do we leverage the sources that we have, but we find new sources to also add into this pool of patients.

And we are very confident that based on these new sources and the pool of patients that we have, the growth will continue. One of the reasons is because of what Rich mentioned, if we can get all these patients tested for MG and then they're negative for AChR, then we need to get on a panel so that it's on the MG panel so that VGCC antibodies are there, then you can rule out MG and then, oh, by the way, they have deposited for VGCC, they have LEMS instead of excluding other things first and prolonging that diagnostic journey. So along with that increased rate of testing for those patients. Now we get new leads coming in because we get that data from national laboratories.

So Sam, I want to build on that, too, because I think Jeff's answer is right on point. When we think about what's going on in the general myasthenia gravis market, the amount of noise direct-to-consumer is going to increase the diagnosis rate for gMG, no doubt about it. But it's also going to bring in a lot of patients who will initially get misdiagnosed with gMG. And therefore, I think our opportunity increases.

One of the ways I like to describe it is the best money -- their DTC campaign is the best money we never spent because it will help raise that up. And we want to be sure they have the physician as a solution. It will take time to get both of those AChR and the VGCC done at the same time, but initially, it will probably be a reflex test. Do you have seronegative AChR, let me go down to the next logical thing. And again, that's an educational opportunity.

There's not as much control in the idiopathic market as there is in the cancer-associated LEMS market. So it's still very much a prospecting business and really using the data that Jeff talked about to identify that patient who's most likely to be a LEMS patient and continuing to leverage that data set that's out there and do education and change the testing. So it looks very similar to the cancer-associated LEMS path, but it's a much more labor-intensive opportunity.

Yes. I'll add to, it's with these leads, we have the most data in the world for LEMS. And we need to better understand the leads that have become patients on therapy and how those patients are doing. So we're benefiting these patients. But the more efficient we can be with the leads we have, then the faster we can help these patients. And we've become more efficient because we've learned more. And that's why we're now pointing the RAMs, our field force in the right direction within it. We score the leads and we'll say this one is a higher priority than this lead over here. So that's why we're seeing an acceleration, and we've been able to sustain this growth.

Yes. I was going to ask how long does a patient typically stay in your patient pool? Is there an average? Or are there, to your point, a couple of different types of patients that move through at different rates?

Yes. So Sam, it's all over the spectrum. And we have seen some patients that unfortunately, they will go on some non-FDA-approved treatments or not just get there based on education, it's the first time the physician -- physicians on average, see one LEMS patient in their career.

On the idiopathic.

On the idiopathic, correct. So it's really educating them on that. But the ones that we've seen convert the fastest on to therapy are those patients that are negative AChR for generalized myasthenia gravis and positive for VGCC. So they've had both tests done, then they convert faster. And in some cases, the RAMS get the lead and within a week, that patient is on therapy. So it's very quick in some cases.

So Sam, it's so rare, right? One physician or one patient per physician. The frustration level is really, really high. And so if it takes 4 or 5 years for that patient to go from first symptom to true diagnosis and the correct diagnosis, crushing that cycle time becomes really positive for the patient. And so that's where we think the testing comes in because it looks so much like -- it's easy to do a differential diagnosis, but you just -- it's so rare. So by helping them with the test, it's not -- the test is not the end all and be all, but it's a great step forward.

Is there a dedicated MG panel that you can add VGCC testing to? Or is it just, again, individual physician-by-physician educating to remind them they should be testing for this for every potential MG patient?

It's a combination depending on the lab, too. Some labs like Quest and Mayo, you can get -- try to get on their labs. So that's what we do. And it's a long process, but we're well underway in discussions on making that happen.

Got it. Okay. Great to hear. So then maybe let's just talk about the overall opportunity. I know you've outlined it before, Rich, but I think it's helpful to maybe say it again. And I'm curious how you think about the overall LEMS and then segmenting it out between the idiopathic and the oncology opportunities.

So there's probably 3,600 to 5,400 patients out there. When we talk about the opportunity, we always talk about it in the most conservative way. We take the smallest patient population, and we take the fact that whatever our price is today, we don't -- we assume no price increases in the analysis. That's not to say we wouldn't have any, but we just assume no price increases. So the overall market opportunity is split 50-50 between idiopathic and cancer-associated LEMS. Because the idiopathic patients live longer, there are a few of them and they live longer. There's about 150, we assume diagnosable every year, but they live a long time.

On the cancer-associated LEMS side, there's about 900 that are there. And so you have about 1,700 that are alive at any point in time, if they're, in fact, living those 17 months we talked about. So as we think about that with the most conservative structure and assuming just a total addressable market of 85% of those patients who are diagnosed because some are too early in their process to require treatment and some might be too late to actually gain a benefit. We assume that's about 85% based on some data we've seen in the marketplace.

And again, all those conservative assumptions, the total addressable market is $1.2 billion. And the upper end is obviously going to be much higher without going into specifics. But again, we're just focused on that. And we have an ongoing patent issue. We have one left. We can talk about that in a second. If we successfully resolve that, we have more than 9 years at the current -- with the state of the current agreements that we have with other generic defendants. We would have more than 9 years to continue to build this market. And the testing, again, and getting that word out on education becomes critically important.

Right. So you've settled 3 of the 4 challengers and those 3 settlements said 2034, correct?

February of 2035.

February of 2035. That's how you get to your 9. Can you talk a little bit about expectations for when that fourth challenger might have a resolution?

So I can't give any specifics because it's ongoing litigation, but thanks for the question. We will continue to do what's right for the company and right for the patients. And so we're going to look to gain the best advantage we can. We believe in our IP. Our IP is very, very strong. And we're excited to bring that to closure, but we won't do anything that hurts the company.

Right. Okay. That's helpful. So we look forward to a resolution potentially in the future for that. And I guess -- so I think that was great on FIRDAPSE. We talked a little bit about FYCOMPA already in the beginning of the call. But maybe just overall for the business, FYCOMPA is sticky, and we're seeing that, but it is losing revenues over time. We have the strong AGAMREE launch that we talked about, continued FIRDAPSE growth. I guess how do you just think about the overall growth of the business as we move into 2026? And I'd love your thoughts on what a potential BD transaction, how that could impact growth and just in broad terms because obviously, you can't be specific about some of these.

When we think about the overall growth of the business, obviously, we knew when we bought FYCOMPA, we knew what the life expectancy was. That was a play that we did to improve the balance sheet and increase our flexibility to acquire more products. And it worked because without that, I'm not sure we would have gotten AGAMREE. So now that the time comes when you have the natural decline of the product to generic incursion. The question is, can we -- with the current things we're doing, overcome that decline. And we believe we can. And not just because of the size of the markets, but Jeff can speak to some of the programs we've instituted. We've had very strong patient adherence programs, but we've really ratcheted that up because we believe once a patient gets on, obviously, it's in their best interest and our best interest to keep that patient on. So we've done that, but we believe that we can outgrow the decline, and we are very confident in that because of the traction we see in the market. But Jeff, do you want to speak to some of the changes we've made around -- specifically around FIRDAPSE?

Sure. So like Rich mentioned, there are multiple levers that you can pull to help grow the business. And one, oncology is a huge opportunity for us, obviously, continued growth in the idiopathic. The other side is the patients that you do have. Our discontinuation rates, our annual discontinuation rate for LEMS for FIRDAPSE is 20%, 15% to 20%. So what we noticed some of the patients that discontinued, they were not reaching their maintenance dose or their optimal dose for therapy. So we've instituted programs and really ramped up efforts with the specialty pharmacy and educational efforts to educate the HCP as well as the patient on that titration process to make sure these patients are giving FIRDAPSE an adequate trial. And we've seen benefits from that because of lower discontinuations of those patients that are initiating therapy. So things like that will help us optimize FIRDAPSE without new patient acquisitions. So we feel like that's definitely going to benefit patients in FIRDAPSE.

Got it. Okay. That's helpful. So -- and is it -- would it still be 15% to 20% year-over-year growth as we move into '26? Or would you, I guess, give us an update early next year?

So we believe for the midterm, 15% to 20% is achievable on a bigger base. This is one of the challenges we get how can you keep doing this? And Jeff's team has really -- so he talked about the pool of patients, but they not only prioritize patients, then they hyperprioritize the patients. So -- and that's -- there's a constant churn there. So we just don't say, okay, it's 500 patients that are in that pool. We're always looking to who is the most appropriate one.

And again, having that confidence of 50% of the patients come from that pool every quarter, every year, we can actually have a really good predictive approach to our business. So yes, we do believe that. And then AGAMREE there's a growth opportunity there. As I said earlier, 95% of patients have been on the steroid, only 70% are. Lots of reasons why it's not higher than 70%. But it's one of those reasons that the adverse events you have with the steroid are so bad that people drop off because the benefit goes away. And if you have a superior steroid, can you grow that to 85%? Can you grow it to 90%, again, of the addressable market, so...

Okay. Okay. That's helpful. And then just you touched on this as well in the beginning, Rich, but just on BD and the potential opportunities out there, can you just remind us what that ideal BD deal looks like in terms of development stage, I think you were saying you could take a little bit of risk, late-stage development or regulatory, but also just the peak sales potential, how much you have to deploy capital-wise?

Sure. So our ideal opportunity is a rare disease. We are therapeutically area agnostic because of the infrastructure we have to support peak sales, about $500 million. And the rationale for that is we would be a very strong player in that subset of the market. If we go much above that, that moves the needle for bigger companies, and then we're not as strong a buyer, if you will. So we would want to be -- or currently doing things that -- acquiring things that are either immediately accretive or nearly immediately accretive. And then we are looking to take on more regulatory risk as the strength of our balance sheet increases because we believe that we want to have future-oriented discussions.

We love the execution of the company, but that's by its nature, a backward-looking metric. We want to be able to have those forward-looking ones. I think one of the best indices of our success as a commercial organization is more than 90% of the opportunities that come through our door are coming to us. We're not seeking them out. So somebody comes in and says, hey, you're a great commercial marketer. We want to talk to you about it. And I think we said in our press release around the most recent earnings call, we've done more than 100 assessments since January. And so we believe we can continue to grow the company through not only the execution on the life cycle management, on the sales and improving the retention of patients, but also through business development.

Yes, absolutely. And then, Mike, I've left you alone this whole session, so I want to make sure you have a question as well. Can you just remind us just a little bit of the financial health of the company, the cash that you have to deploy and I guess, how you're thinking about as we move into '26?

Sure. So we have, as reported at the end of September, $689.9 million of cash and cash equivalents, no funded debt. With the cash flow generation, depending on specifics of any opportunity, we're comfortable that we could pay north of $1 billion for one or a series of acquisitions. I know we announced the buyback. That does not change anything in our BD strategy or our ability to execute on that strategy.

Excellent. Okay. And then -- so Rich, I just want to turn it back to you. Any closing remarks you might have? And anything you think is really important to emphasize for investors?

The company continues to execute, which again, is a great story. There's great interest in other companies leveraging their assets through our infrastructure. And we're excited about those discussions. We will continue to be diligent and discriminatory because we believe we have a good track record of acquiring products that can perform. And we feel that pressure every day to do the right deal, not to just do a deal, but to do the right deal that will continue to grow the company. And we're really excited about the things we're seeing. It's challenging.

The market is very challenging. As interest rates fall, companies have more access to capital, and so may need somebody less like us. So that's one of the reasons why we've talked for the last year about moving a little bit further back into the pipeline to take on opportunities that could accelerate the growth of the company. And we're excited about that.

Yes. As am I. Well, thank you so much. It was wonderful to us having you here today, and it was a great discussion. Thank you.

Thank you very much. We appreciate that.

Thank you.

Greetings, and welcome to the Catalyst Pharmaceuticals Third Quarter 2025 Financial Results Conference Call and webcast. [Operator Instructions]. As a reminder, this conference is being recorded.

It's now my pleasure to turn the call over to Chief Financial Officer, Mike Kalb. Mike, please go ahead.

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's Third quarter 2025 financial results and business highlights. Richard Daly, President and CEO, will lead the call today; and Jeffrey Del Carmen, our Chief Commercial Officer, and I will also present. Additionally, Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer; and Dr. Will Andrews, our Chief Medical Officer, will be available for the Q&A.

Before we begin, I would like to remind you that in our remarks this morning and in the Q&A session, we will make statements about expected future results, which may be forward-looking statements for purposes of federal securities laws. These statements reflect our current expectations, estimates and projections and do not guarantee future performance. They involve risks, uncertainties and assumptions that are difficult to predict and may not prove to be accurate. Actual results may vary from the expectations contained in our forward-looking statements.

These forward-looking statements should be considered only in conjunction with the detailed information contained in our SEC filings, including the risk factors described in our 2024 annual report on Form 10-K filed with the SEC on February 26, 2025, and in our subsequent filings with the SEC, including our third quarter 2025 quarterly report on Form 10-Q, which was filed yesterday, November 5, 2025, with the SEC.

At this time, I'll turn the call over to Rich. Rich?

Thanks, Mike. Good morning, everyone, and thank you for joining us. Today's call will begin with an overview of our portfolio highlights and key strategic growth initiatives, followed by a detailed discussion of our commercial performance and financial results for the quarter. Catalyst delivered outstanding third quarter 2025 results with accelerating momentum, strong demand for our rare disease therapies and continued progress on key strategic growth priorities. These results highlight our growing positive impact on the patient communities we serve and demonstrate our unwavering commitment to operational excellence and delivering long-term value.

Catalyst reported another record quarter in Q3 2025 with total revenue reaching $148.4 million, an increase of 15.3% year-over-year. These results were driven primarily by outstanding performance of FIRDAPSE and the growing success of AGAMREE, along with extended patient preference for FYCOMPA following generic entry. We ended the third quarter of 2025 with a cash position of $689.9 million and no debt, further reinforcing our ability to invest strategically for long-term growth.

Last month, we announced that our Board of Directors authorized a new share buyback program to repurchase up to $200 million of shares of Catalyst's outstanding common stock between October 1, 2025, and December 31, 2026. As mentioned, we believe that we can execute this share repurchase program without impairing the advancement of our business development strategy. Further, we believe that this repurchase program will enable us to provide value to our shareholders, and we are increasingly confident in our long-term outlook. With our strong performance over the first 3 quarters of the year, we are raising our 2025 total revenue guidance to between $565 million and $585 million.

Let's start with FIRDAPSE. FIRDAPSE continues to demonstrate outstanding performance, maintaining its position as the only evidence-based approved product in the U.S. for the treatment of Lambert-Eaton myasthenic syndrome. The brand delivered another strong quarter of sustained growth, generating revenue of $92.2 million, an increase of 16.2% year-over-year. We are confident that FIRDAPSE remains well positioned for sustained organic growth across both idiopathic and cancer-associated LEMS markets, supported by a robust pool of patients and individuals progressing through their diagnostic journey. The strong and visible demand underscores the durability of the franchise and reinforces our leadership in addressing the needs of the LEMS community. Jeff will go into more detail about our growth strategy in these distinct markets in his remarks.

Since we believe that potentially 90% of cancer-associated LEMS patients remain undiagnosed, we see a meaningful opportunity to expand our reach in this high potential underserved population. We expect momentum to build in the months ahead, setting the stage for sustained growth in 2026 and beyond. We remain confident that we'll meet our full year total net product revenue guidance for FIRDAPSE of between $355 million and $360 million. Jeff will cover the specifics in his section of the call.

Moving on to AGAMREE. While FIRDAPSE continues to perform incredibly well, we're equally encouraged by the market potential and performance of AGAMREE. AGAMREE continues to outperform expectations, generating $32.4 million in net product revenue in Q3 2025, a 115.2% increase year-over-year from Q3 2024. These results are largely driven by steady conversion from both prednisone and EMFLAZA, a strong 90% patient retention rate and growing adoption across virtually all of the DMD centers of excellence around the United States. AGAMREE's commercial execution continues to track well with strong patient retention and increasing prescriber engagement, further supported by recent full deployment of our dedicated field team back in April. Continued transitions from both branded and generic therapies, along with growing market receptivity and payer alignment due to an increased appreciation for AGAMREE's potential differentiation from the current standard of care, reinforce our confidence in raising full year outlook to between $105 million and $115 million.

Catalyst continues to actively enroll patients in the SUMMIT study, an open-label 5-year follow-up study designed to evaluate the long-term clinical safety profile of AGAMREE, including potential benefits on behavior, stature, bone health and cardiovascular health.

Finally, let's take a look at FYCOMPA. FYCOMPA delivered higher-than-anticipated results in the third quarter of 2025 with revenue of $23.8 million, which reflects a year-over-year decrease of 25.8%. This performance was driven in part by patients electing to stay on their existing treatment plan following generic entry for FYCOMPA tablets in Q2 2025. Although we anticipate the impact of generic entry to increase going forward as other entrants eventually come to the market, we are raising our full year net product revenue guidance for this product to between $100 million and $110 million.

Now I'd like to reiterate what we believe sets us apart. Catalyst's competitive advantage is built on the strength of not only our field efforts from both our sales and medical teams, but also our patient engagement services that we offer through Catalyst Pathways for patients taking FIRDAPSE and AGAMREE. Catalyst Pathways is our personalized treatment support program for patients who are enrolled in the program and serves as a single source for personalized treatment support, education and guidance to the dosing and titration regimen required to reach an effective therapeutic dose for each patient's therapy. We believe that navigating the health care system with this support is far better for patients needing treatment for their rare diseases and for the health care community in general.

Finally, our strong relationship with health care and rare disease communities continues to drive meaningful stakeholder engagement. Together, our single-source approach enables the patients we serve to streamline access, identify appropriate therapy, receive timely treatment and remain compliant with their care. We believe these differentiating capabilities will enhance prospects for creating uncommon value for our current patients and for other rare diseases patients in the future.

Let's turn to business development. We remain highly disciplined in our business development strategy, and we continue to actively evaluate a broad range of opportunities, many of which are inbounds, reflecting growing awareness of the value we create for patients in need. We also have an aggressive outreach initiative, resulting in over 100 assessments since January. The support services that we have developed through Catalyst Pathways for patients who are taking FIRDAPSE and AGAMREE are industry-leading and can be applied to any rare therapeutic area. So we remain therapeutic area agnostic.

We believe our integrated infrastructure with our sales teams identifying potential patients and Catalyst Pathways providing direct support to patients, getting the patients on therapy, keeping them on therapy and ensuring the patient receives the optimum dose are important components of our continued success and our core capabilities that we see as not universally available through other companies serving patients living with rare diseases.

Now for an update on IP. We continue to advance initiatives to protect long-term value for our portfolio. On August 26, we announced the settlement of our pending FIRDAPSE patent litigation with Lupin. As part of the settlement, Lupin received a license to market generic FIRDAPSE beginning in February of 2035, on the same market entry date as Teva Pharmaceuticals had previously agreed to in their settlement. This settlement leaves only one patent case still pending against Hetero USA, Inc. The trial date has been set for March of 2026, which is prior to the expiration of the automatic 30-month stay set for May 26, 2026. I'd like to reiterate my previous comment, as we have consistently stated, we will vigorously defend our IP for all of our products.

With that, I'll turn the call over to Jeff, who can provide additional insights into our commercial performance. Jeff?

Thanks, Rich. Q3 2025 marked another record-setting quarter for our commercial organization, generating net product revenue of $148.4 million, a 17.4% increase compared to Q3 2024. These results highlight continued strong execution across our portfolio and sustained demand for our differentiated therapies. As Rich noted, Catalyst again delivered organic growth from FIRDAPSE, continued adoption of AGAMREE and stronger-than-expected revenue from FYCOMPA.

Our Q3 2025 net product revenue underscores the strength and resilience of our rare disease platform. FIRDAPSE continues to deliver sustained organic growth. Q3 2025 revenue results of $92.2 million reflect its robust growth trajectory, driven by durable demand and continued execution of our growth strategy, resulting in 16.2% growth in Q3 2025 compared to Q3 2024. New patient enrollments exceeded forecast. Prescription approval rates remained above 90% across both government and commercial payers and discontinuation rates were in line with expectations, tracking below an annualized rate of 20%.

These indicators reflect strong patient adherence and reinforce the foundation for sustained performance. Leading indicators, including new patient starts and refill volumes through October continue to trend positively, reinforcing our confidence in achieving our full year 2025 FIRDAPSE net product revenue guidance of between $355 million and $360 million.

Our next phase of growth for FIRDAPSE will come from both idiopathic LEMS and cancer-associated LEMS. For idiopathic LEMS, we continue to advance our patient identification efforts by augmenting data sources, further expanding the pool of over 500 LEMS patients in active diagnostic stages. With a dedicated sales force of 16 regional account managers and enhanced lead optimization efforts that better target patients most likely to initiate FIRDAPSE treatment, these patients now represent over 50% of new starts each quarter, strengthening our confidence in sustaining future organic growth.

In addition, we have intensified efforts to expand VGCC antibody testing among patients often misdiagnosed with myasthenia gravis, which remains the most common misdiagnosis for LEMS patients. We have seen sustained VGCC antibody testing growth of 9% quarter-on-quarter over the last 2 years, with only approximately 30% penetration on the idiopathic side of the LEMS market opportunity, we have clearly identified the potential sustainable growth.

Our expansion strategy for cancer-associated LEMS is sharply focused on multiple critical levers, broadening access to VGCC antibody testing and streamlining the diagnostic pathway, now further reinforced by the recent NCCN small cell lung cancer guideline update on July 25 and our efforts to update the oncology care pathways and expanding HCP education on those updated pathways. As Rich mentioned, we are continuing to execute on those fronts.

First, we have launched a frictionless testing model designed to remove referral bottlenecks and accelerate the time to diagnosis. Second, the updated NCCN guidelines now include VGCC antibody testing and recommend amifampridine, the only evidence-based approved treatment for patients with LEMS, potentially providing a strong tailwind for market adoption. Third, we are pursuing new collaborations with leading oncology networks to integrate these updated NCCN guidelines into their care pathways. Lastly, we continue to expand our footprint across the community to educate those concentrated oncology centers on the updated care pathways.

With over 90% of cancer LEMS patients still undiagnosed, the unmet need remains significant. We see a clear opportunity to drive awareness, increase access and deliver meaningful impact across the LEMS community. As we move forward, we're executing with precision to accelerate diagnosis, increase adoption and position FIRDAPSE for sustained momentum through 2026 and beyond.

Turning to AGAMREE. AGAMREE continues to demonstrate strong market momentum as a differentiated therapy for Duchenne Muscular Dystrophy. In the third quarter of 2025, we generated net product revenue of $32.4 million, a 115.2% increase over the third quarter of 2024. Adoption is expanding steadily across key treatment centers. To date, each of the top 45 DMD centers of excellence have enrolled at least 1 patient on AGAMREE and 255 unique health care providers have submitted enrollment forms, reflecting broad and growing engagement across the community. Since launch, approximately 43% of patients being treated with AGAMREE transitioned from prednisone and 41% from EMFLAZA, highlighting AGAMREE's broad clinical relevance across established treatment segments.

Reimbursement performance remains robust with success rates above 85%, in line with our expectations. Our commercial team continues to execute effectively, driving targeted provider education and deepening payer engagement to support durable uptake and sustained market expansion. Given the continued strength of these trends, we are raising our full year net product revenue guidance for AGAMREE to a range of between $105 million and $115 million.

Finally, FYCOMPA delivered Q3 2025 net product revenue of $23.8 million, reflecting continued demand following the first generic entry for tablets in late May. We remain cautiously optimistic on FYCOMPA brand loyalty and based on stronger-than-expected performance in the first 9 months, we are raising full year 2025 FYCOMPA revenue guidance to between $100 million and $110 million. As previously noted, we do expect continued revenue erosion from generic competition to impact FYCOMPA performance in Q4 and beyond as more generics enter the market.

In summary, our commercial team continues to execute with strong discipline and focus, delivering robust portfolio performance and advancing our next phase of growth. We are confident that our diversified portfolio, best-in-class commercial capabilities and focused execution position us well to capture the significant growth opportunities ahead.

As we look to close 2025, our priorities remain clear: driving commercial excellence, broadening patient access and maximizing the value of our portfolio to deliver sustainable growth and long-term shareholder value. I would like to thank the entire Catalyst team for their continued dedication and commitment to performance, execution and the patients we serve.

At this time, I would like to turn the call back over to Mike.

Thank you, Jeff. Our performance during the third quarter of 2025 has kept us on pace for another strong year, driven by our solid financial performance, financial discipline and strong execution. Our total revenues for the third quarter of 2025 were $148.4 million, an approximate 15.3% increase when compared to total revenues of $128.7 million for the third quarter of 2024. The third quarter of 2024 included approximately $2.3 million of license and other revenue, which consisted principally of a milestone payment that we earned from our sublicensee, DyDo Pharma, upon its receiving regulatory approval to commercialize FIRDAPSE for the treatment of patients with LEMS in Japan compared to license and other revenue in the third quarter of 2025 of $27,000.

Net income before income taxes for the third quarter of 2025 were $71.0 million, a 24.2% increase year-over-year compared to $57.2 million for the third quarter of 2024. We reported GAAP net income for the third quarter of 2025 of $52.8 million or $0.42 per diluted share. GAAP net income increased by 20.3% year-over-year compared to GAAP net income for the third quarter of 2024 of $43.9 million or $0.35 per diluted share.

Non-GAAP net income for the third quarter of 2025 was $86.1 million or $0.68 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMREE and Ruzurgi of $9.3 million, stock-based compensation expense of $5.7 million, the income tax provision of $18.3 million and depreciation of $100,000. This compares to non-GAAP net income for the third quarter of 2024 of $71.1 million or $0.57 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMREE and Ruzurgi of $9.3 million, stock-based compensation expense of $4.4 million, the income tax provision of $13.3 million and depreciation of $100,000.

Our year-to-date effective tax rate through the first 9 months of 2025 was 23.6% compared to 24.0% through the first 9 months of 2024. The effective tax rate is affected by many factors, including the number of stock options exercised in any given period and is likely to fluctuate in future periods. Cost of sales expense was approximately $22.7 million in the third quarter of 2025 compared to $19.3 million in the third quarter of 2024 and consisted principally of royalties. As a reminder, AGAMREE royalties paid to the product licensor equal 5% of net sales up to $100 million for 2025 and 7% of net sales in excess of $100 million and up to $200 million with additional increases as net sales increase.

The company is also required to make a $12.5 million sales-based milestone payment once AGAMREE's net product revenue for a fiscal year reaches $100 million. When this occurs, this milestone payment will be capitalized and amortized over the estimated remaining useful life of the asset. Further details on our royalty obligations for AGAMREE as well as FIRDAPSE and FYCOMPA are disclosed in our third quarter Form 10-Q.

Research and development expenses were $2.7 million in the third quarter of 2025 compared to $3.3 million in the third quarter of 2024. Our R&D spending in the third quarter of 2025 was comprised mainly of costs to support our 2 ongoing AGAMREE studies.

Selling, general and administrative or SG&A expenses for the third quarter of 2025 totaled $47.5 million as compared to $45.9 million in Q3 2024, reflecting in part an increase in our cancer-associated LEMS activities. As reported, we ended the third quarter of 2025 with cash and cash equivalents of $689.9 million compared to $517.6 million at December 31, 2024. This increase in cash of $172.3 million between the end of '24 and September 30, 2025, was largely driven by $163.8 million in cash generated from operations of the business. This highlights our continued focus on profit optimization and cash flow generation.

We believe our current funds, along with our expected continued generation of cash from operations, continue to allow us the financial flexibility to fund our existing R&D programs, meet our potential contractual obligations and support our strategic initiatives, business development and portfolio expansion efforts, leading to long-term growth and value creation. More detailed information and analysis of our third quarter 2025 financial performance may be found in our quarterly report on Form 10-Q, which was filed with the Securities and Exchange Commission yesterday, November 5, and can be found on the Investor Relations page of our website.

At this time, I will turn the call back over to Rich. Rich?

Thanks, Mike. As you heard on today's call, Catalyst is closing 2025 with strong momentum and an unwavering commitment to deliver value for the patients we serve. This confidence is reflected in our updated guidance, which we raised after careful consideration and with respect to market conditions. This quarter's exceptional results reflect the strength of our commercial execution, the resilience of our business model and the depth of talent across our organization. I want to thank our employees, partners and shareholders for their continued support and dedication.

As we finish 2025 with ongoing momentum, we look forward to continued success. Thank you for joining us on today's call, and I'll now turn the call back over to the operator for questions.

[Operator Instructions] Our first question today is coming from Samantha Semenkow from Citigroup.

Congratulations on the great quarter. First question is on FIRDAPSE. I'm wondering as you start to penetrate and really put a lot of effort into the oncology portion of the LEMS business, are you able to share any anecdotes or data points that you have thus far that can really help us begin to quantify how those efforts are progressing? And then on FYCOMPA, do you have a sense of what proportion of the patients are expected to stay loyal to branded FYCOMPA? I'm just trying to figure out as these additional generics come to the market, how much erosion we're looking at for this fourth quarter?

Thanks for the question, Sam. For the first one, I'll turn it over to Jeff. Jeff, you want to give us some update?

As far as the cancer-associated LEMS and providing maybe some anecdotes or leading indicators on how performance is going, we will do that in future quarters. What we're continuing to do is, like we mentioned on the earnings call, the -- our first goal was really to get that testing in place, which we were successful. Secondly, the updates to the NCCN guidelines, which again, we were also very successful in doing that last July. So very pleased with that. Currently, what we're doing is we're educating and working on educating physicians and group practices and hopefully partnering with them to change their care pathways and then also have screening arrangements put in place. Moving forward, when we are successful with those discussions and those partnerships, we will share some of the leading indicators.

So to build on that as well, oncology is a highly structured and complex marketplace. And so since the NCCN guidelines, which actually gives us the opportunity or license, if you will, to begin those discussions, we got that back at the end of July. And we're really excited about the conversations we're having with the decision-makers in the oncology marketplace. It is a process. We are working through it, and we expect, as we've said in the past that, that will deliver results in 2026. Sam, I'm just going to move on to your next question, if that's okay. So Jeff, the FYCOMPA stickiness, any thoughts on that?

Sure. So as you see by the revenue numbers that we've reported, FYCOMPA continues to perform very well. And we do believe a lot of that is from brand loyalty and the stickiness. As additional generics enter the market later this year, we do expect additional erosion to revenue. However, we do also expect some patients to remain loyal to the brand. As far as a specific percentage, we haven't really provided guidance on that. And we're very confident in our guidance that we did provide for FYCOMPA.

Next question today is coming from Joon Lee from Truist Securities.

This is Asim, on for June. Just a couple from us. Just on the FYCOMPA stickiness, why are patients choosing to stay on FYCOMPA longer? What are you hearing from them? And can you remind us how many generics are currently in the market and how many you could expect by year-end? And then just on FIRDAPSE, are you actively seeing oncology practices following the updated NCCN guidelines? Or would you say that's more of a 2026 story?

I'm sorry, could you just repeat the second question? I just lost you for a second.

Yes. Just on FIRDAPSE, are you actively seeing oncology practices following the updated guidelines? Or is that more of a 2026 story?

Jeff, on the stickiness, obviously, FYCOMPA is a one of one in the marketplace. And then we have one generic in there. So do you want to speak to the potential stickiness?

Sure. So when you take a look at the generic competition right now for FYCOMPA, there's one and that's Teva. Teva's generic is at about a 17-plus percent discount to FYCOMPA. So it's a relatively good market for us when competing against Teva's product. The stickiness, a lot of patients are afraid that to switch medications for antiseizure medications. What they don't want is something where a generic 1 month and then it's different so that they have a breakthrough seizure. So one of the things that they like to do is stay on the brand that they've been on for the last 10, 12-plus years. So that lends to the stickiness of the product. Also, we still have field personnel for FYCOMPA that are out there talking to many of the physicians that prescribe antiseizure medications and financial resources to help patients that are on FYCOMPA. So I think all those things contribute to the stickiness, and that's what we've seen in the revenue that we reported. Does that answer your question about FYCOMPA?

Yes.

Okay. Great. And as far as the NCCN guidelines and the adoption of those, it is more of a 2026 -- part of our 2026, that's when we'll see more of the adoption. But it is significant in our discussions with these group practices in getting the care pathways changed and to adopt the NCCN guidelines. So we're starting to see those discussions pay off. But again, our goal for 2025 and the remainder of this year is really to accelerate screening for these patients, which will turn into hopefully patients that will be diagnosed with small cell lung cancer LEMS next year and then also transition to FIRDAPSE.

[Operator Instructions] Our next question is coming from Leland Gershell from Oppenheimer.

It's Rohan on for Leland. Congrats on another strong quarter. I wanted to ask on AGAMREE, if you've seen any changes in the prescriber base. It's been talked about before how as people get more acquainted with longer-term data from AGAMREE and real world data, more prescribers may be inclined to come on and give their patients AGAMREE. Have you seen any change in the breakdown there or any new prescribers? And more so on AGAMREE, just given the scope of what the differentiated steroid can do, are you exploring any other opportunities outside of Duchenne? And yes, that's all for me.

Rohan, thanks for the question. Jeff, do you want to handle first?

Sure. So for AGAMREE, we're extremely pleased with how the launch has gone thus far over the last 1.5 years plus. And we have seen strong adoption. And like I mentioned, the top 45 centers of excellence for Duchenne Muscular Dystrophy make up about 80% of the steroid prescriptions for boys living with DMD. And each of the top 45 have enrolled at least 1 patient on AGAMREE. So that's strong adoption there. When you take a look at the top 100 centers of excellence, we have over 95% penetration in those markets -- in those centers of excellence. So again, broad adoption. And then also, we've talked about the adoption across both segments of prednisone as well as EMFLAZA.

So very confident and pleased with that performance thus far. And also the 257 health care providers that have prescribed AGAMREE, at least 1 patient on AGAMREE. So again, strong adoption. Now what our focus is to continue to deepen that adoption so that we can get more and more patients within each center on AGAMREE as these physicians see the differentiation of AGAMREE versus the other steroids. So that's our goal moving forward.

On the second question regarding additional opportunities beyond Duchenne. First, we'll turn it over to Will Andrews, our Chief Medical Officer. Will?

Yes. Thank you, Rich. We are evaluating life cycle management opportunities for consideration of add-on indications for AGAMREE. And as an example, you can imagine, we look at disorders that have muscle inflammation that significantly affect function in patients and even other inflammatory disorders. So we are actively evaluating life cycle management opportunities.

Thanks, Will. And I would just emphasize that our life cycle opportunities are focused in the rare space. So we want to continue with the profile that we have with the product. So we're really excited about the things we're exploring right now, and we'll probably be addressing those in future -- on future calls.

Next question today is coming from [ Pavan Patel ] from Bank of America.

The first one is on AGAMREE. I believe you mentioned that you've reached nearly all of the DMD centers of excellence. So maybe if you can provide some more color on the depth of prescribing versus breadth? And what feedback are you getting from physicians and payers about the profile? And what do you see as the main hurdle to full conversion in this patient population? And then on FIRDAPSE, just the growth is pretty strong on a year-over-year basis, and you reaffirmed your full year guidance for that product rather than raising it. So that sort of implies a sequential slowdown. Just wondering if this reflects conservatism? Or are you anticipating that true slowdown in 4Q? And can you elaborate on which growth driver, whether it's new patients, enhanced dosing or persistence is contributing the most to your year-over-year growth?

So I'm sorry, just clarifying your second question, and we'll come back to that. Jeff, do you want to handle the first one on centers of excellence and depth and breadth?

Sure. So very good question. And it truly does vary across all the centers of excellence. What's very unique about Duchenne Muscular Dystrophy is one center of excellence is far different from the next one. So there's not one uniform way that they prescribe steroids for their boys living with DMD. So we have many of these centers with multiple patients on it like 10, 20 and in some cases, many more than that or much more than that. And then we have other centers that only have a handful of patients.

So that's what our goal is. We have to look at these as a unique center of excellence, one center, it means that you know one center of excellence. So we have to identify a variety of ways to help provide education on the differentiation for AGAMREE for that center that's pertinent to them. So hopefully, that answers your question, but it varies across all top 45 and the top 100. And then as far as the payers, when we look at the payer reception for AGAMREE. I mentioned it on the call, over 85% of the patients are reimbursed and it's much more than that. So it's a very strong reception by the payer and the payer landscape, we're very pleased with, and we don't anticipate it changing here [indiscernible].

[ Pavan,] your second question, we just want to clarify, was about the year-over-year strength and what are the growth drivers that we anticipated that you mentioned new patients, dosing. I think there was one -- a third one you mentioned as well.

The last one was persistence of patients. Yes. And then also on the sequential like slowdown implied by the guidance, if you can explain that?

Just to make sure we work on FIRDAPSE, correct. Okay. So Yes. And what I want to talk about here is I did mention this, that the growth from FIRDAPSE will come from multiple segments, both idiopathic LEMS as well as the cancer-associated LEMS. What we're seeing now is the 500 or so patients that -- the pool of patients that we have that are somewhere in their diagnostic journey for LEMS. We are able to help these patients or convert these patients onto treatment appropriately much more quickly. And we're doing that by helping focus our field force, our dedicated field force of 16 [ RAMs ] now on the right leads to help these patients get on therapy. So that's one thing.

Another thing is we are -- we have many initiatives in place to get VGCC antibody testing added to panels, like an example is myasthenia gravis. So once that's added, we'll also see a growth in the number of patients that are tested and hopefully diagnosed with LEMS, which we can then focus on and help these patients get on therapy. Another thing is we've identified additional data sources that are providing more LEMS -- prospective LEMS patients to us, which will then turn into leads and increase our pool of patients. We also utilize machine learning that helps triangulate claims data.

So here's a quick example is we've noticed that patients that have a negative AChR test as well as a positive VGCC test. Those patients are more likely to get on FIRDAPSE treatment and get on treatment sooner. So then we provide those leads to our [ RAM ] team to help those patients accelerate their time to get on product. So that's been very beneficial.

A couple of other things, a pharmacy intervention program. What we're doing is we noticed that in some cases, it takes patients a little longer to titrate than the label to get on to their optimal -- appropriate optimal dose. And our pharmacy outreach and the intervention program, we've seen benefits from that program that has accelerated that time to get on their appropriate optimal maintenance dose more efficiently. So a lot of those things are helping contribute to today's growth, and we are confident we'll continue to provide levers for us to grow in the future. And then in 2026, we expect CA LEMS to more -- contribute more to the growth of FIRDAPSE.

Is that -- [ Pavan ], does that address your question?

Yes, that addresses the question pretty well. And then I guess just like in terms of like the guidance, why not take it up on FIRDAPSE at the product level given the strength thus far? Just what's the rationale behind that? Is that just conservatism? Or is there something that you expect in 4Q, whether there was a one timer in 3Q or something like that?

So we know this product really, really well. For 7 years it's been on the market. So we're confident in our ability to forecast the product. Jeff, do you want to speak at a high level to the performance of new enrollees in the third quarter, just at a high level.

Sure. New enrollees were very strong and much higher than we had forecasted. So it was the highest month that we've had in 2 years.

So -- but those patients, obviously, we don't get a full year from those patients. So we're looking forward to that growth continuing as we go into 2026. So we're seeing very positive trends. We like what we see. We like what we see on the testing growth as well. And there's a touch -- I would say a touch of seasonality in the fourth quarter. So we can look at sequentials and we can look at year-over-year, but we're confident in our continued growth.

Next question today is coming from Luke Herrmann from Robert W. Baird.

Congrats on the quarter. First on AGAMREE, given this week's outcome of confirmatory data from the approved PMOs, which is obviously just really unfortunate for the community, but do you think this can sort of emphasize the importance of an optimal steroid regimen for physicians and patients and their families?

That's a great question. This is a community that's very, very tight. They know they see everything, and they're really engaged with us and obviously, all the companies. When we think about the use of a product like AGAMREE, it's obviously the first product that a patient gets on. It's recommended, it's foundational therapy. And these stories are unfortunate for the community. But as we gain further and further strength and momentum in the market, we believe that we can fill the necessary gaps that might be coming out about as a result of some of these conversations. Jeff, do you want to speak to what we might have heard so far about some of these issues surrounding the studies? Yes.

Sure. I mean, I'll stick to the AGAMREE and steroids. I think what Rich was mentioning too is that steroids are the foundation of treatment for those [ suffering from DMD ] and so that we know that having that is the biggest part of this. And regardless of what other products that come out, the steroids will always be the first thing that they will treat their patients with.

Yes. And I think our patient advocacy and then patient support really helps us to kind of weather that -- those bumps in the market that don't necessarily affect us directly. And the support that we give to the patients through Catalyst Pathways is really a strong opportunity for the patients to get past some of these situations. So we think we can continue to perform well.

Great. And then I just had one on the financials and some commentary on payment timing in relation to the cash balance, I think you noted a lower gross to net expense. Is that material for gross to net going forward? And was that a benefit in 3Q?

Sure. Thanks for the question. It's not insignificant. So from a materiality perspective, certainly, we thought it was noteworthy. And then sorry, what was the second part of that question?

Was -- is this an impact on a go-forward basis? Or did it affect 3Q as well?

It is in Q3 and impacts the future as well. This is a permanent change to our contract terms.

I just want to emphasize that it's a permanent change. We experienced this one-timer mostly because if you think about a shift, we renegotiated a very, very important, very large contract with the buyer, and they used to pay twice a month. Now they only pay once a month. So we received their payment October...

October 2, we received approximately $24.9 million. That under the old terms would have been by September 30.

So for this issue specifically because you're asking about it, we don't expect this one to repeat. So this is a one-timer, and that's why we called it out in the Q. We wanted to be sure that we were clear about how it might affect Q3, but we expect the customer to continue to pay once a month, and we think that's accommodated for or will be accommodated for on a go-forward basis.

The next question today is coming from Sudan Loganathan from Stephens Inc.

This is Kesav on for Sudan. Congrats on the great quarter. My first question is just like roughly with the 17% off the WAC when Teva came in, what's the expected discount on FYCOMPA after more tablet and suspension generics enter later this year? And how does that discount curve look? And then my second one is kind of just on FIRDAPSE. With Hetero moving to trial slated for March 2026, what was the general sentiment during the Markman hearing considering that Teva and Lupin kind of decided to settle?

So on the first part of the -- first question, what we see is a mix shift, as you would expect. So when a generic enters the market, especially one that's so heavily influenced here by Medicaid, we're not affected in the Medicaid on our volume basis. So we did not -- we have not given price. But as we forecasted, we will lose volume. And then there's a mix shift. So there's -- as a percentage, a higher percentage of Medicaid, a lower percentage of commercial. And so that's what causes the price to go down. So the volume changes and it shifts and then we have an impact on price. So does that answer your question?

Yes, it does.

Okay. Great. Now your second question about hetero and where we stand, I'm going to turn it over to Steve Miller. Steve?

Thank you. With regard to the Markman hearing, it was relatively routine. There was a number of items that were simply pushed off to trial. [ Judge said ] simply that's a matter for trial. There were no outcomes that dramatically changed the view of the enforceability of the patents for either side and the strength of the cases. We continue to remain confident that our intellectual property is strong, and we're going to vigorously defend it. I also want to point out that like all the litigants in the past, we continue to have discussions with all of them, including Hetero. And if this last litigant has something to say that we think is in the best interest of the shareholders, we [indiscernible].

We've reached the end of our question-and-answer session. I'd like to turn the floor back over for any further or closing comments.

We're really appreciative of the support that we've received from the market, and we're really appreciative of the work that our employees have done in support of patients. And thank you again for your participation in the call today.

Thank you. That does conclude today's teleconference and webcast. You may disconnect your line at this time, and have a wonderful day. We thank you for your participation today.

Greetings, and welcome to the Catalyst Pharmaceuticals Second Quarter 2025 Financial Results Conference Call. [Operator Instructions] As a reminder, this conference is being recorded.

I would now like to turn the conference over to the Chief Financial Officer, Mike Kalb. Please go ahead, sir.

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's Second Quarter 2025 Financial Results and Business Highlights. Richard Daly, President and CEO, will lead the call today; and Jeffrey Del Carmen, our Chief Commercial Officer, and I will also present. Additionally, Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer; and Dr. Will Andrews, our Chief Medical Officer, will be available for the Q&A.

Before we begin, I would like to remind you that in our remarks this morning and in the Q&A session, we will make statements about expected future results, which may be forward-looking statements for purposes of federal securities laws. These statements reflect our current expectations, estimates and projections and do not guarantee future performance. They involve risks, uncertainties and assumptions that are difficult to predict and may not prove to be accurate. Actual results may vary from the expectations contained in our forward-looking statements.

These forward-looking statements should be considered only in conjunction with our detailed information contained in our SEC filings, including the risk factors described in our 2024 annual report on Form 10-K filed with the SEC on February 26, 2025, and in our second quarter 2025 quarterly report on Form 10-Q, which was filed yesterday, August 6, 2025, with the SEC.

At this time, I'll turn the call over to Rich. Rich?

Thanks, Mike. Good morning, everyone, and thank you for joining us. Catalyst delivered another record-setting quarter in Q2 2025 with total revenue reaching $146.6 million, an increase of 19.4% year-over-year. For the first half of 2025, total revenue grew 30.2% to $288 million, reflecting strong execution and sustained demand for our differentiated portfolio. We ended the second quarter of 2025 with a cash position of $652.8 million, reinforcing our ability to invest strategically for long-term growth.

Based on strong leading indicators, we remain confident in our trajectory and believe that we are on track to achieve our full year 2025 revenue guidance of $545 million to $565 million. Our outstanding progress is underpinned by the balanced performance of our commercial portfolio, which continues to deliver consistent growth and a positive impact on patients.

Let's dive deeper and start with FIRDAPSE. FIRDAPSE generated net product revenue of $84.8 million in Q2 2025, a growth of $7.5 million versus Q2 2024. As we discussed in our Q1 2025 earnings call, last year's second quarter results reflect the timing impact of the February 2024 Change Healthcare cybersecurity breach on our year-over-year growth rate. The temporary impact of the Change Healthcare security breach, which shifted volume from Q1 2024 to Q2 2024 was fully resolved by the end of June 2024. As a result, we believe that comparing FIRDAPSE first half 2025 performance to the same period in 2024 provides the most accurate view of the franchise's continued strength.

Importantly, the underlying demand remains strong, consistent and durable. Year-to-date, FIRDAPSE has delivered $168.6 million in net product revenue, representing a 16.9% increase over the first half of 2024. In 2025, our sales of FIRDAPSE have returned to their expected cadence, and we reaffirm our full year 2025 product -- net product revenue guidance of $355 million to $360 million.

We are confident that FIRDAPSE remains well positioned for sustained organic growth, supported by consistently high prescription approval rates and a robust pool of patients progressing through their diagnostic journey. This strong and visible demand underscores the durability of our franchise and reinforces our leadership in addressing the needs of the LEMS patient community.

To unlock the next phase of growth for FIRDAPSE, we are actively advancing a focused expansion strategy centered on education of HCPs supported by the recent updated NCCN guidelines. Since we believe that potentially 90% of cancer-associated LEMS patients remain undiagnosed, we see a meaningful opportunity to expand our reach in this high potential, underserved population. We expect momentum to build in the months ahead, setting the stage for a sustained growth in 2026 and beyond. Jeff will cover the specifics in his section of the call.

While FIRDAPSE continues to perform well, we are equally encouraged by the accelerating adoption and performance of AGAMREE. AGAMREE continues to outperform expectations, generating $27.4 million in net product revenue in Q2 2025, a new post-launch high and a 213% increase year-over-year from Q2 2024. First half net revenues reached $49.4 million, up 398% from the prior year, driven by steady conversion from both Prednisone and EMFLAZA, a strong 90% patient retention rate and growing adoption across Duchenne's Centers of Excellence.

AGAMREE's commercial execution is tracking well with strong patient retention and increasing prescriber engagement, further supported by our full deployment of our dedicated field team. Continued transitions from both branded and generic therapies, along with growing market receptivity and payer alignment reinforce our confidence in meeting our full year outlook. Backed by focused commercial strategy, AGAMREE is well positioned to continue momentum. We are reaffirming AGAMREE's full year 2025 net product revenue guidance of $100 million to $110 million.

Let's switch to FYCOMPA. FYCOMPA delivered solid results in the second quarter of 2025 with revenue of $34.3 million, reflecting a year-over-year decrease of 6%. First half 2025 revenue reached $70 million, up 4.5% from the same period last year. We anticipate the impact of generic competition going forward and our full year net product revenue guidance of $90 million to $95 million for FYCOMPA remains unchanged.

We recently strengthened our Corporate and Board leadership. I'd like to formally welcome Dr. Will Andrews as our new Chief Medical Officer. Will joined Catalyst on June 2, 2025. Will brings deep pharmaceutical expertise in rare diseases, spanning clinical development, medical affairs, business development and portfolio strategy. His leadership will ensure a scientific rigor and operational excellence as we continue to differentiate AGAMREE within the Duchenne treatment landscape and further solidify our position in the LEMS community. Will's role in leading our medical function is foundational to ensuring the long-term success of our portfolio.

For AGAMREE, Will is leading the SUMMIT study, which aims to generate real-world evidence in support of the appropriate use of AGAMREE. To-date, the study has initiated 19 sites with patient enrollment progressing. As the study progresses, we will provide updates when the interim data becomes available. In parallel, Will is overseeing the execution of our Phase I study comparing AGAMREE, prednisone and deflazacort to determine the potential switching algorithms. Additionally, we are working to assess the immunosuppressive effects of AGAMREE that could help us define the life cycle management potential for -- of AGAMREE.

Initial results are expected by the end of 2025 to early 2026. Will's expertise and insights also enhance our strategic perspective as we advance growth initiatives aligned with our long-term vision. This past Monday, we announced that Dr. Dan Curran joined our Board of Directors. We are pleased to welcome Dr. Curran to our Board of Directors. He brings deep experience in rare disease and a strong track record in business development and strategic growth. His experience advancing transformative therapies and building value across the development continuum align well with our mission and growth strategy.

On the business development and IP front, we remain highly disciplined in employing our business development strategy, actively evaluating a broad range of opportunities that align both strategically and financially with our long-range plan. Our commitment to value-driven growth is unwavering, and we are confident in our -- that our focused and deliberate approach will position us to capitalize on the right opportunities. In parallel, we are advancing on our initiatives to protect and enhance the long-term value of our portfolio.

We are still awaiting a trial date in our ongoing patent litigation for FIRDAPSE with our remaining first filers, but anticipate it to be in Q4 2025 or Q1 2026. We expect more clarity on this issue on or after our Markman hearing, which is scheduled for October 7, 2025. We remain committed to updating our performance related to environmental, social and governance reporting. In June, we published our 2024 ESG report, underscoring our commitment to sustainable growth and responsible innovation. This report is available on Catalyst's website.

In summary, we are entering the second half of 2025 with strong momentum, clearly defined growth drivers and a focused strategy to deliver long-term value and impact for patients.

With that, I'll turn it over to Jeff, who will provide additional insights into our commercial performance.

Thanks, Rich. Q2 marked another record-setting quarter for our commercial organization, underscoring continued strong execution across the portfolio and sustained demand for our differentiated therapies.

As Rich noted, Catalyst delivered sustained organic growth from FIRDAPSE, strong revenues from FYCOMPA and accelerating adoption of AGAMREE. Q2 net product revenue of $146.5 million puts us firmly on track to meet our 2025 guidance, reflecting the strength of our neuroscience business. FIRDAPSE remains the only evidence-based FDA-approved treatment for Lambert-Eaton Myasthenic Syndrome. The brand continues to perform well across both neurology and our emerging oncology segments.

FIRDAPSE continues its strong multi-quarter growth trajectory, driven by durable demand and consistent execution. Prescription approval rates remained above 90% across both government and commercial payers and discontinuation rates were in line with expectations, tracking below an annualized rate of 20%. These indicators reflect strong patient adherence and reinforce the foundation for sustained performance.

Leading indicators, including steady growth in new patient starts and refill volumes through July continue to trend positively, reinforcing our confidence in achieving full year 2025 FIRDAPSE revenue guidance. We continue to advance our patient identification efforts and continue to have a pool of over 500 LEMS patients in active diagnostic stages. These patients consistently account for approximately half of new starts each quarter, giving us confidence in driving sustained organic growth.

To further unlock the next phase of growth for FIRDAPSE, we are actively advancing a focused expansion strategy centered on HCP education, which is supported by the recently updated NCCN guidelines for small cell lung cancer, broadening access to VGCC antibody testing, streamlining the diagnostic journey and accelerating adoption across oncology and neurology settings.

By improving diagnosis, particularly in small cell lung cancer patients and increasing recognition of patients often misdiagnosed with myasthenia gravis, we hope to meaningfully expand the addressable population and drive sustained growth within what we believe is a more than $1 billion addressable market opportunity. As we advance these priorities, we are implementing a 3-step program of targeted initiatives designed to enhance diagnostic access and clinical recognition across key market segments.

First, we are deploying a frictionless testing model that makes it easier for physicians and particularly oncologists to order BGCC antibody screening directly within their practices, eliminating referral delays and helping to significantly reduce time to diagnosis. These efforts represent progress in advancing important diagnostic initiatives to help improve LEMS patient outcomes and accelerate the adoption of FIRDAPSE.

Second, we are pleased to share that ahead of schedule, the NCCN published updated guidelines for small cell lung cancer on July 25, which now include BGCC antibody testing and recommend that amifampridine should be considered as a treatment for cancer patients with LEMS. This milestone strengthens our ability to expand access, accelerate diagnosis and help drive FIRDAPSE adoption in oncology. Most importantly, it represents meaningful progress in improving care for cancer patients living with LEMS.

Our third step involves forging strategic partnerships with leading oncology practices to update care pathways, which are generally structured plans that outline the steps and interventions required for patient care, ensuring standardized and efficient treatment for specific conditions.

We plan to execute comprehensive education and promotion programs to ensure broad understanding and integration of the care pathways. Due to the early updating of the NCCN guidelines, we are accelerating our investment in educating HCPs on the new guidelines and working to incorporate diagnostic and treatment recommendations into the care pathways in targeted large group oncology practices.

Since we believe that potentially 90% of cancer-associated LEMS patients remain undiagnosed, we see a meaningful opportunity to expand our reach in this high potential underserved population. We expect momentum to build in the months ahead, setting the stage for sustained growth in 2026 and beyond.

Turning to AGAMREE. AGAMREE continues to demonstrate strong early market momentum as a differentiated treatment for Duchenne Muscular Dystrophy. Q2 2025 net product revenue was $27.4 million. Adoption continues to expand across key centers. To-date, 93% of the top 45 DMD centers of excellence and 231 unique health care providers have submitted enrollment forms. In Q2, approximately 43% of patients transitioned from prednisone and 42% from EMFLAZA, underscoring AGAMREE's broad clinical relevance across standard treatment segments.

Reimbursement success remains high at approximately 85%, consistent with our expectations. Our commercial and medical teams continue to drive targeted provider education and payer engagement, supporting durable uptake and sustained market expansion. FYCOMPA delivered Q2 2025 net product revenue of $34.3 million, reflecting continued demand despite the first generic approval for tablets following loss of exclusivity in late-May.

As previously noted, we do expect revenue erosion from generic competition to impact FYCOMPA performance in the second half of the year and beyond. That said, based on a strong first half performance and the execution of our mitigation strategies, we are reaffirming our full year 2025 FYCOMPA revenue guidance.

In summary, our commercial and medical teams continue to execute with discipline and focus, delivering strong portfolio performance while advancing the next wave of our growth, led by FIRDAPSE's expansion into oncology. We believe that we are well positioned to seize the growth opportunities ahead, and we remain focused on driving commercial execution excellence, enhancing patient access and scaling our commercial impact across our entire product portfolio.

I want to thank the entire team at Catalyst for their unwavering commitment to the patients we serve and look forward to a successful second half of 2025.

With that, I'll turn the call back over to Mike.

Thank you, Jeff. Our performance during the second quarter of 2025 has kept us on pace for another strong year, driven by our solid financial performance, financial discipline and strong execution. With the continued success of our flagship product, FIRDAPSE as well as the strong growth of AGAMREE, which we launched in mid-March of 2024 and the solid performance of FYCOMPA, which currently has only one generic competitor for the tablets following the patent expiry in May of 2025, we have set the groundwork for what promises to be yet another unparalleled year in 2025.

We are reaffirming our 2025 full year total revenue guidance as initially provided in February and reaffirmed in May. We remain steadfast in our commitment to driving growth and expanding our portfolio to capitalize on emerging opportunities throughout the year.

Our total revenues for the second quarter of 2025 were $146.6 million, an approximate 19.4% increase when compared to total revenues of $122.7 million for the second quarter of 2024. Product revenue net for our lead product FIRDAPSE was $84.8 million, a 9.7% increase year-over-year compared to $77.4 million.

As a reminder, the second quarter of 2024 benefited from the Change Healthcare cybersecurity incident that occurred in the first quarter of 2024, which is evidenced by the fact that our growth in net product revenue for FIRDAPSE for the first half of 2025 compared to the first half of 2024 was 16.9%.

Product revenue net for the second quarter of 2025 for FYCOMPA was $34.3 million compared to $36.5 million in the second quarter of 2024. Product revenue net in the second quarter of 2025 for AGAMREE was $27.4 million as compared to $8.7 million in the second quarter of 2024, representing a year-over-year increase of approximately 213%. Net income before income taxes for the second quarter of 2025 was $69.3 million, a 24.2% increase year-over-year compared to $55.8 million for the second quarter of 2024.

We reported GAAP net income for the second quarter of 2025 of $52.1 million or $0.41 per diluted share. GAAP net income increased by 27.7% year-over-year compared to GAAP net income for the second quarter of 2024 of $40.8 million or $0.33 per diluted share.

Non-GAAP net income for the second quarter of 2025 was $86.4 million or $0.68 per diluted share, which excludes from GAAP net income; amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMREE and Ruzurgi of $9.3 million; stock-based compensation expense of $7.6 million; income tax provision of $17.2 million and depreciation of $100,000.

This compares to non-GAAP net income for the second quarter of 2024 of $69.6 million or $0.56 per diluted share, which excludes from GAAP net income; amortization of intangible assets related to our acquisitions of FYCOMPA, AGAMREE and Ruzurgi of $9.3 million; stock-based compensation expense of $4.4 million; the income tax provision of $15.0 million; and depreciation of $100,000.

Our year-to-date effective income tax rate through the first half of 2025 was 22.6% compared to 24.5% through the first half of 2024. The effective tax rate is affected by many factors, including the number of stock options exercised in any given period and is likely to fluctuate in future periods.

Cost of sales expense was approximately $20.6 million in the second quarter of 2025 compared to $15.4 million in the second quarter of 2024 and consisted principally of royalties. As a reminder, AGAMREE royalties paid to the product licensor equal 5% of net sales up to $100 million, 7% of net sales in excess of $100 million and up to $200 million with additional increases as net sales increase beyond $200 million. The company is also required to make a $12.5 million sales-based milestone payment once AGAMREE's net product revenue reaches $100 million, [ that ] this milestone payment when earned, will be capitalized and amortized over the estimated remaining useful life of the asset. Further details on our royalty obligations for AGAMREE are disclosed in our Q2 2025 Form 10-Q.

Research and development expenses were $4.4 million in the second quarter of 2025, up 46% from $3.0 million in the second quarter of 2024. Our R&D spending in the second quarter of 2025 was comprised mainly of costs to support our ongoing AGAMREE studies.

Selling, general and administrative or SG&A expenses for the second quarter of 2025 totaled $45.9 million compared to $40.7 million in the second quarter of 2024. The increase in SG&A expenses for the second quarter of 2025 was primarily driven by increased personnel costs, including the implementation of 2 dedicated sales forces for FIRDAPSE and AGAMREE, which became effective on April 1, 2025.

As reported, at June 30, 2025, we had cash and cash equivalents of $652.8 million compared to $517.6 million at December 31, 2024. The increase in cash of $135.2 million was primarily driven by $131.3 million in cash generated from operations of the business, underscoring our continued focus on profit optimization and strong cash flow generation.

We believe our current funds, along with our anticipated continued generation of cash from operations, continue to provide us with the financial flexibility to fund our existing R&D programs, meet our potential contractual obligations and support our strategic initiatives, business development and portfolio expansion efforts, leading to long-term growth and value creation.

More detailed information and analysis of our second quarter 2025 financial performance may be found in our quarterly report on Form 10-Q, which was filed with the Securities and Exchange Commission yesterday, August 6, 2025, and can be found on the Investor Relations page on our website.

At this time, I will turn the call back over to Rich.

Thanks, Mike. As we look ahead, Catalyst remains focused on and confident in our ability to drive sustained growth and long-term value. We are executing a clear strategy rooted in a strong commercial foundation. Our focus and execution will continue to drive our performance and enable us to deliver on our commitments while making a meaningful difference for patients.

Our teams are energized by the opportunities ahead, and we remain committed to delivering differentiated therapies with operational excellence and responsible growth. Thank you to our Catalyst teams for their unwavering dedication to patients and to all of you for your continued support. We look forward to continuing to execute our growth initiatives as we advance into the second half of 2025.

I'll now turn the call back over to the operator. Thank you.

[Operator Instructions] First question that we have comes from Samantha Semenkow of Citibank.

A couple first on FIRDAPSE. Can you speak more about your strategy for educating oncologists on the LEMS opportunity? And I'm curious what metrics are you using to track the success of the campaign?

And then as you continue to focus on the oncology opportunity, when do you expect that segment of the business to begin representing a greater proportion of revenues versus historical experience? I believe in the past, you said it's about 20% to 25% of revenues come from oncology patients.

Thanks, Samantha. I appreciate the question. I'm going to turn that to Jeff. Jeff, do you want to take that one?

Sure. Sam, good to hear from you. As we noted in the script, I think the -- our approach to oncology and the addressable market there for cancer-associated LEMS, the first thing we really wanted to do was increase the -- provide frictionless testing for these patients. And we were able to make that happen where oncologists and all HCPs could order our no-cost VGCC antibody test within their office. So that shortened the time to the diagnostic.

Secondly, the NCCN guideline changes. So you heard that in July 25, that -- those changes happen. So those were the first 2 steps.

And now we're working on working with the group practices and trying to put together some arrangements with them for screening. So that's the third step.

How are we going to educate these physicians? We're focused on digital marketing and media. We're also doing congresses and conferences and publication plan, working with our medical team on that to do abstracts as well. So those are the things that we're doing to help educate physicians about this important opportunity to diagnose LEMS patients.

How we're going to track it? Is through increased VGCC antibody testing from oncologists. We're also tracking it, like you said, we anticipate an increase over time of our mix of patients that are cancer-associated LEMS patients. This year, our primary goal is to increase screening for cancer-associated LEMS patients. 90% of these patients are undiagnosed. So if we can help these patients get diagnosed, then we anticipate a larger addressable market that we should start seeing an increase in that percentage in 2026 and beyond. So that's how we're looking at this, Sam.

That's super helpful. Just one follow-up for me. You mentioned on the prepared remarks that there's a Phase I DMD switching study from prednisone or from EMFLAZA to AGAMREE. I'm wondering how do you plan to leverage this data as you commercialize AGAMREE or is it for additional studies that you're planning? Just any context you could share there would be very helpful.

Thanks, Sam. I'm going to turn that over to Bill Andrews, our Chief Medical Officer. Obviously, we're looking at -- we'll be looking for the data, and I think a lot of that will determine how we leverage it. So obviously, the study is ongoing. So I think it becomes a little challenging to answer your question directly.

But Will, do you want to take that?

Yes. Thank you, Rich, and hello, Sam. The study is an exploratory trial, and we are looking forward to seeing data later this year and then early into 2026. And that data will describe -- be descriptive and will help indicate whether we need to do future studies to further delineate potential switching, et cetera.

The next question we have comes from Joon Lee of Truist Securities.

Congrats on the quarter. This is Asim on for Joon. Just a couple from us. So what impact are you seeing on FIRDAPSE and AGAMREE uptake following the increase in sales force size that became effective on April 1?

And then as a follow-up on FIRDAPSE, just help us think about this growth over the next few quarters. Should we still be expecting a mid-teens growth rate on a year-over-year basis?

Thanks, Asim. I think, again, it's -- Jeff is going to answer that question. So thanks for the question. Jeff?

Thanks for the question. Like you said, we deployed dedicated sales forces for both AGAMREE and FIRDAPSE in April, so beginning in the second quarter. What we've seen, it's still too early to tell. But based on performance, you can see that there are increased engagements that have taken place. We are -- also deeper relationships now within these accounts and also more frequent interactions with many of the health care providers and our targeted physicians.

We're also seeing it from our leading indicators. As I mentioned on the call, with FIRDAPSE, we're exceeding -- or meeting and exceeding the enrollments thus far in July. So very strong, and we also have a strong pipeline there. With AGAMREE too, we've seen the 231 unique physicians that have enrolled since launch. So that also increased from the second quarter. So very, very satisfied with the results thus far, and we'll continue to track that moving forward.

As far as performance for FIRDAPSE and what you should expect. We reiterated guidance for the balance of this year. And also, like I just mentioned, the strong leading indicators. So we do believe in our business and the foundation of our business for FIRDAPSE, so we expect continued growth.

The next question we have comes from Jason Gerberry of Bank of America.

A couple for me. Just given all that's going on with ELEVIDYS in the, I guess, nonambulatory group of patients and some of the commentary earlier in the year around queuing, I'm just kind of wondering how that impacts the quarterly kinetics of AGAMREE uptake, if at all?

And then with FIRDAPSE, I'm wondering if you could just maybe comment on sort of the time lag for the NCCN recommendation to have more of an impactful commercial impact.

Jason, thanks so much. So from a commercial perspective, we'll take the ELEVIDYS question. We'll turn it over to Jeff. And if they're more of a clinical, we'll have Will jump in on that.

But Jeff, do you want to take that one first?

Thanks for the question, Jason. And with AGAMREE and steroids specifically, they are the foundation of treatment for these boys living with DMD. So the impact of the unfortunate news about ELEVIDYS really hasn't impacted much the use of steroids. And we do strongly believe that AGAMREE is the steroid of choice.

Will, do you want to jump in there on the clinical?

Happy to, Rich. Thank you. We see the new additions to the NCCN guidelines as critical to help [ users. ]

[ Is this ] for ELEVIDYS? For DMD.

Yes, I'm sorry.

And the second question was on NCCN. Yes.

On ELEVIDYS, I would add medically that I agree with Jeff from the perspective of that being an entirely different modality of treatment. And as Jeff said, it's unfortunate as to what's happened, but we still see, of course, corticosteroids and vamorolone as foundational treatment for these kids that is unaffected, we believe, by what's happened with ELEVIDYS.

To answer your second question on the NCCN guidelines, we see that as critical to helping educate oncologists, clinical oncologists on the disease and very importantly, on how to better diagnose the disease and that amifampridine is a critical option for them in the treatment.

And I'll just add, the way we see the cadence working out, Jeff and the team have done a really good job of getting this frictionless testing in there, which we believe is the first step. So that the physician can order the test and the report will come to their office. Prior to this patient had to actually go to a remote site, get a test and then get the test results themselves. So that's the first step.

The NCCN guideline really is the bridge to care pathways within the practice. So as we get this out there, and it was just released, obviously, last week, we get this out there, the opportunity then is to work through those oncology practices get these NCCN guidelines embedded as the standard and NCCN is the true standard within oncology, get that embedded and then begin talking to the group practices about how they want to use it and how we can be supportive.

And then as Jeff mentioned earlier on an earlier question, those educational efforts working hand-in-hand with the GPOs and then beyond the GPOs as well become important. Jeff?

Thanks, Rich. And Jason, to your question, when do we expect this to hit commercial and increase some of the net revenue. The way we look at this, again, is increased screening this year and then next year with more patients thankfully being diagnosed, the addressable market increases. And then that can help with the treatment with FIRDAPSE. So we expect that to take place probably in the second half of next year and beyond is when we start seeing converting some of these diagnosed patients onto FIRDAPSE therapy. Hopefully that helps.

The next question we have comes from Leland Gershell of Oppenheimer & Co.

Just one from us, just on OpEx, maybe for Michael. Just wondering, given the realignment in the sales for FIRDAPSE and AGAMREE affected a few months ago. Just wondering how you see SG&A expense rolling forward? It looks like it was pretty flattish from Q1 to Q2. Just wondering if we should see a bit of a bump and the increase into the second half.

Thanks, Leland, and I hope all is well. So as you know, we did not quantify SG&A guidance. We did talk qualitatively at the beginning of the year. Some of the folks that we had hired were in, obviously, a little before April 1. So I think the flat is a little bit to what we'd expect. But to your point, with the acceleration of the NCCN guidelines, I do think that we will see a little bit of an uptick in the second half.

The next question we have comes from Luke Herrmann of Baird.

Two from me. First, on the Change Health disruption. I was wondering if you could share your thoughts on whether all the loose ends have been closed here or if there's potential for any future anomalies.

And then second, recent language from the administration on pharma tariffs, which has obviously been sort of constantly evolving. I see that FIRDAPSE is manufactured in the U.S. and Canada. And I think you've had a process underway to consider addition of third-party manufacturers for AGAMREE. I guess, has there been any recent evolution in thinking around the manufacturing strategy?

Thanks, Luke. Thanks for the questions. So the Change Healthcare issue, the disruption we saw there really was -- as we mentioned in the script, really was a pull sort of a push into the second quarter of 2024. We believe and we see the natural cadence of FIRDAPSE returning post June of 2024. And now we believe that effect is washed through the system. So we're very confident about that because we can see -- because of our specialty pharmacy relationship, we can see what's happening there at a very granular level.

And you may remember, in our second -- first quarter call in May last year, we mentioned that we saw it the day it began to happen. So we were able to take rapid action. So having that specialty pharmacy is a real benefit for us.

On the tariff side, you are correct. When you think about the volume of sales based on our guidance, about 66% of sales come from FIRDAPSE as it relates to our guidance for the year. And we feel really well protected there. And you are also correct on working with third-party manufacturers for AGAMREE, and transitioning that to the U.S.

And we were working on that well before this current administration came into play. So we're confident we'll be able to do that in the near term, but it's still going to take a while to actually validate the [ plant. ] But we feel really good about the opportunity to shield that from tariffs as well in the mid- to long term because of the ability to move it to the U.S.

The next question we have comes from Jennifer Kim of Cantor.

Maybe to start with FIRDAPSE. Are there any -- is there any color you can give around the average dose you've reached for the quarter and maybe expectations on that growth for the rest of the year? And then anything you can say about discontinuation rates for the quarter?

Jeff, do you want to take that?

Sure. Thanks, Rich. And yes, the -- first, I'll answer the discontinuation rates. Discontinuation rates were below 20%, the annual discontinuation rate of 20% for FIRDAPSE. So hopefully, were you asking about -- Jennifer, were you asking about FIRDAPSE specifically on discontinuation?

Yes.

Okay. So less than 20% annual discontinuation. And what was your first question again?

Dose.

The dose. So since the label expansion in June of last year, we have seen significant increase to the average daily dose, approximately about 4 milligrams, which is what we had expected. So we -- the dose is performing according to what we had expected.

Okay. That's helpful. And maybe a question on FYCOMPA. I think right now, Teva is the only generic for the oral tablet that's launched. I just want to clarify, do they have a 100-day exclusivity? And I asked because I wonder whether guidance could prove to be on the conservative side for that product.

So Teva is the only player in the space right now for a generic. They have 180-day exclusivity. As to whether or not the guidance is too conservative. So far, as we mentioned in the call, we've booked $70 million in sales. Our guidance is $90 million to $95 million. However, we think that there's an opportunity to be cautious here because this is a significant risk. If there is another generic player that could come into the market, and they have -- we have not seen them yet. And so we're taking what we believe is a prudent position for the balance of the year.

And we will expect generic -- additional generic competitors by mid-December. And as you know, Jennifer, sometimes they can load the channel into November. And so that could affect our ex-factory sales. So we believe this is a prudent approach to the forecast.

The next question we have comes from Sudan Loganathan of Stephens Inc.

Catalyst Pharma team, congrats on another strong quarter. First one, I want to ask how you view the potential for PTCT's Translarna FDA approval for DMD treatment to affect how AGAMREE will continue to fit in, retain market share and continue to grow in this space?

And then secondly, what is your current stance on near-term strategic initiatives to offset FYCOMPA's LOE? Are there any ongoing discussions with potential partners regarding a commercial stage asset or a late-stage pipeline asset?

I think, it's appropriate to turn that to Jeff for the first part.

Yes, Sudan. Just like -- similar to ELEVIDYS, the way we look at this, is AGAMREE and steroids are [ finest of ] the treatment. Translarna does not impact that at all. So we just continue to feel strongly about AGAMREE's potential moving forward because we do believe it is the best and differentiated steroid out there for boys living with DMD. And as far as...

Well, sorry, Sudan, could you just repeat your question? I lost you for a second. Repeat the second question.

Yes. The second one was just on your current stance on the near-term strategic initiatives to offset the FYCOMPA LOE, if there's any ongoing discussions with either commercial stage asset or late-stage pipeline asset that you're kind of leaning towards in this market environment? Or how you're viewing that going forward?

Thanks for clarifying. So on our business development front as we look to offset -- we think of our business into 2 elements, right? The business development front, bringing in new assets. We are incredibly busy. We believe this is probably one of the best buying environments we've seen in the last 15 or 20 years. And our team is assessing multiple opportunities at any point in time.

And as mentioned on previous calls, we continue to see 80% of the opportunities coming to us inbound. So other companies will approach us or other parties will approach us about opportunities. So we're really excited about building -- continuing to build the portfolio.

In addition to that, a second pillar of our business is life cycle management. And when we talk about the opportunity around AGAMREE, looking for opportunities to better understand the molecule and how it might play in the orphan and rare space, we continue to do that work. And on the commercial front in diversifying our opportunities with FIRDAPSE, we believe the oncology play and the dedicated sales force can continue to work to offset the loss of FYCOMPA.

Thank you. Ladies and gentlemen, that then brings an end to our question-and-answer session. Thank you for joining today's conference. You may now disconnect your lines.