Greetings, and welcome to the Catalyst Pharmaceuticals Fourth Quarter and Full-Year 2024 Financial Results Conference Call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions]. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Mike Kalb. Thank you, and you may begin.

Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's fourth quarter and full-year 2024 financial results and business highlights. Leading the call today is Richard Daly, Catalyst's President and Chief Executive Officer. We're also joined by Jeff Del Carmen, our Chief Commercial Officer. Further, for the Q&A session, Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer; and Dr. Gary Ingenito, our Chief Medical and Regulatory Officer will be available for questions. 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 relate to our current expectations, estimates and projections and are not guarantees of future performance. They involve risks, uncertainties, and assumptions that are difficult to predict and may prove not 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. At this time, I'll turn the call over to Rich.

Thanks, Mike. Good morning, everyone, and thank you for joining us today. 2024 was an exceptional year marked by record revenue growth and a strong fourth quarter that showcased our scalability and financial strength, reinforcing the success of our strategy. For the full-year 2024, total revenues grew by 23.5% year-over-year to $491.7 million exceeding the upper end of our previous guidance and highlighting our ability to capitalize on market opportunities, while maintaining operational excellence. Full-year net product revenue for 2024 reached $489.3 million an exceptional of 23.4% increase over 2023, fueled in part by our continued successful launch of AGAMREE, which began on March 13, 2024. In Q4 2024, total revenues and net product revenues reached a record $141.8 million an increase of 28.3% and 30% over Q4 2023, respectively. The increase was driven by the continued organic growth of FIRDAPSE, steady contributions of FYCOMPA and new revenue from AGAMREE, which accounted for a portion of the year-over-year increase. Looking ahead, we expect this momentum to continue with 2025 total revenue forecasted to be between $545 million and $565 million. We ended the year with a robust cash position of $517.6 million and no debt, providing an enhanced capacity to invest in strategic growth opportunities. Catalyst competitive advantage is built on the commitment of our outstanding team and the strength of our patient engagement services, such as Catalyst Pathways. Additionally, our strong relationship with health care and rare disease communities drives meaningful stakeholder engagement. Together, our holistic approach enables the patients we serve to streamline access, identify appropriate therapy, receive timely treatment and remain compliant with their care. FIRDAPSE sustained momentum reflects the strength of this approach, delivering 14 consecutive quarters of 15% or more growth year-over-year. As the only evidence based treatment for LEMS in U.S, we believe that…

Thanks, Rich, and good morning, everyone. We are thrilled with our exceptional performance in 2024, marked by full-year net product revenue of $489.3 million and total revenues of $491.7 million surpassing the upper end of our 2024 full-year total revenue guidance of $475 million to $485 million. This outstanding achievement was driven by FIRDAPSE' full-year revenue reaching a record high of $306 million the successful U.S. commercial launch of AGAMREE and the continued strong contribution from FYCOMPA. Let's start by reviewing our advancements with FIRDAPSE, the only evidence-based FDA approved treatment for Lambert-Eaton myasthenic syndrome. In the fourth quarter, net product revenue amounted to $82.5 million showcasing a remarkable 18.3% year-over-year growth. Furthermore, FIRDAPSE' net product revenue for 2024 showed strong 18% growth year-over-year, driven by a steady influx of new patient initiations and a low annual discontinuation rate of under 15%, exceeding internal expectations. Prescription approval rates exceeded 90% for all types of payers, including government and private commercial insurers. Patients enrolled in Catalyst Pathways, including those with Medicare coverage accessing third-party foundation assistance experienced an average monthly co-pay of less than $2. Now I'd like to turn to AGAMREE. We are proud of AGAMREE's successful commercial launch and its growing recognition as a breakthrough treatment for Duchenne muscular dystrophy. Full-year 2024 net product revenue reached $46 million surpassing the revised guidance of $40 million to $45 million. In Q4 2024, AGAMREE delivered strong net sales of $21.1 million driven by increasing adoption. Adoption has been robust with 93% of the top 45 DMD Centers of Excellence and 209 unique healthcare providers submitting enrollments for AGAMREE. We continue to see strong transitions from both corticosteroid segments with 44% coming from Prednisone and 43% coming from EMFLAZA. Additionally, 85% of shipments were reimbursed with an average approval time of approximately…

Thank you, Jeff. As Rich and Jeff mentioned, Catalyst's fourth quarter and full-year 2024 financial performance resulted in another record breaking year, driven by exceptional execution by all facets of our business. In addition to our outstanding results, I would like to highlight the incredible work done by the Catalyst team with commercial launch of AGAMREE in March of 2024. Now on to 2024 results. Our total revenues for 2024 were $491.7 million, a 23.5% increase when compared to total revenues of $398.2 million for 2023. Product revenue net for 2024 from our lead product FIRDAPSE was $306 million, an 18.4% increase year-over-year compared to $258.4 million for 2023. Product revenue net for AGAMREE was $46 million for the period from the commercial launch on March 13, 2024 through year end. Product revenue net for FYCOMPA was $137.3 million compared to $138.1 million for 2023. As mentioned previously, 2024 FYCOMPA net product revenue was adversely affected by gross to net changes compared to 2023 when our gross to net for FYCOMPA was booked under a size more favorable arrangements with distributors and government agencies. Starting in 2024, all such costs have been tied to arrangements between us and those distributors and government agencies. Since our costs under these arrangements are higher than the size costs, we were impacted by an increase in gross to net deductions for FYCOMPA therapy causing a corresponding decrease in FYCOMPA net product revenue. During 2024, we recognized approximately $2.4 million in license and other revenue, which consisted primarily of a $2.1 million milestone payment earned upon DyDo receiving product approval to commercialize FIRDAPSE for the treatment of patients with LEMS in Japan. 2023 license and other revenue was approximately $1.7 million which included a $1.4 million milestone earned as a result of data submitting at…

As we embark on 2025, we are energized by the opportunities ahead and remain focused on delivering meaningful advancements for patients with rare diseases. Our passion, expertise, and unwavering commitment will continue to drive us forward as we execute our strategic priorities and expand our impact. I want to extend my gratitude to our employees, partners, and stakeholders for their dedication and support. Your contributions are instrumental in our success, and I look forward to what we will accomplish together in the year ahead. I'd like to turn the call back to the operator. Thank you.

Thank you. We will now be conducting a question-and-answer session. [Operator Instructions]. Our first question comes from the line of Charles Duncan with Cantor Fitzgerald. Please proceed with your question.

Thank you. Good morning, Rich and team. Congrats on a great year of progress and thanks for taking our question. I had a question about FIRDAPSE and really the guidance. I'm wondering if Jeff can provide a little bit more color on the new patient adds as well as the low discontinuation rate that you're seeing? And then with regard to guidance for FIRDAPSE, it's fairly narrowed 355 to 360 versus, for example, AGAMREE, which is broader. And I'm kind of wondering why you have so much precision on the guidance at this stage with regard to FIRDAPSE? Thanks.

Charles, thanks for the question. Obviously, we have a lot more experience with FIRDAPSE. And I think, Jeff -- I'll turn the call over to Jeff. But the pool of patients that we have and the continued confidence that we have in the brand and the uptake, I think Jeff can speak to that more clearly than I can. So Jeff?

Thanks, Rich, and good morning, Charles. As we've mentioned, we have over the 500 plus pipeline leads and those are those patients that are diagnosed with LEMS, but not yet on FIRDAPSE. Those patients typically contribute about 50% to our new enrollments each month or any given quarter. So we have a good handle on that, what we have in front of us and how and when we can help those patients transition on to therapy. So that gives us confidence in that sense. Also the discontinuation rate has been very steady, since we launched this back in 2019. And actually in 2024 full-year, the annual discontinuation rate was 15%. So that was very strong. So we're very confident about that. And as Rich mentioned, our experience with this product and what we've seen, it's what you see typically with rare disease products in this sense is that you have a -- for a while you get a steady state of enrollments, you know what -- how many enrollments you're going to get every given month. So that allows us and gives us confidence as to how we project or forecast 2025.

Is that helpful, Charles?

Yes, it is helpful. And I had just one follow-up and this is more of a strategy question with regard to the deployment of cash. You're building cash nicely in the last year. And I guess I'm thinking about FYCOMPA and perhaps revenue being reduced in '26. Is it your plan to try to replace that revenue with another effort in epilepsy or are you not so concerned about epilepsy specifically and investing further in that, but what would you like to do in terms of the FYCOMPA revs in '26?

So from a business development standpoint, we remain open to orphan and differentiated products that we think can make a difference, so for patients. If it's epilepsy, we would -- we stand behind the FYCOMPA acquisition as something that helped us build our war chest, if you will. But we want to stay in the orphan space. Retail is not a place we want to spend a lot more time in the future. If it's epilepsy and it's orphan, we would be interested in that. But we spent, we have a very wide aperture for acquisitions or partnerships for products.

That's helpful. Thank you.

Thank you.

Thank you. Our next question comes from the line of Jason Gerberry with Bank of America. Please proceed with your question.

Hey, guys. This is Pawan [ph] on for Jason. Congrats on the quarter. First question, I wanted to ask you guys what the new indication that you're pursuing with AGAMREE is, which you noted on the PR? And then the second question maybe on M&A, is the company looking at gene therapy, which has proven challenging for others in biopharma? And just wanted to get your thoughts on continued efforts towards revenue diversification? Thank you.

Thanks for the question. Regarding the new indications, so in the 10-K, we did speak to some of the work that we're doing to further classify how we might go forward with AGAMREE. I'll turn that over to Gary, our Chief Medical Officer. Gary, do you want to address the work that we're doing there?

Sure. Thank you. Yes, of course, our focus has been on the successful launch of AGAMREE, and we continue to look at rare diseases, which can benefit from a unique corticosteroid in that. And in terms of that, we're further characterizing AGAMREE in terms of its ability to provide immunosuppression and look at the characteristics of the product. That will allow us to better assess it for all the potential possibilities in the rare disease space.

And to your question about gene therapy, obviously, when we look at the opportunity within the orphan space, we see the vast majority of orphan conditions have a gene component. So this is something we track very, very closely. We think about it in very similar ways that we think about our other opportunities, non-gene therapy opportunities. However, we look at it with a little bit maybe a little bit more closely in a couple of components. Obviously, the opportunity has to be close to market, has to be immediately accretive or nearly immediately accretive. The CMC has to be very, very clean, because we think this is incredibly challenging. I think you all know I come from a cell therapy background, obviously different from gene, but closely aligned on the CMC component. So we look at that, we really want to be sure that we truly understand the CMC component of it. And there has to be a clear need. We've seen the uptake of gene therapy products be incredibly challenged. So we really want to understand the differentiated nature of the product for the therapy, and we want to understand what's going on in the market space. So we would consider it. However, we want to be really careful in understanding how the product might perform given the performance of past gene therapy products. Is that helpful?

Yes, that was great. Thank you, guys. Really appreciate the help.

Sure.

Thank you. Our next question comes from the line of Joon Lee with Truist Securities. Please proceed with your question.

Congrats on the quarter and thanks for taking the questions. This is Asim Rana on for Joon Lee. Just a question on the gross net. So you mentioned a gross net change for FIRDAPSE in 2025. How much of a change should we expect from '24? And then what proportion of FIRDAPSE patients are currently on the 100 milligram dose? Thank you.

Thanks. First question, we'll turn over to Mike and then the second question, we'll turn over to Jeff. Mike, do you want to take that one?

Sure. Thanks, Rich. 2024, we saw a very small impact from the IRA, less than 1%, if you look at the gross to net. We're thinking that based on the book of business that could be in the 3% to 3.5% range for 2025. And we'll continue to increase. As a reminder, we're under the small manufacturer, so there's a phased in approach.

Yes, over years, right?

Over the coming years, yes.

And I'll answer the question about the dosing. What I can give you right now is our average daily dose has increased. We saw since the label expansion in June, the average daily dose increased about 2 milligrams and we have forecasted for 2025, because of the increased availability or flexibility to physicians to optimize treatment for these patients, and an additional 2 milligrams built into the forecast for FIRDAPSE. At this time also there are about 20% of our patients that are at about a 100 milligrams.

Thank you.

Thanks.

Thank you. Our next question comes from the line of Samantha Semenkow with Citi. Please proceed with your question.

Hello. This is Benjamin Paluch on for Sam. Thanks for taking our questions. Maybe starting with AGAMREE, can you speak to the factors that you expect to drive demand forward in 2025 and that support your 2025 guidance of $100 million to $110 million. How should we think about the cadence of the launch throughout the year?

Thanks, Benjamin. I'll turn it over to Jeff for the demand question.

Thanks for the question. And regarding AGAMREE, we mentioned the percent mix of the transition. So the boys living with DMD, 44% coming from the Prednisone segment and about 43% coming from EMFLAZA. And that gives us extreme confidence in the continued growth because we're seeing transitions across both segments. Initially, when we launched, we expected more of our transitions to come from EMFLAZA for a variety of reasons. So having that gives again, gives us confidence. We continue to expect transitions from both segments moving forward at a steady pace. I did mention the competitive landscape that we're seeing. So what we expect is over time, over this year, we'll see some of that effect of the queuing effect that I mentioned will be playing itself through. So probably in the middle of the year and we'll start seeing more acceleration in enrollments, which is built into the forecast that we provided. Does that help at all?

Yes, that's helpful. Thank you so much. And then maybe moving over to FIRDAPSE. Can you speak to your expectations for revenue contribution from small cell lung cancer patients over 2025? How much growth are you expecting from this segment over the next several quarters?

Yes, Jeff will take that one as well.

Sure. At this time, we see about 20% of our revenue or our patients are from the small cell lung cancer, LEMS or cancer associated LEMS segment. And we expect in '25 to really focus on initiating or getting screening programs implemented. So it's really about helping those 90% of undiagnosed small cell lung cancer LEMS patients get diagnosed. So that's where our focus. So as far as contributions this year or growth from the small cell lung cancer LEMS segment, it's going to be very minimal in 2025, but we expect that to grow in out years.

Yes. So I'll just add to that. We've done a -- we’ve had a really thoughtful approach here in developing the KOL community around this in academic institutions. Jeff and the team have done a really, really good job making sure we will work, we work closely with the KOLs to get the opportunity for understanding of how this would work. And now pivoting that effort now to community, whereas Jeff said, the majority, the vast majority of small cell lung cancer is treated, the KOLs are in position, I think our messaging is in position, and so we could pivot now and begin to work with those physicians in the community. So we're really excited about this opportunity.

Great, thank you so much.

Thanks.

Thank you. Our next question comes from the line of Joe Catanzaro with Piper Sandler. Please proceed with your question.

Great. Hey, everybody. Thanks for taking my questions. Maybe first one following up on those recent comments you made, Jeff, on the queuing effect you're seeing in DMD. Wondering if you could elaborate a little bit more on what you mean by the queuing effect and anything you guys can do to shorten the time a patient spends in that queue and maybe somewhat relatedly what you've seen or heard that has sort of led you to make the decision to separate the sales force for FIRDAPSE and AGAMREE later this year? Thanks. And I have maybe a quick follow-up.

Sure. Thanks for the question. And first thing is a competitive landscape within the Duchenne space is a great thing for the patients and we want to acknowledge that. In June of last year, ELEVIDYS as you know had an expansion of their label. So that allowed for boys four years of age and older, regardless of ambulatory status, to be eligible or considered for ELEVIDYS treatment. When that happened, a lot of patients and their families and healthcare providers wanted to see if they could be evaluated or are they a fit for ELEVIDYS treatment. So this is just as an example. So when that happens, these patients in this treatment team, they do not want to change their treatment, their steroid if they're on Prednisone, they stay on Prednisone until they're evaluated and either treated or decided that it's not the right fit for them. If they are treated, then typically what they'll do is they'll wait about 60 days post ELEVIDYS treatment before they will consider switching their steroid. So it's that type of queuing effect. It's not just those patients that are on a new product, it's as they're being evaluated for treatment on any of these new products. So that's why it may delay, just delay the initiation of AGAMREE. But this happened in June and we've seen it playing through and we expect it to work its way through probably by the middle of this year. So we don't anticipate this being a long-term challenge for AGAMREE. We just see it as a slight delay for initiation to AGAMREE for some patients.

Is that helpful, Joe?

Yes, that's helpful. Maybe my follow-up on FYCOMPA. I know historically you guys have said that the epilepsy market could be resilient to generics. Wondering if your guidance for 2025 factors some of that resiliency in? And I think you alluded to this a little bit, but any new efforts that you'll employ to maybe slow that erosion trajectory? Thanks.

Yes. So we see the resiliency there. Obviously, we have the opportunity to sell the products with under the patent through the end of May, the solid dose and then the suspension through the end of November. So we're excited about that and we'll continue to make those efforts. The resiliency is there in the fact that there's that stickiness that we continually talk about. However, once the generics come on the market, we will see price erosion and volume erosion. So we expect to have that opportunity to continue to work with the payers, and we are working with the payers right now to continue to get favorable position in the marketplace and hold it. We do expect the patients to want the brand, a significant portion of them want the brand and the payers to want to work with us to hold that favorable position. So unlike traditional products, traditional retail products that lose the majority of their volume in eight weeks post patent expiry, we expect to hold on to a considerable amount of our volume throughout the year.

Great. And I wanted to respond. I forgot to Joe, forgot to respond to your message about splitting up the sales force. So what we're doing by having a dedicated sales force, it's allowing us more of a focused approach. I mentioned earlier the really crowded competitive landscape for Duchenne specifically. And when you look at that, a dedicated sales force for AGAMREE will allow us to optimize engagement with the treating HCP team, so that team across any center of excellence and support the continued growth. So that's why we made that decision. Now also for FIRDAPSE, when you evaluate the LEMS opportunity with FIRDAPSE, we've talked about this. We see significant opportunity for organic growth into the future. And by allowing this dedicated or specialized approach for AGAMREE or for FIRDAPSE also that will allow us for those deeper relationships to help find these patients and help these patients get diagnosed and transition to treatment.

Great. Thank you. Appreciate you circling back on that. Thanks for taking my questions.

Thanks, Joe.

Thank you. Our next question comes from the line of Joel Beatty with Baird. Please proceed with your question.

Hi, thanks for taking the questions. The first one is related to AGAMREE. In the competitive DMD marketplace, what are you seeing in terms of generic erosion for EMFLAZA and how much could that impact the opportunity for AGAMREE to grow?

Thanks for your question. We have not seen much generic entrant into this or conversion from and branded EMFLAZA at this point. From what we've seen, we haven't seen much. And we don't see that impacting AGAMREE and we have not really factored that into or made assumptions based on that into our forecast for AGAMREE in 2025. We haven't seen any changes to payers and how they've reacted to AGAMREE because of generic EMFLAZA. So that's what gives us confidence that we'll have minimal impact to AGAMREE in 2025.

So Joel, remember, EMFLAZA has been on market for a couple of years. Patients, if they are doing step therapy, they've tried Prednisone, they've tried branded EMFLAZA, making them step back to a generic EMFLAZA, not likely to happen. So right now and for the foreseeable future with the steadiness of the patient population, it's not likely to happen, not likely to have a large effect on the ability to take that patient who's already been on Prednisone or a version of EMFLAZA, it's just not likely to happen at this time and again for the foreseeable future. Does that make sense?

Yes, that does make sense. Thank you. And then maybe one last question related to SG&A in 2025. In the prepared remarks, you mentioned an increase this coming year. Could you quantify that at all?

Joel, thanks. We haven't quantified. If you recall, we did not quantify last year either or expecting the increase as we laid out, and we'll give a little bit more clarity as the year progresses, and part how long does it take to fill up in positions, et cetera.

Makes sense. Thank you.

Thank you.

Thank you. Our next question comes from the line of Leland Gershell with Oppenheimer. Please proceed with your question.

Hey, Leland.

Hey this is Rohan on for Leland. Thanks for taking the question.

Hi, Rohan. How are you doing?

Good, how are you? Just a couple of questions for me. Now that we have greater clarity on the FIRDAPSE IP protections moving forward, how does that shape further investment into the franchise? And also, how do you think about the extended commercial runway and how it changes the approach to business development when evaluating different size and structured deals?

Great question. Thank you for the questions. So remember, there are still two other litigants out there, right? So we're still working to defend the entire estate and we want to be sure that we're doing that. But the question is a really good one. As we look at the opportunity, should we end up with a full 10 year opportunity, we would look at it as if it's a brand that's going to be on the market for 10 years and you would continue to look and seek opportunities to invest. And as we talked about with an earlier question, you can see us working a bit more aggressively on the oncology side because if there's an opportunity to build that part of the business, which is about 50% of the business, we would continue to do that. We want to -- we may go back and look at some other research opportunities as well. There may be opportunities there. We have to fully vet those opportunities as well. But we'll continue to look at the opportunities and see if there's a way to continue to build the business. Lifecycle management has many forms. It could be through research, it could or development of a product, I should say, or it could be through further commercialization efforts as well. So we're going to consider all avenues to fully optimize the value of the asset.

Got it. And we saw that the guidance for FYCOMPA reflects some expectations of possible generic erosion. I mean, patients in The States tend to see greater brand adherence than in others. And could you speak to the maybe what you're seeing commercially that is driving the lower guidance?

So what will happen so we'll drive the lower guidance is we'll be able to maintain the trend that we're on. The product is my standard comment is it's like the mailman, it delivers. It just keeps delivering. It will deliver through the end of May. Should a generic come on the market in June, it will be fine. But once it goes off patent, some patients are price sensitive. That's the nature of patients. Some patients are brand sensitive. They want to maintain and that's especially true in the -- I'm sorry, in the epilepsy market. There will be some contracting we'll do with payers to maintain our position, that will be an erosion of price and we will lose some volume. So there'll be a mix of price and volume. But as I said in response to an earlier question, it will be much more sustainable than you would see with a typical retail product. That is the nature of epilepsy products. So that's our expectation at this point in time, and we will continue to give updated guidance as the market evolves.

Thank you. Congrats on another great quarter.

Thank you.

Thank you. Our next question comes from the line of Sudan Loganathan with Stephens. Please proceed with your question.

Congrats on the call. This is Felix Ampomah in for Sudan Loganathan. I have two questions, one on FIRDAPSE. Given the fact that the product has done very well in the marketplace, how well have you penetrated the LEMS market? If you can give color on the percent, that would be very helpful. And then one other question is on the SUMMIT trial for AGAMREE. If you can give us potential expansion opportunities that you can it's out there, that would be helpful for us to model the company very well.

Sorry, the second part of the question was SUMMIT? Was that SUMMIT? Okay. So in January at JPMorgan, we gave a market valuation for LEMS at over and this is I just want to be really clear, this is at today's prices and a conservative view of the market, assuming there are 3,600 patients in the market and the addressable size of the market, not the total 100% of the market. Addressable patients within the market, the market is valued at over $1 billion. The LEMS market is valued at over $1 billon. So to just give a very simple answer, what's our market penetration? Last year, we sold $306 million. So our market penetration would be about 30%. We believe there's tremendous upside in this market. And given the settlement with Teva, should we prevail in the other two suits, we have a runway prevail in a similar way with the other two suits. We have a runway of 10 years and we're all alone. So we think there's tremendous upside in this market and we intend to invest appropriately to see that upside come to fruition. On SUMMIT, we intend to use the SUMMIT study to see if we can make it clarify the differentiation of AGAMREE from other steroids in the market. We believe it's a unique steroid, and we will invest appropriately in that study. Whether or not that can result in a label change, I'll let Gary address that. But there's obviously opportunities to publish, whether it's the posters or publications in journals or not? But Gary, do you want to take the question?

Sure. Thank you. As Rich said, we really are looking to get the long-term safety information that we expect to generate with SUMMIT out into the public. The fastest way to do that is through posters, presentations, abstracts, publications. Label expansion depends upon the overall strength and amount of data. We certainly have planned and look forward to the study, but having that potential the way it is designed, but will depend upon what we see as we go along. However, I believe that the information to be generated on the different parameters of bone health, growth velocity, cardiac safety, fractures, cataract formation will all be very important data to make available as soon as it is ready to the prescribing population.

Thank you.

Thank you very much.

Thank you.

Thank you. We have reached the end of the question-and-answer session. And this also concludes today's conference. You may disconnect your lines at this time. We do thank you for your participation.