Hello, and welcome to the Catalyst Pharmaceuticals Inc. Fourth Quarter and Full Year 2023 Financial Results Conference Call and Webcast. [Operator Instructions] A question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. It's now my pleasure to turn the call over to CFO, Mike Kalb. Please go ahead, Mike.

Thank you. Good morning, everyone and thank you for joining our conference call to discuss Catalyst's fourth quarter and full year 2023 financial results and business highlights. Leading the call today is Richard Daly, CEO. We are also joined by Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer; and Jeffrey Del Carmen, our Chief Commercial Officer. Further for the Q&A session, 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 2023 annual report on Form 10-K. At this time, I'll turn the call over to Rich.

Thanks, Mike and welcome everyone to the call. First, I'd like to say that our entire team is proud of the work that we do on behalf of the orphan and rare disease communities that we serve, and we're especially excited to be speaking you to today on Rare Disease Day. 2023 was an outstanding year for Catalyst and we are proud of all that the team has achieved. We're pleased to highlight the progress we have made during this transformational year that we just completed, and we are proud to provide insight into the year ahead. But before the team dives into this, I'd like to offer some high level comments. We operate our business around four key pillars. First, commercial execution with supreme focus on patient care and access. Second, portfolio expansion through business development. Third, lifecycle management and partnerships, and finally, financial discipline. Our discussion today will provide you with updates on each of these areas. 2023 was a transformational year for Catalyst. Let's start with our first pillar, our commercial execution. The core FIRDAPSE business remains strong, evidenced by our sustained organic growth. We continue to reach more patients in this market by working diligently to increase awareness of FIRDAPSE and the disease it treats. LEMS, to further enhance our business in 2023, we expanded our commercial portfolio with our acquisition of FYCOMPA. The seamless integration of FYCOMPA franchise into our company has yielded promising results. We are pleased with the product's performance. This addition enriches our portfolio by enhancing income diversity, fostering stable revenue streams, and fortifying our balance sheet in preparation for future acquisitions. Our second pillar, portfolio expansion. In addition to FYCOMPA in the third quarter of 2023, we licensed the North American rights to AGAMREE, a U.S. NDA filed asset for Duchenne's…

Thanks, Rich and good morning, everyone. First, I want to commemorate the Rare Disease Day in all the people touched by rare diseases. We are pleased with the outstanding performance delivered in 2023, highlighted by full year combined total revenues of $398.2 million, exceeding the upper end of guidance of $395 million. This remarkable performance was fueled by FIRDAPSE reaching an all-time high of $258.4 million, and the consistent strong contribution from FYCOMPA, which generated $138.1 million in net revenues. 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 revenues amounted to $69.8 million, showcasing a remarkable year-on-year growth, 15%. Moreover, the overall net revenues of 2023 displayed a robust 20.8% growth compared to the previous year, propelled by consistent initiation of new patients in an annual low discontinuation rate, trending below 20% consistent with previous years and 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 copay of less than $2. We are confident in the continued FIRDAPSE organic growth in 2024 will be between 15% and 20%, building upon the momentum from 2023. This growth will primarily be fueled by several key factors. Firstly, the conversion of a portion of the 500 identified LEMS patients FIRDAPSE treatment will play a significant role. Additionally, the anticipated approval of the 100 milligram expanded label in June, 2024 should help contribute to this growth trajectory, providing patients the opportunity for a higher daily dose when appropriate. Furthermore, the expansion of educational programs targeting small cell lung cancer, LEMS healthcare providers is poised to accelerate LEMS diagnosis rates. Lastly, our efforts…

Thank you, Jeff. I would first like to discuss our development efforts to increase the indicated maximum dose of FIRDAPSE. As previously reported in October of 2023, the FDA accepted for review the company's supplemental new drug application to increase the indicated maximum 80 milligram daily dosage of FIRDAPSE to 100 milligrams for the treatment of Lambert-Eaton Myasthenic Syndrome, or LEMS. Catalyst continues to anticipate approval of the application on or about June 4th, 2023, which is the PDUFA action date granted by the FDA. About 40% of patients are on the current indicated maximum dose of 80 milligrams per day, and their physicians would like the option to increase the daily dosage above 80 milligrams per day based upon disease progression. Catalyst believes that this expansion of the FIRDAPSE label, if approved, would address an important opportunity for patients and their healthcare providers and may lead to about a 5% to 10% increase in the prescribed average daily dose of FIRDAPSE over time. In 2023, we focused on expanding our intellectual property portfolio for FIRDAPSE, resulting in the granting of two additional patents for FIRDAPSE. These patents were issued in 2023 and 2024, bringing the total number of patents protecting FIRDAPSE to nine. This further strengthens our intellectual property estate for FIRDAPSE, which has patent protection to 2037. Catalyst expansion to global markets is continuing to progress. Our partner in Japan DyDo Pharma achieved a notable milestone in December of 2023, having successfully submitted their NDA for FIRDAPSE to the Japanese PMDA. We anticipate a priority review cycle time of nine months, and if approved a potential launch in early 2025 with 10 years of market exclusivity in Japan after approval. Next, I'll move on to our newest approved product AGAMREE for the treatment of Duchenne Muscular Dystrophy, or…

Thank you, Steve. Catalyst's fourth quarter and full year 2023 financial performance resulted in another record breaking year, driven by exceptional execution by all facets of our business. In addition to our outstanding results, the company successfully closed two transactions in 2023, the acquisition of the U.S. rights to FYCOMPA and the North American license for AGAMREE. Both transactions provide the company with additional opportunity to continue to create incremental net positive cash flow through disciplined execution. I'd like to take a moment to reiterate some of the key accounting implications and accounting treatment of AGAMREE transaction. As a result of the FDA approval of AGAMREE on October 26th, 2023, we made a $36 million milestone payment to Santhera in the fourth quarter of 2023, which was capitalized on the balance sheet as an intangible asset and will be amortized on a straight line basis over its estimated useful life of 10.5 years. I would like to reinforce that based on the timing of the AGAMREE approval, amortization expense was prorated in the fourth quarter of 2023. Catalyst also made a strategic equity investment in Santhera, which was recorded with an initial fair value of $13.5 million and will be mark-to-market value on a quarterly basis using the observable market price. Now on to 2023 results. Our total net revenue for 2023 -- sorry -- for 2023 was $398.2 million, an 85.9% increase when compared to total net revenue of $214.2 million for 2022. Product revenue net for 2023 for FIRDAPSE was $258.4 million, a 20.8% increase year-over-year compared to $213.9 million for 22. Product revenue net for FYCOMPA was $138.1 million for '23. As mentioned in our press release, 2024 FYCOMPA net product revenue will be adversely affected by gross to net adjustments. In 2023 our gross to net…

Thanks, Mike. I'd like to thank the leadership team and all our employees at Catalyst for the effort they put forth to deliver such outstanding results in 2023. We are off to a strong start in '24 with continued momentum to sustain our long-term durable growth. We continue to be committed to excellent execution. We are focused on increasing the scope and scale of our business through our disciplined approach in identifying derisked innovative therapies that address critical unmet medical needs in the rare orphan, neuro and adjacent rare orphan therapeutic areas. We also hope to increase our geographic footprint through ex-U.S. partnerships. In short, we are prepared to invest in opportunities that add value to Catalyst, further grow our portfolio, and provide improved care for patients. Our team is dedicated to improving the lives of more patients by working on key lifecycle management projects that have the potential to expand the use of the products we offer. We believe our financial rigor, the strength of our financial results and our cash flow position us to advance our critical initiatives. In summary, we are focused on our strategic priorities for 2024. Number one, continue to deliver double-digit growth for our portfolio. Number two, successfully launch and commercialize AGAMREE. Number three, expansion of our product portfolio and our geographic partnerships in the CNS orphan and orphan adjacent therapeutic spaces. And we look forward to providing you with updates on our progress. At this time, we'll turn it back -- turn the call back over to our operator, Kevin. Kevin?

Thank you. We'll now be conducting a question-and-answer session. [Operator Instructions] Our first question is coming from Charles Duncan from Cantor Fitzgerald. Your line is now live.

Hey, good morning, Rich and team. Congratulations on a transformative year and appreciate the guidance going forward. I had a quick question on FIRDAPSE with regard to that label expansion. Steve did a great job, helping us understand the percentage of patients really that are looking for a higher dose. But I'm wondering what is the driver of that? Would there be anticipated short-term efficacy or long-term durability?

Charles, we believe that it will be long-term durability. There's really two driving forces behind that. The first is disease progression as these patients age. And so, they very gradually need a higher dose. The other thing also is that there are a handful of fast metabolizers out there, who have higher weights that also need a higher dose. And so we'll be able to support those patients if and when the FDA approves this 100 milligram maximum daily dose.

Got it. And then with jumping over to AGAMREE, one quick question for Jeff or others on the team. I guess I'm wondering if you could provide a little bit more color on the registry. I think you mentioned that you would be starting that and anticipated size and timing. Any updates from that that you anticipate that could drive awareness of vamorolone value proposition versus other standard-of-care? Thanks.

Charles, the registry -- the size is going to depend on how many patients agree to join the registry. It is voluntary. We anticipate that a significant number of patients will join the registry, and be tracked for as long as they are to remain in the patient registry. In terms of duration, it will be carried on for many years as we collect data for long-term safety evaluation of the AGAMREE.

Gary, do you want to make any commentary about the number of sites, excuse me, number of sites as well?

So we will be looking at all of the sites that potentially enroll, and commercial or to have commercial AGAMREE patients and evaluate them for their ability to collect data rigorously in the registry. So at the present time, we haven't set a specific number, but we'll, again, as Steve said, look at how many patients are prescribed, what the locations are in order to determine site and patient numbers.

But you'd say -- just a sense of, it's a pretty broad -- we're looking at a pretty broad-based approach though. Would that be safe to say?

Yes.

Right. Okay. So we'll follow up. I am clear, Charles.

Got it. Thanks. Thanks Rich for the advice.

Thank you. Next question is coming from Joon Lee from Truist Securities. Your line is now live.

Great. Congrats on another strong quarter and thanks for taking our questions. I'm really impressed that you're able to provide guidance on AGAMREE, which has yet to launch. I can only imagine that you must be getting a lot of inbound interest to be able to provide a guidance. Is this why you think just 10 additional salesforce will be sufficient to launch into a 10,000 plus DMD patient space? And will it be more word of mouth awareness or do you have any DTC efforts in mind, and I have a quick follow up?

Sure. So thanks for the question, Joon. We -- with DMD, it's a very defined market. There are only primarily 250 prescribers that prescribe 95% of the corticosteroids for boys living with DMD. Those 250 prescribers are housed in about 90 to 100 centers of excellence. So it's a focus call point that our team has the capacity, the team that was selling FIRDAPSE had the capacity to handle and handle effectively. And these are very experienced and tenured people. So we're very confident in that structure. Now, as far as the pent-up demand that you had mentioned, and it sounds like -- as I mentioned on the call, we've been accepting precommercial launch enrollments since December. And based on those enrollments and the number of enrollments that we've seen and also the other stakeholders that have talked about their interest in going on AGAMREE, we do believe we're confident in the number that we provided. So that's one thing. As far as DTC, we have non-personal promotion along with our salesforce efforts to help, provide education on AGAMREE as well as DMD. That'll be focused on not only the physicians, but also to other stakeholders like patients and caregivers.

Great. Thanks for the color. And on the continued strength of the -- your FIRDAPSE franchise, you mentioned conversion of 500 additional LEMS patients that you've identified and the anticipated approval of the higher 100 milligram dose. But how does a small cell lung cancer LEMS patients factor into that guidance? Is that really in the guidance, or is it more upside or help us navigate that component of the population? Thank you.

So Joon, great question about that and it is factored in. The small cell lung cancer LEMS opportunity is factored into the growth potential, but we also see it as upside from there, too. Like I mentioned, about 80% of these small cell lung cancer LEMS patients are unfortunately undiagnosed at this point. So our efforts to accelerate that diagnosis or the diagnosis for these patients will increase the addressable market over time, which will help with our growth, too.

Great. Congrats again on the strong quarter. Thank you.

Thank you. Thanks very much, Joon.

Thank you. Next question is coming from Joe Catanzaro from Piper Sandler. Your line is out live.

Yeah. Hi, guys. I appreciate you taking my question and congrats on all the great progress here. So, Steve, I think you mentioned the percent of patients that are on the current maximum dose of FIRDAPSE. But I think you've previously said that you would expect even patients on lower doses to be taken to higher doses. I wonder if that's still your expectation and if that's sort of factored into the FIRDAPSE guidance for 2024. Thanks, and I have one quick follow up.

I'll let Jeff address the -- factored into the guidance, but with regard to your question about patients being on doses below 80 milligrams per day, yes, there are patients on that. The neuromuscular physician community is somewhat conservative. It's a go slow -- start low and go slow mentality with regard to medication treatment. Many of them like to leave what I'll call some headspace in terms of the dose that patients are receiving so that if the patient gets worse, they can increase the dose. With the increase in the daily maximum dosage, we would expect some of those conservative physicians who have been concerned about the overall efficacy of say a 60 milligram dose that a patient may have been receiving may be more likely to increase the dose knowing that they still have more room to further increase the dose for those patients. So, that is -- you are correct that some of those patients at below 80 milligrams may increase as well. And Jeff, did you want to address factored into--?

Sure, Joe. Before I get into that question, just wanted to reiterate the percentages that, 40% of our patients are currently on 80 milligrams. Then there's an incremental 3% of patients are on greater than 80 milligrams currently. Over time, we do expect the average daily dose to increase and that increase, the gradual increase is factored in to the forecast. But keep in mind with the PDUFA date for this June, it's over time. So it's just the impact from June through December of this year.

Okay. Thanks. That's helpful. And then maybe my follow up question on sort of future cadence of business development and maybe one for you, Rich. As we look back at sort of the historical approach to business development, how are you thinking about potential opportunities moving forward?

So, great question. So obviously we feel that our balance sheet is prime right now and we are really confident and Preethi and her team are continuing to look. We have a very wide aperture. So we consider ourselves an orphan company, obviously in the CNS space, so that would include epilepsy as well. And we're looking at those opportunities that are not only just in CNS because we believe our infrastructure is one that supports getting the patient on and keeping the patient on. That's critical. We see that as a foundational element of who we are as a company, but we believe that infrastructure is transferrable to other orphan states outside of CNS, metabolic, cardiovascular, et cetera. So our aperture is quite wide. So we believe our opportunities are very, very broad. So we continue to scan the landscape. We have a number of opportunities that we're evaluating at a very deep level and we're continuing there and we're, I would say, appropriately aggressive in our search for new opportunities that we believe fit with who we are and our ability to serve the patient community.

Thank you.

Thank you. Our next question is coming from Leland Gershell from Oppenheimer. Your line is now live.

Yes. Good morning, everyone. Thanks for taking my questions and great to see the strong guidance on AGAMREE, as well as overall. Just a few questions from me. Actually on FYCOMPA, looks like -- with the guidance, looks maybe a bit more flattish year-on-year and '24 than I think previously company indicated maybe sort of a mid to high single digits growth. Just wondering if -- any dynamic there we should be aware of. And then second, also on FYCOMPA, is there any update with respect to IP exclusivity? I think kind of base case we have this protected until sort of spring of '25 and then that extended '26. Just wondering if there's any update on the IP there. Thank you.

Sure, Leland. So, Mike will take the first part and Steve will take the second part.

Sure. Morning, Leland. Thanks for the question. On FYCOMPA, as we mentioned in the press release as well as on the call, our arrangements now with wholesalers and distributors and government agencies are through us as opposed to for 2023, they were through [indiscernible] and as is common for companies of our size, smaller companies, smaller product portfolios, those rates are going to be slightly higher.

Hi, Leland. With regard to the intellectual property, we have completed our work with FDA to [technical difficulty] expiration date of May of 2025 is the -- will be -- the end of exclusivity for that patent for the composition of matter. However, we are still evaluating whether or not to enforce the polymorph patents and we hope to conclude that evaluation in the near future.

I just want to make a final comment about FYCOMPA. One of the reasons why we like this asset a lot is for the financial opportunity. We believe that this compound has life, significant life beyond its patent expiry because of the stickiness of epilepsy drugs. Patients with epilepsy do not like to change drugs, even post patent expiry. So there is a great willingness for the patient to continue on their branded medication [technical difficulty]. So there is a significant opportunity for this compound post expiry to continue to deliver good return for the company. And that was baked into our calculus when we bought the product. So we continue to see this as a very good opportunity to serve patients, and to build the balance sheet and again, help us to build our portfolio as we go forward. So we see this as a really good, smart financial play and a good play for patients as well.

All right. Thanks. That's very helpful. Then just one question on FIRDAPSE, if I may. I think, the mention of about 500 remaining diagnosed, but untreated patients, if I have that correctly. Just wondering if you could point at any particular hurdles that you may face in converting those untreated, but diagnosed patients onto FIRDAPSE. Thank you.

So Leland, 50%, about 50% of our new enrollments each month or any given quarter come from these leads. These patients that are diagnosed, but never have been treated with FIRDAPSE. We use a variety of sources to continually backfill those patients. So as one patient gets converted on to FIRDAPSE, we find new leads to backfill those patients. So we always try to maintain those 500 or so patient leads. As far as hurdles to get on, a lot of times it's just -- it's a matter of these patients waiting to get in to see their physicians. Whether it's a three month or six month timeframe that they're waiting to get in. That's primarily it. There are some patients within these 500 that have been on the -- or a patient lead for over a year. But most of these -- the newer leads can be converted within 30 days. That's how we see it. But again, we backfill those patients, converted with new leads over time.

I think, Leland, it's all part of the -- what we would call, this is the patient journey. And so it's really dependent upon the physician and the patient where they come in and how aggressive they want to be with their own therapy. And so once we identify them, we keep calling on the physician and working with the physician to expedite therapy if it's appropriate, but it really comes down to physician. But once we identify the physician who has the patient, we can then begin to help the physician understand the disease state and then the patient journey and how FIRDAPSE can help that patient.

I appreciate the color. Thank you.

Thank you. End of Q&A:

Thank you. We reached the end of our question-and-answer session. And ladies and gentlemen, that does conclude today's teleconference and webcast. We do thank you for your participation. You may have a nice day.

Thank you.