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Greetings, and welcome to the Catalyst Pharmaceuticals' Second Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen-only mode. A brief 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, Michael Kalb, CFO. Thank you, Michael. You may begin.

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's second quarter 2024 financial results and business highlights. Richard Daly, President and CEO will be leading 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 $Scientific 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 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 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 2023 annual report on Form 10-K filed with the SEC on February 28, 2024 and in our second quarter 2024 quarterly report on Form 10-Q which we filed yesterday, August 7, 2024 with the SEC. At this time, I'll turn the call over to Rich.

Thanks Mike, and thank you all for joining today's call. Today we'll review our second quarter 2024 corporate performance, provide an update on our regulatory and strategic growth initiatives, dive deeper into individual product performance, and conclude with a comprehensive overview of our financial results. In the second quarter 2024, we achieved outstanding results across our commercial portfolio with total revenues of $122.7 million, a 23.2% increase over Q2 2023, underscored by a record organic revenue growth from FIRDAPSEE. The successful ongoing commercialization of AGAMREE for Duchenne’s Muscular Dystrophy, and the continued solid performance of FYCOMPA. We are reiterating our full year 2024 net product guidance for both FIRDAPSEE and FYCOMPA. AGAMREE continues to deliver strong performance and we are updating our AGAMREE full year 2024 net product revenue guidance from the $25 million to $30 million range we provided earlier this year to the $35 million to $40 million. As a result of our strong performance, we are also updating our full year 2024 total revenue financial guidance. Previously, we guided to a full year total revenue range of between $455 million to $475 million. Due to the solid performance of both FIRDAPSEE and FYCOMPA and the outstanding uptake of AGAMREE, we now believe that we will achieve total revenue at the upper-end of the range. Jeff will provide further details about our commercial performance during this discussion, and Mike will give a deeper dive into the financials later in this call. Now, let's review our regulatory, clinical and business development milestones. First up regulatory, in May, we received US approval for FIRDAPSEE's expanded maximum daily dose of 100 milligrams for treating LEMS up from the previous 80 milligrams. This milestone enhances treatment flexibility for healthcare professionals, aligning with our mission to address the evolving patient needs and optimize outcomes for the LEM patient community. As awareness of this new dosing option grows among patients and physicians, we anticipate a gradual increase in FIRDAPSEE daily dosing over the coming quarters. In parallel, we continued to actively collaborate with our partner in Japan, DyDo Pharma, as we await the next regulatory milestones for FIRDAPSEE, the NDA decision from Japanese regulatory authorities, which is expected at the end of Q3 or the beginning of Q4 this year. In clinical, the summit study, a five year initiative to collect long term safety and quality of life data for AGAMREE, continues to advance. The study, once initiated, will gather long term patient safety and quality of life data, offering a deeper understanding of the product's potential benefit for patients. Site identification and initiation are on track. Annual analysis of patient data is planned, potentially leading to future FDA submissions for updated product labeling, furthering our commitment to enhancing patient care and providing the latest safety information. Moving to business development subsequent to the end of Q2 in July 2024, we entered into a license agreement with Kye Pharma for them to commercialize AGAMREE in Canada. As Kye already holds commercial rights for FIRDAPSEE in Canada. Kye expects to submit an application to Health Canada for regulatory approval of AGAMREE in early 2025. Our growing partnership with Kye is a great example of our commitment to improving access for patients in targeted ex-US markets. In our efforts to build our portfolio, we hear consistent feedback from potential partners. First, our strong commercial execution across a diverse portfolio makes US an excellent partner for companies looking for a US partner. Second, our launch capabilities engender trust with potential partners and are an attractive selling point. Third, our strong balance sheet makes us a formidable negotiator in acquiring new assets. Our strategy remains focused on delivering value for our stakeholders by continuing to execute our buy and build plan. With the ongoing commercialization of AGAMREE, we continue to prove our capabilities in identifying, integrating and launching products that deliver value for patients, providers and payers. We are confident in our ability to leverage the focused execution of our strategic initiatives while maintaining our commitment to advancing patient care, unlocking growth potential and delivering value for stakeholders. Underpinned by our exemplary track record and dedicated team, we believe that we are ideally positioned to drive further growth and achieve sustained success. To summarize, we remain focused on our threefold growth strategy, outstanding execution, portfolio diversification and ex-US expansion through partnerships. First, let's focus on outstanding execution. Our consistent performance continues to demonstrate our commitment to improving the lives of patients we serve and delivering on the promise of growing the company. Second, portfolio Diversification our expanded focus in orphan therapeutics and adjacent orphan assets has yielded increased opportunities and our team is rigorously evaluating each for fit and value. We continue in our tireless efforts to bring on new products and we are excited about our prospects. We remain opportunistic in pursuing the right deals at the right time and we are well positioned to leverage our strong balance sheet to capitalize on accretive or near accretive opportunities. Third, targeted ex-US partnerships. We are building alliances with companies that can leverage our portfolio outside the US in high value markets to bring improved care options to patients and providers. As we add to our portfolio, we expect to build our global presence through these cost effective partnerships. In the second quarter, we successfully navigated an increasingly dynamic business environment delivered on our top line across the portfolio, successfully drove the commercialization of the AGAMREE and expanded our commercial footprint. We will continue to work for the remainder of the year to deliver on our strategy, executing and delivering our plan, diversifying our portfolio and expanding our ex-US partnerships. Now I'll turn the call to Jeff, who will provide a commercial update.

Thanks Rich and good morning everyone. We are incredibly pleased to report the tremendous quarterly performance across our entire commercial portfolio. These results showcase our strategic focus and operational excellence that drive our commercial success. As Rich mentioned, Catalyst had an excellent Q2 driven by the combination of sustained organic growth of FIRDAPSEE, stable revenues from FYCOMPA and the strong commercial launch of AGAMREE. Q2 total net revenues of $122.7 million, a 23.2% growth versus the same quarter last year, positions catalysts well to achieve the upper end of our previously provided combined 2020 total revenue guidance of between $455 million to $475 million. I will now provide a breakdown of Q2 net revenues by product. First, let me start with FIRDAPSEE performance. Q2 net sales of $77.4 million represent a 19.2% increase over the same quarter last year, a direct result of steady new patient enrollments, an annual discontinuation rate below the forecasted 20% and a high refill rate. Net new patients in Q2 were above our forecast and the highest quarterly total since Q1 2022. Prescription approval rates were greater than 90% across all payers, government or private commercial insurers, ordering patterns and refill rates of 95% in Q2 or greater than historical norms of 90%. We expect to return to 90% refill rates in Q3 and Q4 and the resulting 15% or greater growth quarter versus the same quarter in 2023. We remain confident in achieving FIRDAPSE full year 2024 guidance of between $295 million and $310 million. Our demonstrated strategic growth initiatives continue to drive organic growth. We maintain a pipeline of about 500 diagnosed LEMS patients in various stages of their diagnostic journey, not yet on FIRDAPSEE. Half of our new patient enrollments typically come from this group, a prime reason for our ability to consistently deliver 15% to 20% annual growth. Furthermore, LEMS education programs targeting healthcare providers and potential LEMS patients continue to yield a high level of voltage gated calcium channel antibody testing, shortening the diagnostic journey for LEMS patients and leading to more patients being eligible for treatment with FIRDAPSEE. Lastly, as we have mentioned before, more than 90% of small cell lung cancer LEMS patients are undiagnosed. Therefore, we are working with key Thoracic Oncology Institutions to institute screening for newly diagnosed small cell lung cancer patients, resulting in a greater diagnosis of small cell lung cancer LEMS patients. Now let me provide an update on FYCOMPA. Q2 net revenues of $36.5 million further fortifies our revenue diversification. We expect continued stable net revenues in the second half of 2024, in-line with the full year 2024 net revenue guidance of between $130 million and $135 million. As I mentioned last quarter, epilepsy regional account managers are enhancing FIRDAPSEE's share of voice by conducting secondary FIRDAPSEE calls with healthcare providers. This strategy leverages the 45% overlap in physician call points between FYCOMPA and FIRDAPSEE, boosting FIRDAPSEE's visibility. We have seen early indicators that this tactic is working as several patient enrollments have been a direct result of this initiative. Let's shift our focus to AGAMREE. Q2 product net revenues were $8.7 million. Thanks to our dedicated efforts leading up to the March 13, 2024 product launch, we successfully exceeded our initial launch expectations. Thus far, 46% of new enrollments are transitions from EMFLA

Thanks Jeff. Our performance during the second quarter of 2024 has set us on pace for another strong year driven by our solid financial performance and strong execution. With the successful launch of AGAMREE in mid-March, along with the continued performance of FIRDAPSEE and FYCOMPA, we have established the foundation to achieve another revenue record breaking year in 2024. As you heard from Jeff, we had an outstanding quarter in product revenue performance. In the second quarter of 2024, net income before income taxes was $55.8 million, a 15% increase compared to $48.5 million for the second quarter of 2023. We reported GAAP net income of $40.8 million or $0.33 per diluted share, an increase of 8% year-over-year compared to Q2 of 2023 of $37.8 million or $0.33 per diluted share. Coupled with our disciplined approach to cost management, this increase in net income is driven by organic growth of FIRDAPSEE and FYCOMPA and the addition of AGAMREE. Non-GAAP net income for the second quarter of 2024 was $69.6 million or $0.56 per diluted share, which excludes from GAAP net income amortization of inter tangible assets related to our acquisitions of FYCOMPA, AGAMREE and RU

Thanks Mike. The momentum in our portfolio is exciting. FIRDAPSE continues to grow as predicted due to the incredible focus of our field teams. AGAMREE is off to a great launch due to the strong partnership between our medical, commercial and patient advocacy teams and FYCOMPA continues to deliver in the highly competitive market. Because of the relentless focus of our epilepsy team, we had a robust start to this year and we look forward to a strong second half in 2024. We are confident in our ability to capitalize on the opportunities ahead. Our unwavering commitment to advancing patient care, unlocking growth potential and delivering value to our stakeholders remains at the forefront of our efforts, supported by our exemplary track record and our dedicated team, we are ideally positioned to drive further growth and achieve sustained success. At Catalyst, our mission is resolute to pioneer innovative medicines for patients with rare orphan diseases. We look forward to providing updates on our progress and we thank our catalyst team for their commitment and all of you for your continued support. We now turn the call back over to our operator. We look forward to answering your questions. Thank you.

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

Hey, good morning, Rich, Jeff and team. Congratulations on a really nice two q and thanks for taking our questions. I had a couple, first of all, on AGAMREE. Do you believe that there was any bolus in the first quarter of sales because patients or caregivers were waiting for the drug to be, to be approved and launched? And then secondarily, I know you don't have any experience with this yet in this market, but what would you anticipate for summer in terms of, patient and prescriber attendance, if you will?

Charles, thanks for the question. This is something we thought a lot about, especially as we queued up the patients beginning in December of last year. I'm going to turn it to Jeff. Jeff will give you the answer. Thanks.

Hi, Charles. And thanks for your question. There was significant pent up demand leading up to the commercial launch that we had in March. So, yes, to answer your first question, we did see significant interest prior to that, which helped the late launch, the late part of Q1, but also it helped Q2. So again, strong pent up demand there. We are seeing, again, significant demand and interest moving forward. What we see is a lot of the sites, I mentioned it on the call, 80% of the high volume centers of excellence for DMD have enrolled at least one patient, and we've seen greater than 140 healthcare providers prescribe or submit an enrollment for AGAMREE Now, what we expect moving forward are the increase in the depth of interest from those physicians in the centers as we move into Q3 and Q4. So we are excited about the demand so far, and we expect that to continue.

So, Jeff, as far as Charles, question regarding the summer. Do you see any cyclicity so far in the summer, any downward trend or upward?

We see continued strong interest. Charles. So we expect the launch trajectory to stay the same.

And then, Charles, to the first part of your question, this bolus we saw, so we, I think we referred to this in the first quarter call, was as a step-up. So we had a, very light first quarter because we only had two weeks. We expected to have this nice step up, and then what we is a continued traditional curve, and that's what I think we're experiencing. Thanks for the question.

That's helpful. Quick question on FIRDAPSE. Congratulations on the approval of the new higher dose. I had a question about that in terms of the future adoption and persistency, if you will, for patients, will the new dose impact discontinuations either fewer discontinuations due to increased efficacy or satisfaction? Or will it perhaps result in more discontinuations due to less tolerability? What's your sense of how that adoption of that higher dose will go for continued use?

Good question, Charles. And what I can tell you is, again, our annual discontinuation rate is under 20%. And within that, we see the majority of the discontinuations happen within six months of prescribing or initiating therapy on FIRDAPSEE. So at this time, we don't expect the discontinuation to change or increase because of that more flexibility in the dose going up to 100 milligrams.

Okay, last quick question for Rich. Bigger picture strategy. Thinking about some of what you have to bring to the party in terms of, in licensing, do you have a goal for yet this year? And are you focused more on neuromuscular or perhaps rare epilepsy or other areas of, call it neuro innovation? Thanks.

Great question, Charles. So our business development strategy remains the same. We're looking at orphan and differentiated orphan. We're looking at things that are accretive or nearly accretive, and we want to bring in income diversity. So we like the opportunities in neuro because we feel like we have a great footprint there. But we're not stopping. We're not just isolating ourselves to neuro because we believe that the infrastructure that we have behind the outward facing organization, the sales organization, the patient advocacy organization, really supports just about any orphan condition that we would have. So we really feel like we can bring that, that strength to anything. So we like the neurospace, but we really want to move within the neuro space if the opportunity presents itself when it does present itself or outside. So we're looking very broadly, we want to take advantage of those opportunities that come our way.

Thank you. Our next question is from Samantha Semenkow with Citibank. Please proceed with your question.

Hi, good morning, and thanks for taking the question. And let me my congratulations on a really nice and strong quarter. So while still really early in the launch for AGAMREE, I'm wondering how you think about the patient mix will evolve over the next several quarters. It seems the percentages that are coming from menthalaza and prednisone thus far really holding steady from what you shared on the first quarter. And I'm wondering how generic EMFLAVA is fitting into the overall steroid landscape as we'll? And then I have a follow up.

Thanks, Samantha. Those are great questions. I'll turn it right over to Jeff for that.

Hi, Samantha. As far as the mix moving forward, we expect it to remain the same. Like you said, it's pretty flat and similar to what we saw early on in the launch, and it's carried forward. We're very excited about that because it shows the strong interest in the adoption of AGAMREE across all patients that were on steroids, not just a specific segment. So excited about that. As far as the generic entrance for inflaza coming over, and we have not seen an impact so far on adoption of AGAMREE for those patients. So no impact early on.

Got it. Great. And then just on FIRDAPSEE, I wonder if you could share just any updates on the process or the timing of the ongoing FIRDAPSEE. Paragraph four, litigation, and thank you.

Steve, do you want to take that?

Sure. Well, Samantha, as it's generally bad practice to comment on ongoing litigation. I can tell you that we are working diligently to bring the cases to a favorable a manner as possible for catalyst.

Thank you. Our next question is from Jason Gerberry with Bank of America. Please proceed with your questions.

Hey, good morning. Thanks for taking my questions. Maybe just first on Biz Dev, are you generally inclined to do more deals funded with cash and backend success based milestones or talk about maybe appetite to take on some leverage to go after some larger targets that could be more transformational?

Great question, Jason. So we would be looking for opportunities to add to the income statement right. Right now and look for that opportunity. I think we could handle a deal or two with the cash that we have, and then I think we would probably look to dip into the leverage opportunity after that. I think we would be looking to look for more of a transformational opportunity. We like the building of the income diversity as a stepwise function for the company. We think that's the best path forward for us. We like our product mix at this point in time, and we want to build up that strength of the income statement and continue to build the balance sheet. And at that point, I think looking at more of a transformational opportunity. Mike, any comments?

I think you covered it. We have a strong balance sheet. We're ready. Yep. So it sounds like maybe more of a sequencing of deals or something that involved leverage would be after other deals that maybe improve your profitability in your cash flow base, then you'd consider larger transformational deals. Is that the right way to think about it? I think so, yes. When you think about what we're trying to do is build up the strength of the company. We feel like we're really in a great position right now. We feel like we're strong. We know we're strong. And then as we think about the long term of the company, we want to work our way back in the pipeline. So as we build that future orientation of the company, we want to have that strong cash flow, strong balance sheet to be able to take on that transformational opportunity.

Okay. And then if I can ask a question about FIRDAPSE, just the growth that you're seeing, it looks like maybe it's two thirds volume, maybe a third price mix. I assume that that volume is mostly patients that don't have small cell lung as a component of their disease. So is that kind of what you're seeing in terms of the mix and what's driving this kind of 15% to 20% growth?

It's pretty accurate. There. We are seeing from the non small cell lung cancer LEM patients, we've seen steady about low 20% as far as our mix goes, of our total LEM patients that have small cell.So we expect that it's our opportunity moving forward to help that share. As I mentioned on the call, about 90% of those patients, small cell lung cancer LEMS patients are undiagnosed. There are undiagnosed at this time. So we're applying a lot of resources towards helping those patients get diagnosed sooner. So that's where our growth will be coming from. But as far as today, you're accurate about how you described dividing up that growth.

Our next question is from Joon Lee with Truist Securities. Please proceed with your question.

Good morning. This is Osma on for Joon. Congrats on the quarter, and thanks for taking the questions. It was mentioned in the prepared remarks that refill rates were higher than historical norms referred at. Can you explain what led to that and why you expect to return back from 90% in the following quarters? And then also on FIRDAPSEE, how soon after approval of the 100 milligrams dose did you start seeing patients which had a higher dose, and how much of an impact do you think they had in the quarter? Thank you.

Thanks, June, and thanks for your support as well. Jeff, you want to take that?

Sure. As I had mentioned on the Q1 earnings call, we saw some impact from the change healthcare that impacted the refill rate in February. Overall for the quarter in Q1, we were at about 86%. And so traditionally, our historical norms are 90%. In April and May, we saw a recovery there, and we saw higher than the 90% historical norm refill rate, and it actually went up to 95% because of that. So patients that were impacted by that in Q1 filled their rates, filled their prescriptions more in Q2, we expect that to normalize and return to the 90% for Q3 and Q4 and still delivering the 15% or greater growth for FIRDAPSEE. As far as the 100 milligram expansion, it's a great thing for patients and healthcare providers. It really does give them the flexibility to manage and treat I their LEM symptoms. And also just a quick step back, 40% of LEMS patients prior to the label expansion were on 80 milligrams. And those patients, the average daily dose went up by about a milligram, 1.1 milligram. Since that label expansion, we did not expect or do not expect that bolus to just happen immediately. We expect that increase of the average daily dose to reach its full impact in the subsequent quarters. Thank you.

So I just want to clarify a statement Jeff made. 50% growth is 15% growth over Q2 2020. Just to clarify.

So we've turned to that growth as we do a comparison. It's. We look at Q3 2024. The 15% growth we'll see is versus Q3. Q2 '20 or, sorry, Q3 versus Q3 2023. Thanks.

Thank you. Our next question is from Leland Gershall with Oppenheimer and company. Please proceed with your question.

Hi. Good morning, Mike. Congrats on the strong quarter as well. And thanks for taking the question. Just a question again, on AGAMREE these patients with Duchenne often are not seeing their specialists that frequently. If they're medically stable, maybe once every three months, sometimes once every six months. I'm just wondering if you've been seeing patients coming in to switch to a AGAMREE because it's now available, or are they sort of waiting for their next scheduled deployment to change steroids? Just sort of an observational question, if you have any perspective there. Thank you.

Hi, Leland. It's really center of excellence specific. Some sites had that pent up demand, like I mentioned earlier, where they were ready to go as soon as it became commercially available. And then over time, they will wait for other patients to come in and have that discussion. And in other cases, there's outreach going on to the boys living with DMD and their caregivers about switKyeng. So it really is specific. It's dependent on the staff and the physicians, how they want to go, the process that they put in place.

Our next question is from Joe Catanzaro with Piper Sandler. Please proceed with your question.

Great. Hey, everybody, thanks for taking my questions. Maybe, maybe. First one, honing in on for depth a bit more curious. Jeff, what do you attribute the sort of highest level of new patient starts in the quarter, too. I know you cited a couple things. Maybe you could just speak to that, which of those you expect to sort of continue, and maybe which factors maybe fade, if at all? That'd be super helpful. And then I have one quick follow up. Thanks.

Sure. Hey, Joe, as we constantly mention, and I think it's very important in ultra orphan disease, is to maintain that pipeline that we always do. And that's over 500 patients that are in various stages of their LEMS journey. And so we have ongoing conversations between the field and the healthcare providers with those patients. That's where we have the most success. And about 40% to 50% of our new enrollments come from those leads. So as a team, what we constantly focus on are increasing the voltage. Gated antibody testing, the VGCC antibody testing, and then when those patients are tested and if they are positive, we usually can identify where those patients are through our lead sources, and then we try to help those patients by giving those leads to our rants. That's how we ensure that we always have a robust pipeline for future enrollments. And we will continue to do that. And we're always on the search to find additional sources of new leads.

Great. That's helpful. And then my quick follow up, I guess, on portfolio expansion. Rich, I read into maybe some of your comments as there being sort of inbound inquiries to you guys, wondering if you could characterize the current projects that you're diligent. how many of those, what percent originated as inbound inquiries versus outbound inquiries? Thanks.

That's a great question, Joe, really. I think what we've seen now is an uptick in inbound inquiries because of the performance that we see. We're seeing, I guess the best way I could characterize it is that we are, we're looking at the performance of the company and now the execution. We're getting these inquiries because we're a place that people want to place their products. So I would say we're probably at a 30, 30%, 35% of the inquiries, I'm sorry, of the products that we're talking about are inbound, where people are calling us and saying, hey, we'd like to talk to you about an opportunity, and I think that's really exciting. you can go from one product to three products in the space of about 18 months, and you're really hustling for products. And we continue to hustle for products. I want to emphasize that we are working diligently to bring on new products that fit who we are. And our I differentiated, which is critical for us to have companies approach us with products that are in our space and fit. We're excited about that. So I'd say about 30, 35%.