Catalyst Pharmaceuticals, Inc. Q1 FY2024 Earnings Call

Greetings and welcome to the Catalyst Pharmaceuticals First Quarter 2024 Financial Results Conference Call. As a reminder, this conference is being recorded.

Thank you. Good morning, everyone, and thank you for joining our conference call to discuss Catalyst's first 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 be presenting. Additionally, Dr. Steven Miller, our Chief Operating Officer and Chief 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 first quarter 2024 quarterly report on Form 10-Q, which was filed yesterday with the SEC. At this time, I'll turn the call over to Rich.

Thanks, Mike. Welcome to the Catalyst Q1 2024 Financial Results Call. Today, we will discuss our excellent performance in the first quarter as well as our continued focus on execution, provide an update on clinical and regulatory milestones, delve into our portfolio diversification approach, and finally, provide insight into our continued delivery on financial discipline. In Q1 '24, we delivered another quarter of strong performance with total revenues of $98.5 million. This performance, along with our January equity raise, enhanced our substantial cash position, which totaled $310.4 million as of March 31, 2024. During the quarter, our agility and focus on execution enabled our team to respond to opportunities in the market and drive continued growth in FIRDAPSE, successfully launch AGAMREE and deliver on consistent performance for FYCOMPA. I want to congratulate our team for continuing to meet the challenging targets we set for them. We are poised for promising growth as demonstrated by the significant milestones achieved. FIRDAPSE continues to deliver strong double-digit growth, a tremendous testament to our ability to drive the sustained success of the brand for more than 5 years. Additionally, we successfully launched AGAMREE for Duchenne's muscular dystrophy, or DMD, on March 13. Although just over 2 weeks of launch performance are included in our Q1 financials, we are very excited by AGAMREE's performance and potential when reviewing AGAMREE's uptake trends. Based on physician and patient response, we are experiencing a great deal of momentum, and this speaks to the tremendous market conditioning we did prior to the launch as well as our ability to successfully integrate AGAMREE into our Catalyst Pathways patient services infrastructure. Although it's early in the launch, we believe AGAMREE is positioned for a significant step-up in Q2 and beyond. Jeff will provide further details about product performance during his discussion. Let's transition to product development and regulatory. For AGAMREE, we are preparing to launch our previously reported long-term registry study of AGAMREE, which we have rebranded as the SUMMIT study. This study aims to gather long-term patient safety and quality-of-life data, offering a deeper understanding of the product's potential long-term benefits for patients. We recognize that a fundamental challenge in the traditional steroid use in treating DMD is in the safety impact for the long-term use of these steroids. Long-term effects can cause doctors and patients to: number one, delay initiation of steroids; number two, dose too low to accrue the therapeutic benefits of steroids; and number three, discontinue steroid use too early. We have initiated the SUMMIT study designed in accordance with FDA guidance for the use of real-world data to evaluate the long-term safety and quality-of-life benefits of AGAMREE. The AGAMREE profile may offer patients and physicians the opportunity to start therapy sooner, optimize the effective dose and stay on therapy longer with the goal of potentially improving the care of patients with DMD. Moving on to FIRDAPSE. We are eagerly awaiting the June 4, 2024, PDUFA date of our supplemental New Drug Application seeking to increase the FIRDAPSE maximum daily dose from 80 milligrams to 100 milligrams. If our sNDA is approved, we believe that it creates the potential to provide increased therapeutic flexibility for patients and the physicians who treat them. Internationally, we continue to work with our partner in Japan, DyDo Pharma, as they await word from Japanese regulatory authorities on the FIRDAPSE Japan NDA that they filed late last year, with a decision expected around the end of Q3, beginning of Q4 this year. Next, regarding business development and portfolio diversification. We are working on 3 fronts to increase our BD opportunities. First, expanding our portfolio. We are aggressively exploring opportunities to expand our portfolio in this orphan CNS and orphan-adjacent spaces. We are taking a comprehensive approach to decision-making, ensuring alignment with our overarching strategy for optimal success and our long-term vision. Second, we are working to expand our partnerships. We are actively pursuing opportunities to expand the partnerships for FIRDAPSE in the APAC region beyond Japan and looking to enter agreements in Lat Am. There is a recognized need for LEMS treatment options like FIRDAPSE in both regions. Additionally, we are pursuing partnerships for AGAMREE with ex-U.S. partners in North America. Third, we are exploring opportunities to diversify and expand the use of our existing products through life cycle management. Our teams are fully immersed in developing an AGAMREE life cycle plan, seeking to open up new avenues for growth for this product, and we expect to provide an update on this project during the second half of this year. Our accomplishments in the first quarter reflect our strong performance with steady product revenue contributions and a promising product launch, further adding to our financial strength and driving our growth. We are grateful to our team for delivering excellent results this quarter, which gives us further confidence as we reaffirm our full year 2024 total revenue guidance in the range of $455 million to $475 million. Mike will provide additional details on our financial performance during his discussion today. Since the outset of 2024, we have surged forward with formidable momentum, laying down solid groundwork to drive short- and long-term value. We remain excited about the journey ahead. I'll now turn over the call to Jeff Del Carmen, who will provide an overview of our commercial performance.

Thanks, Rich, and good morning, everyone. Fueled by the successful execution of our strategic growth initiatives across our entire commercial portfolio, we are exceedingly pleased with our first quarter 2024 performance. As Rich mentioned, Catalyst had an excellent start to 2024 driven by the combination of sustained organic growth of FIRDAPSE, stable revenues from FYCOMPA and the strong initial commercial launch of AGAMREE. Q1 total net product revenues of $98.4 million positions Catalyst well to achieve our 2024 full year total revenue guidance. First, let me start with FIRDAPSE. Q1 2024 net sales were $66.8 million, representing a 16% increase over the same quarter last year, in line with our forecasted growth projections, a direct result of a sustained cadence of new patient starts, high compliance and consistent low discontinuation rates. Net new patients in Q1 2024 were the highest total since Q1 2022. We believe that these facts demonstrate that our underlying FIRDAPSE business is solid. Prescription approval rates were greater than 90% across all payers, government or private commercial insurers. As expected, net product revenues in the first quarter reflected the typical Q1 seasonality and the reset of deductibles and co-pays at the beginning of the year. In addition, some new patient and refill prescription adjudications were temporarily impacted by the Change Healthcare cyberattack, particularly in February. Given the intensely symptomatic nature of LEMS, we exercised Catalyst's no-patient-left-behind approach and provided 5 days of free drug to any FIRDAPSE patient who could not fill their prescription. It is our mission to ensure continuity of care for the patients we serve, and we believe that what we did supports that commitment. I am also glad to report that at the end of the day, we subsequently received reimbursement for all the 5-day bridge prescriptions provided. The FIRDAPSE refill rate was temporarily affected in February, but we saw a rapid rebound in filled prescriptions in March back to historic norms. Our strategic growth initiatives will continue to drive organic growth moving forward. We maintain a pipeline of approximately 500 LEMS patients in various stages of their diagnostic journey who are not yet on FIRDAPSE. Tracking these LEMS patients is a prime reason for our ability to consistently deliver 15% to 20% annual growth as half our new patient enrollments typically come from this group. Furthermore, LEMS education programs targeting health care providers have already resulted in consistently higher voltage gated calcium channel antibody testing rates, which will shorten the diagnostic journey for LEMS patients and lead to more patients being eligible for treatment with FIRDAPSE. Now let me provide an update on FYCOMPA. We achieved solid Q1 net revenues of $30.4 million, further fortifying our revenue diversification. We expect continued stable net revenues for the rest of 2024. Further, all epilepsy regional account managers have been trained on FIRDAPSE and LEMS. And moving forward, these RAMs will conduct secondary calls with health care providers that account for the 45% overlap in FYCOMPA and FIRDAPSE physician call points, further increasing FIRDAPSE's share of voice and the ability to identify new LEMS patients. Let's turn to AGAMREE. We successfully commercially launched AGAMREE on March 13. Early indicators of new patient enrollments show strong interest, exceeding initial expectations. This is primarily due to penetration of both the EMFLAZA and the prednisone patient segments. Payer approval rates are greater than 90%, averaging less than 30 days to approval. Based on our experience with FIRDAPSE, we expect this to improve over time. Further, to date, 63% of the DMD centers of excellence have enrolled at least one patient on AGAMREE. Since most of the prescriptions for DMD patients are written by doctors who practice in these centers of excellence, we believe this is a very positive sign for the acceptance of AGAMREE. In summary, we believe that we are making meaningful progress on sustained organic growth for FIRDAPSE, continuing to generate stable net revenues for FYCOMPA and exceeding our expectations for our revenues for AGAMREE. We are highly focused on continued commercial execution excellence and leveraging our strategies to maximize our demonstrated capabilities. I want to thank the entire team at Catalyst for their unwavering commitment to patients and look forward to continuing with a successful 2024. I will now turn the call back over to Mike.

Thank you, Jeff. As Rich noted, our performance during the first quarter of 2024 has set us on pace for another strong year driven by our solid financial performance, financial discipline and strong execution. We remain steadfast in our commitment to driving growth, leveraging strategic partnerships and expanding our portfolio to capitalize on emerging opportunities throughout the year. We intend to continue our commitment to grow through strategic partnerships and portfolio expansion in 2024. To support this objective, in January 2024, Catalyst sold 10 million shares of its authorized but unissued common stock to raise net proceeds of approximately $140.7 million for the purposes of funding potential future acquisitions and for general corporate purposes. Our total revenues for the first quarter of 2024 were $98.5 million, an approximate 15% increase when compared to total revenues of $85.4 million for the first quarter of 2023. Product revenue net for our lead product, FIRDAPSE, were $66.8 million, a 16% increase year-over-year compared to $57.5 million in Q1 2023. Product revenue net for the first quarter of 2024 for FYCOMPA increased to $30.4 million compared to $27.8 million for the portion of the first quarter of 2023 in which we recognized revenue following our acquisition of the product rights in late January of 2023. As a reminder, we anticipate that 2024 revenue for FYCOMPA will be affected by differences in variable consideration compared to 2023 when revenues were booked under a size-cost arrangements with distributors and government agencies. Effective January 1 of this year, these costs are tied to arrangements between the company and those distributors and agencies, which are higher. Finally, we launched AGAMREE in mid-March. And for the short time during the first quarter that the product was commercially available, product revenue net was $1.2 million. In the first quarter of the calendar year, like many companies in our industry, we are impacted by the reset of patient insurance deductibles. Further, in the first quarter of 2024, we reported expenses related to our annual commitment to make charitable contributions to foundations that fund LEMS patient support programs throughout the year. Cost of sales was approximately $12.5 million in the first quarter of 2024 compared to $9.9 million in the first quarter of 2023 and consisted principally of royalties. As a reminder, royalties for FIRDAPSE increased by 3% when net product sales exceed $100 million in any calendar year. As a result, we expect cost of sales to trend higher as this year progresses. Additionally, I would like to note that AGAMREE also carries a royalty in the range of low double digits to mid-20%, depending on sales achievements within a calendar year. Further details on our royalty obligations for AGAMREE are reported in our Q1 '24 Form 10-Q. Research and development expense was $2.6 million in the first quarter of 2024, slightly down from $3.6 million in the first quarter of 2023. At this point, as noted in the 2024 guidance we provided earlier this year, we believe that R&D expense for 2024 will be similar to 2022. SG&A expenses for the first quarter of 2024 totaled $46.9 million compared to $29.7 million in Q1 2023 as G&A expenses moderately increased as a percentage of total operating expenses to 66% for Q1 2024 compared to 60% for Q1 2023. The overall difference in SG&A expenses in the first quarter of 2024 was principally due to the incremental selling and marketing expenses to support the launch of AGAMREE and operating expenses for FYCOMPA being present for a full quarter in Q1 2024 when compared to approximately 2/3 of Q1 2023 as well as the noncash stock-based compensation charges of $3.9 million relating to the retirement of the company's former CEO and CFO based on the terms of their respective separation agreements with the company. Our effective tax rate for the first quarter of 2024 was 20% compared to 20.8% for the first quarter of 2023. Although lower in the first quarter of 2024, we still expect our annualized effective tax rate to be slightly higher than the annual effective tax rate in full year 2023 of 24.4%. For 2024, the difference to the statutory federal income tax rate of 21% was primarily driven by state income taxes and anticipated annual permanent differences. 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. Net income before income taxes for the first quarter of 2024 was $29.1 million, a 22% decrease year-over-year compared to $37.3 million for the first quarter of 2023. We reported GAAP net income for the first quarter of 2024 of $23.3 million or $0.20 per basic and $0.19 per diluted share, a decrease of 21% year-over-year compared to GAAP net income for the first quarter of 2023 of $29.6 million or $0.28 per basic and $0.26 per diluted share. The decrease in both net income before income taxes and GAAP net income reflects a full quarter of operating expenses pertaining to FYCOMPA as well as substantial incremental operating expenses to prepare for the launch of AGAMREE. It also includes one-time noncash stock-based compensation expense that I mentioned earlier. Non-GAAP net income for the first quarter of 2024 was $46.8 million or $0.40 per basic and $0.38 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 $8.2 million; the income tax provision of $5.8 million; and depreciation of $86,000. This compares to non-GAAP net income in the first quarter of 2023 of $46.8 million or $0.44 per basic and $0.41 per diluted share, which excludes from GAAP net income, amortization of intangible assets related to our acquisitions of FYCOMPA and Ruzurgi of $6.5 million, stock-based compensation expense of $2.9 million, the income tax provision of $7.7 million, and depreciation of $69,000. As previously reported, we ended the first quarter of 2024 with cash and cash equivalents of $310.4 million compared to $137.6 million at December 31, 2023. The increase in cash of $172.8 million was largely driven by our January 2024 capital raise, which added approximately $140.7 million in net cash proceeds. The remaining increase in cash is driven primarily by $31.9 million in cash generated from business operations. We believe our current funds 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 first quarter 2024 financial performance may be found in our quarterly report on Form 10-Q, which was filed with the Securities and Exchange Commission yesterday and can be found in our Investor Relations page on our website.

Thanks, Mike. At Catalyst, our mission is resolute: to pioneer first-in-class innovative medicines for patients with rare and difficult-to-treat diseases. With an expanding portfolio of commercial products and a track record of operational excellence, we are primed for ongoing success in delivering transformative therapies to the market. We look forward to providing updates on our continued progress. At this time, I'll turn the call back over to the operator, and we look forward to answering your questions.

Our first question comes from Joon Lee with Truist Securities.

This is Jeremy on for Joon. Can you just share any specific doctor or patient feedback on the AGAMREE experience? And then any further updates on your BD plans? How far along are you in that search? And is there a specific therapeutic area you're targeting, such as epilepsy?

Thanks for the question. Jeff, do you want to handle the first part?

Sure. It's a little early to get some specific feedback about physicians and the efficacy that their patients are seeing with AGAMREE. What we can tell you is the physicians and the patients are extremely happy with the process to get on AGAMREE and to get the drug. So that's first and foremost. But we do anticipate feedback about their patients here shortly.

Great. As to the second part of the question, so we are aggressively pursuing opportunities, as we mentioned on the call, in the orphan space and what we call the orphan-adjacent. So we believe our infrastructure is one that really supports patient care. So when you think about what we did in the quarter and really making sure that our patients had uninterrupted care, I think it really speaks to the strategic advantage we have as a company. So we believe that our infrastructure supports essentially any orphan opportunity. And so we are out aggressively seeking opportunities across the orphan space. We believe what we're seeing our continued success, our ongoing success with FIRDAPSE and our burgeoning success with AGAMREE really supports that. So we are looking pretty far and wide in the orphan space. So I'd say we're essentially agnostic because we believe that our infrastructure would support just about any orphan opportunity.

And our next question comes from the line of Charles Duncan with Cantor Fitzgerald.

Rich and team, congratulations on a nice quarter to start the year. I had a couple of them, one on AGAMREE, one on FIRDAPSE and then one BD. So on AGAMREE, you mentioned new patient enrollments, very good to hear. Can you give us some color on where you're taking patients from? Is it more EMFLAZA? Or is it more prednisone? And any color on that as well as pacing of information from the new SUMMIT study?

Great. So Charles, great. Thanks for the questions. And Jeff, do you want to handle the first part of the question? And we'll have Steve handle the second part.

Sure. When we look at the enrollments that we're getting so far for AGAMREE, 47% of our new enrollments are from patients that were currently on EMFLAZA. 43% of our patients were coming from patients that were on prednisone. And there are just a handful or a smaller percentage that were naive patients to any corticosteroid.

And then, Charles, your second question, I believe you said the pacing of information from the SUMMIT study, is that correct?

Yes.

Sure. Thank you, Rich, and thank you, Charles. The SUMMIT study is scheduled to be a 5-year study. We anticipate doing analysis approximately once a year, but it would depend on the nature and significance of findings that we observe during the course of the study. At the very least, the information will be published and made available to the medical information staff. And at the most, it will be submitted to the FDA under the FDA's guidance for real-world data and its use for updating product labeling.

Okay. That's helpful. And then quickly on FIRDAPSE, looking forward to the PDUFA date, possible dose expansion. Could you remind us of, call it, roughly what percentage of patients are at maximum dose? And how that decision, if it is positive to expand dosing, might impact demand for FIRDAPSE?

So Charles, currently 40% of active FIRDAPSE patients are on the maximum daily dose of 80 milligrams. Additionally, 4% of patients are on a dose greater than 80 milligrams, which requires direct approval through the specialty pharmacy. We believe there is considerable potential for patients to adjust to their optimal dose. Over time, we expect the average daily dose, which is currently around 61 milligrams, to rise to about 65 or 66 milligrams. We anticipate this increase to occur over the next 12 months following the label expansion.

Got it. Final question, BD, returning to the earlier discussion. It seems you're focused on the AGAMREE launch. Given the interest in additional assets, is there a plan to pursue that in 2024, or is it more dependent on available opportunities?

It's a great question, Charles. We continue to explore various opportunities under Preethi's leadership. Our main focus is to successfully launch AGAMREE, which we are doing well. However, we want to ensure we pursue the right opportunities. We aim to expand the CNS franchise and keep an eye on market opportunities to make the right deals at the right time. It's important that we capitalize on the opportunities that arise. While I believe we can engage in additional deals, the timing and the suitability of those deals must align with our goals. We plan to approach this wisely to benefit our patients, leverage the talent within our organization, and, of course, support our shareholders.

And our next question comes from the line of Jason Gerberry with Bank of America.

One BD question for me and then one AGAMREE question. Just on the BD front, I'm just curious if there's a general profile range in terms of size of deal and stage development that you look at. Would you be willing to bring in an asset that wouldn't deliver sales and profit in the short term? And how FIRDAPSE litigation outcomes sort of shape your M&A aperture? And then on AGAMREE, maybe a little surprised just to hear life cycle management discussion this early in the launch. Is this something that you'll update in the second half that could be beneficial within the IP window of AGAMREE or the regulatory exclusivity window of AGAMREE or something that's kind of a longer-term, IT-extending approach? If you can comment on that, that would be great.

Thank you for your questions. Regarding business development size and opportunity, at this stage in our company’s lifecycle, we are focused on pursuing opportunities that are immediately or nearly immediately beneficial. We want to leverage the balance sheet that Mike mentioned during the call. When considering size and opportunity, our goal is to be very strategic and to seek out options that can enhance the company’s balance sheet right away. I am not ready to commit to a specific size at this moment. Our assessment will revolve around those opportunities that are either close to market or currently in the market. We aim to expand our capabilities to enable larger opportunities in the future, consistent with our buy-and-build strategy. We want to strengthen our market presence. Regarding how the FIRDAPSE litigation affects our thinking, Steve can provide details on that timing, but we expect a substantial runway with FIRDAPSE and its sales. This gives us the chance to grow our portfolio, and we intend to fully capitalize on that as much as we can for now. Concerning life cycle management, we believe it is crucial to explore opportunities for AGAMREE to develop applications for rare and orphan conditions that require a steroid. We aspire to be the preferred steroid option. In my previous experiences, companies often wait too long to consider these matters. Therefore, our focus is on completing the necessary studies, identifying opportunities, and ultimately creating value for patients and shareholders alike.

And our next question comes from the line of Samantha Semenkow with Citi.

This is Eric on for Sam. What do you think is driving the high percentage of patients switching from prednisone in your early launch? And how do you see that evolving as this progresses?

Thanks for your question. We do think that the conversion from prednisone over to AGAMREE is basically because of the safety profile. And many of these patients have been on prednisone for a significant amount of time and have seen or experienced those side effects. And they're looking to transition over to AGAMREE to benefit from the more positive safety profile. So over time, we do expect this mix to be about what it is today, which is very encouraging. Initially, during planning and forecasting, we expected the initial launch to be primarily from the EMFLAZA patients or those patients that are used to being on a branded corticosteroid. We are encouraged by this because now we're now opened up to the entire corticosteroid market for boys living with DMD. And that's what we're seeing and that's what we expect moving forward.

Our next question comes from the line of Joe Catanzaro with Piper Sandler.

Have a few on AGAMREE. If I recall correctly, I think you had said there were about 28 patients in the EAP program. Wondering if you could just sort of speak to what percent of those patients have been able to transition to commercial reimbursed drug. And then maybe if you could also sort of speak to the current cadence of intake forms, maybe how the current rate compares to your prelaunch experience, where you indicated there was a sort of indication of significant pent-up demand. So wondering if current cadence of intake forms sort of continues to point to that pent-up demand. And I have maybe one follow-up.

Sure. Jeff, do you want to take that?

Sure. I can address the situation regarding the pace of enrollments. We began accepting pre-commercial launch enrollments in December, and we experienced significant pent-up demand. We were aware that the centers of excellence were waiting for the official announcement of the commercial launch date, which delayed some enrollments. After the announcement, we started to see volume increases that confirmed our confidence in the launch. Since the commercial launch, enrollments have remained steady at a high level, surpassing our forecasts, which is very encouraging. Joe, could you remind me of your first question about AGAMREE?

Yes. Sure. It's about the EAP patients, sort of what percent have been able to transition to commercial reimbursed drug.

Correct. Of the 28, over half of those patients are on reimbursed shipments right now, and the rest are still in process.

Okay, great. For my follow-up on AGAMREE, you mentioned an expected increase in the second quarter. Could you provide some context on how much of that will come from patients already on the drug completing a full three months of treatment versus new patient starts?

Sure. So just to start off, as Jeff mentioned, we saw this nice curve of patients enrolling prior to launch, and physicians were waiting. And then with the announcement of the launch, we did see an increased enrollment, which was great. So we expect this rollover to happen. And as Jeff mentioned in his comments, it takes about 4 weeks to convert these patients from enrollment to reimbursement. So that's going to roll over into the second quarter. So we're really excited about that. And these physicians waiting to launch obviously, now we're going to see that impact in the second quarter. But Jeff, do you want to take the rest of it?

Well said and well stated, Rich. And just to add on to that, we do expect the current positive payer landscape to improve even more over time. So that 4 weeks that Rich mentioned, we expect that to reduce within 6 months. We do potentially see it to about 1 to 2 weeks from time of complete enrollment to reimbursement shipment. That's our expectation. And then the other part is balanced by a significant volume of enrollments that we continue to see. So on both ends is why we have confidence in that step-up in Q2.

We have reached the end of our question-and-answer session. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.