Earnings Call Transcript - CPRX Q4 2023

Michael Kalb, CFO

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. Furthermore, 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.

Richard Daly, CEO

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 with you 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 a 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 the 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 muscular dystrophy. AGAMREE was approved in the fourth quarter and remains on track for the launch of AGAMREE before the end of the first quarter in 2024. We're excited to have this innovative product in our portfolio. More importantly, we know through our outreach to patients, caregivers, and providers that the Duchenne's community is excited as well. We are well prepared for the successful launch of AGAMREE, providing further revenue growth and diversification, while addressing a significant unmet need for improved therapeutic options for in-patients. The future potential for AGAMREE represents an opportunity for incremental growth that can continue to propel the company forward. We are committed to our buy-and-build strategy, and our results bear testament to its success. In 2023 alone, we allocated approximately $300 million toward acquiring assets without debt, bolstering the company's commercial position. Furthermore, our recent common stock offering combined with our robust balance sheet underscores our ability to persistently pursue strategic investments aimed at expanding and diversifying our portfolio. Jeff will provide an overview of our performance in 2023 with respect to commercializing FIRDAPSE and FYCOMPA and our plans for our portfolio in 2024, including AGAMREE in his section this morning. Pillar number three, lifecycle management and partnerships. We await the FDA's decision on the expansion of the FIRDAPSE label to include a 100 milligram dosing, which is expected in the second quarter of 2024. We believe this has the potential to provide an important option for patients who live with LEMS. We are also making great strides in our ex-U.S. partnerships. Japanese regulatory authorities have accepted DyDo's NDA for FIRDAPSE, and we expect a decision on their application later this year. This also bodes well for expanding our ex-U.S. opportunities, as the Japanese NDA acceptance triggers the expansion of our territorial rights for FIRDAPSE in certain APAC and LatAm markets. Finally, pillar number four. We are well known for our financial discipline, and we plan to continue to deliver the rigor you have come to expect. Mike will go into great detail in his section of the call. Now to our 2023 results. We delivered outstanding performance in 2023, exceeding our guidance for the year, delivering record total revenues, both organically and through the expansion of our product portfolio. These results continue to drive our momentum as we move through 2024. Our full year total revenue for 2023 was $398.2 million, an 85.9% increase year-over-year compared to $214.2 million in 2022. Additionally, we achieved record fourth quarter total revenues of $110.6 million, representing an 82% increase compared to $60.8 million in quarter four of 2022. We achieved our results by delivering 21% revenue growth for the full year of 2023 for FIRDAPSE, up sales of $258.4 million, and delivering $138.1 million in sales for FYCOMPA. We are proud of these results, and we thank our team for delivering such a strong performance. Looking ahead to 2024, our full year 2024 guidance reflects our confident outlook projecting sustained growth and strategic advancements in line with our long-term objectives. We believe that 2024 will provide opportunities for continued growth for our portfolio, and we are forecasting full year total net revenues of between $455 million and $475 million, representing a 14% to 19% increase in our total net revenues compared to 2023. To provide you with more color on each of these pillars, I'll turn the presentation over to the team. Let's begin with Jeff Del Carmen, our Chief Commercial Officer. Jeff?

Jeffrey Del Carmen, Chief Commercial Officer

Thanks, Rich, and good morning, everyone. First, I want to commemorate the Rare Disease Day and 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 of 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 and 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 that 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 to broaden the addressable LEMS market through increased VGCC antibody testing will further bolster growth prospects. As a reminder, we shared an abstract at the World Conference on lung cancer in September, which projected that the prevalence of LEMS in the United States is at least 3,600. Data suggests it could potentially reach 5,400 individuals. This figure marks a notable rise from the previously estimated prevalence of 3,000. Our estimate stems from a thorough analysis of real-world data, particularly emphasizing the prevalence of LEMS diagnosis among patients with small cell lung cancer. Presently, we gauge that over 80% of small cell lung cancer LEMS patients remain undiagnosed, indicating a substantial growth opportunity. Now I'd like to share some key updates regarding our progress with FYCOMPA. The commercialization of FYCOMPA has surpassed our initial expectations. In May 2023, we seamlessly integrated FYCOMPA into our product offerings as a strategic and highly accretive product. We actively engaged with healthcare providers named key opinion leaders, expanded our outreach to establish vital partnerships with patient advocacy groups to increase awareness, furthering our mission, and fostered valuable relationships that have played a significant role in FYCOMPA's success. FYCOMPA's net revenues have exceeded the anticipated full year estimate of $130 million, reaching a total product revenue of $138.1 million, surpassing expectations by 6%. This achievement was propelled by a robust end to the year, with revenues for Q4 2023 reaching $39.3 million. We forecast 2024 FYCOMPA net revenues will be approximately $130 million to $135 million based on changes in gross to net. When products transition to companies with a smaller portfolio like we experienced with FYCOMPA, you typically encounter higher fees from service providers such as wholesalers. Let's turn to AGAMREE, a promising novel anti-inflammatory corticosteroid aimed at addressing a significant gap in treatment for the estimated 11,000 to 13,000 boys living with Duchenne Muscular Dystrophy, or DMD. As previously disclosed, we are on track for a commercial launch in the first quarter of 2024. AGAMREE will be integrated into our neuromuscular franchise, capitalizing on the team's proven capabilities, commercial expertise, and experience. We have finalized the recruitment of 10 commercial personnel to bolster the impending launch, primarily focusing on marketing and patient services. Our existing neuromuscular salesforce, comprising 16 regional account managers and two area business directors, will assume responsibility for both FIRDAPSE and AGAMREE. Furthermore, AGAMREE will benefit from the support of our top-notch Catalyst pathways program, ensuring that all eligible patients have access to the product. Based on the feedback from our constituents and analysis of the DMD market, the wholesale acquisition cost of AGAMREE will be at a slight discount to EMFLAZA. We will implement the same financial programs as we have for FIRDAPSE, with a goal to ensure the average out-of-pocket cost per patient will be less than $2 per month. More details on the commercial launch will be available in the coming weeks. We began accepting pre-commercial launch new patient enrollments on December 1st, 2023. Based on the volume of new patient enrollments and observed pent-up demand, our 2024 net revenue guidance for AGAMREE is between $25 million and $30 million. In conclusion, we are extremely proud of our achievements in 2023 and maintain strong confidence in meeting our targets for 2024. As we prepare for the launch of AGAMREE, we will leverage our established commercial proficiency. I express sincere appreciation to the entire Catalyst team for their unwavering dedication to patients, and I eagerly look forward to a prosperous 2024 ahead. I will now turn the call over to Dr. Steven Miller, our Chief Operating Officer and Chief Scientific Officer for an update on R&D activities.

Steven Miller, Chief Operating Officer and Chief Scientific Officer

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's 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 DMD. AGAMREE was approved on October 26th of last year and was granted new chemical entity exclusivity and orphan drug exclusivity, conferring market exclusivity to October 2027 and 2030, respectively. This FDA granted exclusivity is augmented by six orange booklist patents that provide protection to 2040 for AGAMREE. As part of our ongoing effort to maximize value, we will continue to seek opportunities to strengthen AGAMREE's patent protection and explore new indications for labeling. AGAMREE, over vamorolone in clinical studies, has shown efficacy for treatment of DMD patients, while holding the potential for a reduction in the burden of steroid-associated side effects compared to steroids. This includes the potential for notable benefits for bone health, growth, and behavioral outcomes based on preliminary data. Out of the estimated 11,000 to 13,000 DMD patients in the U.S., roughly 90% have undergone corticosteroid treatment at some stage. Presently, it is estimated that about 70% of DMD patients currently under treatment receive steroids. Steroids serve as the cornerstone of DMD therapy, highlighting the pressing demand for a more tolerable steroid option. The short-term safety and efficacy of vamorolone were demonstrated in a 24-week randomized double-blind placebo and active-controlled study, and it is well-tolerated and was associated with maintenance of muscle strength and function. To determine what specific safety benefits AGAMREE may have, if any, relative to other approved corticosteroids, Catalyst is investing in a registry to study the long-term safety and quality of life in males with DMD who are treated with AGAMREE. As this data is collected and analyzed, Catalyst will periodically seek to update the AGAMREE prescribing information with the latest information regarding the safety of the drug based on FDA guidance documents for real-world data. Education and information on new and unique products is the focus of Catalyst medical science liaisons for our products. Critical support to the medical community by providing information that is in the public domain to the medical community to educate them on these rare disease states, and on the academic research that may have been published about these medications. This assists healthcare providers in selecting the best treatment for their patients. Vamorolone has generated a high level of interest within the healthcare community. The neuromuscular MSL team is engaging with the DMD medical community since AGAMREE's approval in October of last year. There are numerous publications on vamorolone's pharmacology and effect in the DMD patient population. I'll now turn the call over to Mike Kalb, our Chief Financial Officer.

Michael Kalb, CFO

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 opportunities 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 the 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 marked-to-market value on a quarterly basis using the observable market price. Now on to 2023 results. Our total net revenue 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 2022. Product revenue net for FYCOMPA was $138.1 million for 2023. 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 for FYCOMPA was booked under a size arrangements with distributors and government agencies. However, starting in 2024, all such costs are tied to arrangements between us and those distributors and government agencies. Since our costs under these arrangements are likely to be higher than a size cost, we expect to be impacted by an increase in gross to net deductions for FYCOMPA, thereby causing a corresponding decrease in FYCOMPA net product revenue. As Jeff mentioned, this is typical for smaller companies with smaller product portfolios. Net income before income taxes for 2023 was $94.5 million, a 9.7% decrease year-over-year compared to $104.7 million for 2022. Net income for 2023 was adversely impacted by the $81.5 million charge for in-process R&D resulting from the AGAMREE license acquisition. We reported U.S. GAAP net income for 2023 of $71.4 million or $0.67 per basic share and $0.63 per diluted share, a decrease of 14% year-over-year compared to GAAP net income for 2022 of $83.1 million or $0.80 per basic and $0.75 per diluted share. The decrease in net income is attributed to the one-time AGAMREE related IP R&D expense recorded during the third quarter of 2023. Non-GAAP net income for 2023 was $223.2 million or $2.10 per basic and $1.96 per diluted share, which excludes the IP R&D expense, the income tax provision of $23.1 million, amortization of intangible assets related to our acquisitions of Ruzurgi, FYCOMPA, and AGAMREE of $32.6 million, stock-based compensation expense of $14.3 million, and depreciation expense of $316,000 from GAAP net income. This compares to non-GAAP net income for 2022 of $113.9 million or $1.10 per basic and $1.02 per diluted share, which excludes from GAAP net income the income tax provision of $21.6 million, stock-based compensation of $7.9 million, amortization expense for Ruzurgi and tangible assets of $1.1 million, and depreciation expense of $141,000. The above represents an approximate 96% increase of non-GAAP net income year-over-year. Without consideration for potential M&A activity, we expect amortization expense for our acquired licenses and intangible assets to be approximately $9.3 million beginning with the first quarter of 2024 and continuing until the depletion of the intangibles related to FYCOMPA in 2027. Our effective tax rate for 2023 was 24.4%. We anticipate our effective tax rate to increase slightly for 2024. Our tax rate is affected by many factors and therefore may fluctuate quarterly. Cost of sales were approximately $52 million in 2023 compared to $34.4 million in 2022 and consisted principally of royalties. As a reminder, royalties for products paid to our licenser for that product increased by 3% once net product sales exceed $100 million in any calendar year. Research and development expenses were $93.2 million in 2023. This compares to $19.8 million in 2022. As previously mentioned, the driver behind the increase in research and development expenses relates to the one-time AGAMREE IP R&D expense during the third quarter of 2023. Relative to the normal course of business, absent another acquisition, we expect R&D costs in 2024 to trend similarly to R&D costs in 2022. SG&A expenses for 2023 totaled $133.7 million compared to $57.1 million in 2022. The increase in SG&A year-over-year is principally due to expenses related to the integration of FYCOMPA, including commercial expenses incurred under the transaction service agreement and an increase in the headcount principally in sales and marketing, as well as certain increases in corporate headcount required to support the company's growth. During the fourth quarter of 2023, the company incurred significant selling and marketing expenses for the preparation of the AGAMREE launch. With regard to the first quarter of 2024, we anticipate a significant increase in 2024 SG&A expenses compared to Q1 of 2023 due to the upcoming AGAMREE launch and the accompanying support of three commercial products compared to two in Q1 2023. Further, I would like to note that consistent with prior years, as a result of U.S. GAAP requirements, the company expects an increase in SG&A expenses in the first quarter of 2024 compared to other quarters due to its 501(c)(3) donations. As reported, we ended 2023 with cash and cash equivalents of $137.6 million compared to $298.4 million at December 31st, 2022. The decrease in cash of $160.8 million was largely driven by approximately $293.3 million in payments in connection with acquisitions including equity securities, which was offset by net positive cash flows generated from operations of $141.4 million. On a related note, I would like to mention that on January 9th, 2024, the company issued 10 million shares of common stock for total net cash proceeds of approximately $140.1 million, which is intended to fund the potential acquisition of new product candidates, as well as for general corporate purposes. 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 fourth quarter and full year 2023 financial performance may be found in our Annual Report on Form 10-K, which was filed with the Securities and Exchange Commission yesterday, February 28th, and can be found on the investor relations page of our website www.catalystpharma.com. And with that, I'll turn the call back over to Rich.

Richard Daly, CEO

Thanks, Mike. I want to express my gratitude to the leadership team and all our employees at Catalyst for their hard work in achieving remarkable results in 2023. We have started 2024 strongly and are maintaining the momentum needed for sustained long-term growth. Our commitment to excellent execution remains steadfast. We are concentrating on expanding the scope and scale of our business by carefully identifying innovative therapies that address significant unmet medical needs in the rare orphan, neuro, and related therapeutic areas. We also aim to enhance our geographic presence through partnerships outside the U.S. In essence, we are ready to invest in opportunities that will add value to Catalyst, further develop our portfolio, and enhance patient care. Our team is focused on improving the lives of more patients by engaging in key lifecycle management projects that could broaden the application of our products. We are confident that our financial discipline, robust results, and cash flow will enable us to pursue our crucial initiatives. To summarize, our strategic priorities for 2024 are as follows: first, to continue achieving double-digit growth for our portfolio; second, to successfully launch and commercialize AGAMREE; third, to expand our product portfolio and geographic partnerships in the CNS orphan and related therapeutic areas. We look forward to keeping you updated on our progress. Now, I will turn the call back over to our operator, Kevin.

Operator, Operator

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

Charles Duncan, Analyst

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?

Steven Miller, Chief Operating Officer and Chief Scientific Officer

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.

Charles Duncan, Analyst

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's value proposition versus other standard-of-care? Thanks.

Steven Miller, Chief Operating Officer and Chief Scientific Officer

The size of the registry will depend on how many patients choose to participate, as participation is voluntary. We expect a significant number of patients to enroll and be monitored for as long as they stay in the registry. The duration of the registry will extend over many years as we gather data for the long-term safety evaluation of AGAMREE.

Richard Daly, CEO

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

Gary Ingenito, Chief Medical and Regulatory Officer

So we will be looking at all of the sites that potentially enroll and commercialize 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.

Richard Daly, CEO

But you'd say it's a pretty broad-based approach, would that be safe to say?

Gary Ingenito, Chief Medical and Regulatory Officer

Yes.

Charles Duncan, Analyst

Got it. Thanks. Thanks Rich for the advice.

Joon Lee, Analyst

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 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?

Richard Daly, CEO

Thank you for your question, Joon. The market for DMD is quite specific, with around 250 prescribers responsible for 95% of corticosteroid prescriptions for boys with DMD. These prescribers are located in approximately 90 to 100 centers of excellence, which makes it a targeted area for our team, who are well-equipped to manage and effectively reach these prescribers. They are experienced professionals, so we feel positive about the team structure. Regarding the pent-up demand you've mentioned, we have been accepting pre-commercial launch enrollments since December. The number of enrollments we've seen and the interest from various stakeholders in AGAMREE make us confident in the projections we've shared. Additionally, our approach includes non-personal promotion along with our sales force efforts to educate both physicians and other key groups such as patients and caregivers about AGAMREE and DMD.

Joon Lee, Analyst

Great. Thanks for the color. And on the continued strength of 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.

Richard Daly, CEO

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.

Joseph Catanzaro, Analyst

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.

Steven Miller, Chief Operating Officer and Chief Scientific Officer

I'll let Jeff address the guidance, but regarding your question about patients on doses below 80 milligrams per day, yes, there are patients receiving those doses. The neuromuscular physician community tends to be conservative, often adopting a cautious approach to medication treatment. Many prefer to keep some flexibility in the dosage, allowing for adjustments if a patient's condition worsens. With the increase in the daily maximum dosage, we anticipate that some of these conservative physicians, who may have had concerns about the efficacy of a 60 milligram dose, will be more inclined to raise the dosage now that there is more room for adjustment. Therefore, you are correct in noting that some patients on doses below 80 milligrams might also increase their dosages. Jeff, would you like to address the guidance?

Jeffrey Del Carmen, Chief Commercial Officer

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 into 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.

Richard Daly, CEO

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 transferable 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.

Leland Gershell, Analyst

Good morning, everyone. Thank you for taking my questions and it's great to see the strong guidance on AGAMREE as well as overall. I have a few questions. Firstly, regarding FYCOMPA, the guidance suggests it might be flatter year-on-year in 2024 compared to what the company previously indicated, which was growth in the mid to high single digits. I'm curious if there are any dynamics we should be aware of. Secondly, on FYCOMPA, is there any update regarding IP exclusivity? I believe we have protection until around spring of 2025, extended to 2026. I'm wondering if there's any update on the IP. Thank you.

Richard Daly, CEO

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

Michael Kalb, CFO

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 another entity, and as is common for companies of our size, smaller companies with smaller product portfolios, those rates are going to be slightly higher.

Steven Miller, Chief Operating Officer and Chief Scientific Officer

Hi, Leland. With regard to the intellectual property, we have completed our work with the FDA to establish that the expiration date of May 2025 is 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.

Richard Daly, CEO

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 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 after patent expiry. So, there is a significant opportunity for this compound post-expiry to continue to deliver good returns 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, again, helping 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.

Leland Gershell, Analyst

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.

Richard Daly, CEO

About 50% of our new enrollments each month or each quarter come from patients who are diagnosed but have not yet been treated with FIRDAPSE. We use various sources to continually find new patients, so as one patient starts treatment with FIRDAPSE, we seek out new leads to replace them. We aim to keep around 500 patient leads at all times. A significant challenge for these patients is often just the wait to see their physicians, which can take three to six months. While some patients in the 500 have remained as leads for over a year, most of the newer leads can be converted within 30 days. We continue to backfill converted patients with new leads over time.

Joseph Catanzaro, Analyst

I appreciate the color. Thank you.

Operator, Operator

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.

Richard Daly, CEO

Thank you.