Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q4 2024 Earnings Conference Call February 27, 2025 8:30 AM ET Company Participants Michael Kalb - Executive Vice President & Chief Financial Officer Richard Daly - President and Chief Executive Officer Jeff Del Carmen - Executive Vice President, Chief Commercial Officer Steven Miller - Executive Vice President, Chief Operating Officer and Chief Scientific Officer Gary Ingenito - Chief Medical and Regulatory Officer Conference Call Participants Charles Duncan - Ca ...

Catalyst Pharmaceuticals (CPRX) Q4 2024 Earnings Call February 27, 2025 08:30 AM ET Company Participants Michael Kalb - Executive VP & CFORichard Daly - President & CEOJeff Del Carmen - Chief Commercial OfficerGary Ingenito - Chief Medical & Regulatory OfficerBenjamin Paluch - Equity Research Senior AssociateRohan Mathur - Equity Research AssociateFelix Ampomah - Senior Associate Conference Call Participants Charles Duncan - AnalystPavan Patel - AnalystAsim Rana - Equity AnalystJoseph Catanzaro - Director & ...

Core Insights - Catalyst Pharmaceutical (CPRX) reported a revenue of $141.82 million for the quarter ended December 2024, reflecting a year-over-year increase of 28.3% and surpassing the Zacks Consensus Estimate by 6.87% [1] - The earnings per share (EPS) for the quarter was $0.70, which is an increase from $0.53 in the same quarter last year, resulting in an EPS surprise of 34.62% compared to the consensus estimate of $0.52 [1] Revenue Breakdown - Product revenue from FIRDAPSE was $82.52 million, exceeding the average estimate of $81.54 million from five analysts [4] - Product revenue from FYCOMPA reached $38.22 million, surpassing the average estimate of $32.83 million [4] - Product revenue from AGAMREE was reported at $21.08 million, exceeding the estimated $17.84 million [4] - Total product revenue was $141.81 million, which is a 30% increase compared to the year-ago quarter and also above the average estimate of $132.21 million [4] - Revenue from licenses and other sources was only $0.01 million, significantly below the average estimate of $0.85 million, marking a drastic year-over-year decline of 99.3% [4] Stock Performance - Over the past month, shares of Catalyst have returned -8.9%, underperforming the Zacks S&P 500 composite, which saw a -2.3% change [3] - The stock currently holds a Zacks Rank 4 (Sell), indicating potential underperformance in the near term [3]

Core Insights - Catalyst Pharmaceuticals reported total revenues of $491.7 million for the full year 2024, reflecting a year-over-year growth of 23.5% driven by strong commercial performance [1][11][12] - The company expects total revenues for 2025 to be between $545 million and $565 million, indicating confidence in continued growth momentum [1][22] Financial Performance - Full year 2024 product revenue, net was $489.3 million, a 23.4% increase from $396.5 million in 2023 [3][12] - Q4 2024 net product revenue was $141.8 million, representing a 28.3% increase compared to Q4 2023 [1][11] - GAAP net income for 2024 was $163.9 million, a significant increase of 129.5% from $71.4 million in 2023 [3][17] - Non-GAAP net income for 2024 was $276.3 million, up 95.1% from $141.6 million in 2023 [3][18] Product Performance - FIRDAPSE product revenue for 2024 was $306.0 million, an 18.4% increase year-over-year [1][9] - AGAMREE product revenue for 2024 was $46.0 million, reflecting a successful commercial launch [1][9] - FYCOMPA product revenue for 2024 was $137.3 million, showing a slight decline of 0.6% compared to 2023 [3][9] Strategic Developments - Catalyst successfully launched AGAMREE in the U.S. on March 13, 2024, for the treatment of Duchenne muscular dystrophy [9][10] - The company received FDA approval for an increased maximum daily dose of FIRDAPSE to 100 mg on May 30, 2024 [9] - Catalyst entered into a settlement agreement with Teva Pharmaceuticals regarding FIRDAPSE patent litigation, preventing Teva from marketing a generic version until February 25, 2035 [9][10] Future Outlook - For 2025, FIRDAPSE's product revenue is expected to range between $355 million and $360 million, while AGAMREE's revenue is anticipated to be between $100 million and $110 million [22][24] - FYCOMPA's product revenue is forecasted to be between $90 million and $95 million, influenced by the anticipated loss of patent exclusivity [25] - Research and development expenses for 2025 are expected to be between $15.0 million and $20.0 million, depending on ongoing studies and potential acquisitions [27]

Financial Position - As of December 31, 2024, the company had cash and investments of approximately $517.6 million, sufficient to support operations for at least the next 12 months[64]. - The main objective of the company's investment activities is to preserve capital for acquisitions and operations while maximizing income without taking significant risks[505]. - The company does not utilize derivative financial instruments in its investment portfolio, focusing on liquidity and preservation of principal[505]. - The company's exposure to interest rate risk is primarily related to cash and cash equivalents invested in highly liquid money market funds and U.S. Treasuries[505]. - Market risk is influenced by fluctuations in interest rates, foreign exchange rates, and commodity prices, which can affect operational results and cash flows[504]. Product Development and Commercialization - The company aims to commercialize FIRDAPSE® for LEMS and AGAMREE® for DMD, with ongoing efforts to expand market awareness and access[66]. - The company estimates there are at least 3,600 LEMS patients in the U.S., with approximately half diagnosed, indicating a significant potential market[71]. - The company has a buy-and-build strategy focused on acquiring high-value, orphan designated assets to enhance its product portfolio[65]. - The company has expanded its commercial territory for FIRDAPSE® to include Japan and most of Asia, as well as Central and South America[74]. - The company is conducting a Phase 1 trial for AGAMREE® to evaluate its efficacy against other steroids, aiming to develop additional indications[66]. - The company has settled a dispute with Jacobus, acquiring rights to develop and commercialize RUZURGI® in the U.S. and Mexico, along with related patents[77]. - The company pays Jacobus an annual royalty of 1.5% on net sales of amifampridine products in the U.S. for 2022-2025, increasing to 2.5% thereafter[78]. - The FDA approved FIRDAPSE® for pediatric patients aged six years and older on September 29, 2022, expanding its market reach[80]. - The company is required to conduct post-approval studies, including a pediatric safety study related to FIRDAPSE®[81]. - FIRDAPSE® was launched in the U.S. in January 2019, supported by a field force of approximately 40 personnel and nine medical science liaisons[83]. - The company plans to split its commercial field-based force into two units by early Q2 2025, focusing separately on FIRDAPSE® and AGAMREE®[84]. - AGAMREE® was approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged 2 and older on October 26, 2023[123]. - The company made a $75 million payment to Santhera for the exclusive North American license of AGAMREE®, with additional milestone payments of $36 million due following FDA approval[128]. - Clinical trials for AGAMREE® showed significant improvements in functional endpoints compared to placebo, validating previous findings[124]. - AGAMREE® is protected by multiple patents with expiration dates ranging from August 2028 to July 2040[131]. - The company is preparing to add additional third-party manufacturers for the supply of AGAMREE® after January 1, 2026[140]. - The company is executing strategies in anticipation of the patent expiry of FYCOMPA® to address potential generic competition[121]. - The company is actively pursuing acquisitions to diversify its product portfolio in rare disease therapeutic categories, although no definitive agreements have been made recently[152]. Regulatory Environment - The FDA's approval process for drugs requires substantial time and financial resources, with a significant application fee of $4,310,002 for fiscal year 2025[166]. - The company must comply with FDA regulations and cGMP standards to avoid delays in product launches and shipments[142][154]. - The FDA may impose clinical holds on trials, which can delay the development process and affect market entry[158]. - The FDA may issue a complete response letter if the NDA submission or manufacturing facilities evaluation is unfavorable, outlining deficiencies and requiring additional testing or information[167]. - Post-approval requirements include strict regulations on marketing and promotion, and the FDA may require a risk evaluation and mitigation strategy (REMS) to ensure drug benefits outweigh risks[169]. - Drugs can only be marketed for approved indications, and changes to conditions require submission and FDA approval of a new NDA or sNDA[170]. - Adverse event reporting and periodic reports are mandatory after NDA approval, and manufacturers must comply with cGMPs to maintain product approval[171]. - Controlled substances are classified into five schedules, with FYCOMPA® classified as Schedule III, indicating a lower potential for abuse compared to Schedules I and II[172]. - Upon NDA approval of a new chemical entity, the drug receives five years of marketing exclusivity, preventing ANDA submissions for generic versions during this period[178]. - ANDA applicants may be eligible for a 180-day exclusivity period if they are the first to submit an ANDA with a Paragraph IV certification[189]. - For fiscal year 2025, application fees for ANDA are $321,920, with facility fees varying based on domestic or foreign status[191]. - The regulatory approval process in foreign countries requires adequate evidence of safety, quality, and efficacy, similar to FDA requirements[192]. - The European Union allows drug approval applications through centralized or decentralized procedures, impacting marketing authorization across member states[193]. Market Access and Pricing - The company's ability to commercialize products relies significantly on adequate financial coverage and reimbursement from third-party payors, including Medicare and Medicaid[194]. - There is increasing scrutiny on pharmaceutical pricing, particularly for orphan drugs, which may impact the company's pricing strategies[198]. - The company has engaged a dedicated team of market access account managers and reimbursement experts to secure coverage for rare disease therapies[199]. - The Orphan Drug Act provides a seven-year exclusivity period for drugs approved for rare diseases, but this exclusivity is subject to scrutiny and potential modification[202]. - The out-of-pocket limit for Medicare Part D enrollees is set to be $2,000 in 2025, adjusted for inflation thereafter[223]. - The branded pharmaceutical fee for manufacturers has remained at $2.8 billion annually since 2019[231]. - The Medicaid drug rebate for innovator products is generally the greater of 23.1% of the average manufacturer price (AMP) or the difference between AMP and the best price[219]. - The company is required to report pricing information to CMS quarterly for drugs reimbursable under Medicare Part B[222]. Competition - The company is facing intense competition in the pharmaceutical industry, with many organizations having greater financial and technical resources[144]. - The approval of FIRDAPSE® has shifted LEMS treatment from unapproved drugs to a commercially marketed product, although competition from compounding pharmacies remains[145][146]. - FYCOMPA® is the first and only AED targeting the AMPA receptor, but the company anticipates competition from generic manufacturers after patent expiration[147][148]. - The DMD treatment market is highly competitive, with corticosteroids remaining the first-line treatment despite new gene therapies entering the market[149][150]. Employee and Organizational Matters - The company had 181 employees as of February 24, 2025, with 127 in the commercial organization and 11 in R&D[234]. - The company is committed to creating a diverse workforce and maintaining a harassment-free work environment[236]. - The company provides competitive compensation packages, including base salaries, performance bonuses, and stock options[235]. - The company emphasizes employee health, safety, and wellness through various innovative programs[239]. - The company utilizes several full-time consultants primarily working with the commercial organization[234].

Core Viewpoint - Catalyst Pharmaceuticals, Inc. is set to release its fourth quarter and full-year 2024 financial results on February 26, 2025, followed by a conference call on February 27, 2025, to discuss these results and provide a business update [1][2]. Company Overview - Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing novel medicines for patients with rare and difficult-to-treat diseases [1][4]. - The company has a proven track record of bringing life-changing treatments to market and emphasizes patient care and accessibility through comprehensive support services [4]. - Catalyst is headquartered in Coral Gables, FL, and has been recognized on the Forbes 2025 list as one of America's most successful mid-cap companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's fastest-growing companies [4]. Conference Call & Webcast Details - The conference call and webcast will take place on February 27, 2025, at 8:30 AM ET, with dial-in numbers provided for US/Canada and international participants [2][3]. - The webcast will be accessible on the company's website, and a replay will be available for at least 30 days following the event [3].

Core Viewpoint - The content emphasizes the importance of conducting personal due diligence and consulting competent advisors before making any financial decisions, particularly in the context of investments and trading [1]. Group 1 - The website provides general news, personal analysis, and third-party content intended for educational and research purposes [1]. - It explicitly states that the information does not constitute any recommendation or advice for investment actions [1]. - Users are advised to perform their own research and consider their financial situation before making decisions [1]. Group 2 - The website includes information about complex financial instruments such as cryptocurrencies and contracts for difference (CFDs), which carry a high risk of losing money [1]. - It encourages users to understand how these instruments work and the associated risks before investing [1].

Core Insights - Catalyst Pharmaceuticals, Inc. has been ranked fifth in Forbes' list of America's Most Successful Mid-Cap Companies for 2025, showcasing its strong performance among 100 companies [1][2][3] - The ranking reflects Catalyst's continued growth, moving from fourth place in the previous year's list of Small-Cap Companies, indicating a positive trajectory in earnings, sales, return on equity, and total stock return over the last five years [2][3] Company Overview - Catalyst Pharmaceuticals is a biopharmaceutical company focused on improving the lives of patients with rare diseases, emphasizing the development and commercialization of innovative therapies [4] - The company has a strong commitment to patient care and accessibility, providing a comprehensive suite of support services to ensure patients receive necessary treatments [4] - Catalyst is headquartered in Coral Gables, Florida, and is actively seeking to expand its global presence through strategic partnerships, while also being recognized as one of North America's Fastest-Growing Companies on the 2024 Deloitte Technology Fast 500™ List [4]

Core Insights - Catalyst Pharmaceuticals has announced the launch of FIRDAPSE® (amifampridine) Tablets 10 mg in Japan by its sub-licensee DyDo Pharma, aimed at improving muscle weakness in patients with Lambert-Eaton myasthenic syndrome (LEMS) [1][2] Company Overview - Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapies for rare diseases, with a commitment to patient care and accessibility [6] - The company has a strong presence in the U.S. and is actively seeking to expand its global footprint through strategic partnerships [6] Product Information - FIRDAPSE is the only FDA-approved therapy for LEMS in adults and pediatric patients aged six years and older, characterized as a rare autoimmune disorder [2][3] - The mechanism of action for FIRDAPSE involves blocking potassium channels, enhancing neuromuscular transmission, and improving muscle function [3] Market Expansion - The launch of FIRDAPSE in Japan represents a significant step in expanding the geographic reach of Catalyst's product portfolio, enhancing access to life-changing therapies for patients in Japan [2][6] - DyDo Pharma, a wholly-owned subsidiary of DyDo Group Holdings, is responsible for the commercialization of FIRDAPSE in Japan [5]

Core Viewpoint - Catalyst Pharmaceuticals has reached a settlement with Teva Pharmaceuticals regarding the patent litigation for Firdapse, which has positively impacted its stock price, increasing by 16.7% [1][5]. Group 1: Settlement Agreement - The settlement resolves the patent litigation initiated by Catalyst and SERB against Teva for filing an abbreviated new drug application (ANDA) for a generic version of Firdapse before the expiration of applicable patents [2]. - Teva has agreed not to market its generic version of Firdapse in the U.S. before February 25, 2035, unless specific exceptions arise [5]. - Ongoing litigation against other defendants, such as Hetero and Lupin, continues regarding Firdapse patents [6]. Group 2: Product Performance - Firdapse is currently approved in the U.S., EU, and Japan for treating Lambert-Eaton Myasthenic Syndrome (LEMS) in adults, with a label expansion for pediatric patients approved in 2022 [3]. - Firdapse accounted for 65% of Catalyst's net product sales in 2023, generating $223.5 million in revenues in the first nine months of 2024 [8]. - Catalyst also markets two other drugs, Fycompa and Agamree, which provide additional revenue streams [9]. Group 3: Recent Developments - Catalyst acquired U.S. rights for Fycompa from Eisai and North American rights for vamorolone from Santhera Pharma in 2023 [10]. - Catalyst's stock has outperformed the industry, gaining 14.5% over the past three months compared to a 7.7% decline in the industry [4].