• Alexion Pharmaceuticals for rare neurological and neuromuscular disorders.
• BioMarin Pharmaceutical, but focused on rare neurological conditions.

• FIRDAPSE (amifampridine): A medication approved for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults.
• AGAMREE (vilobelimab): A treatment for IgA nephropathy (IgAN).

Catalyst Pharmaceuticals (CPRX)

Catalyst Pharmaceuticals primarily sells its pharmaceutical products, such as FIRDAPSE, to a limited number of major pharmaceutical wholesalers. These wholesalers then distribute the products to pharmacies, hospitals, and other healthcare providers, who in turn dispense them to individual patients.

Based on their SEC filings, the major customer companies for Catalyst Pharmaceuticals are:

• McKesson Corporation (Symbol: MCK)
• Cencora, Inc. (Symbol: COR) - formerly AmerisourceBergen Corporation
• Cardinal Health, Inc. (Symbol: CAH)

These three customers collectively account for substantially all of Catalyst Pharmaceuticals' gross product sales.

• Curia
• Thermo Fisher Scientific Inc. (TMO)
• Eisai Co., Ltd. (4523.T)
• Nippon Shinyaku Co., Ltd. (4516.T)

Richard J. Daly, President and Chief Executive Officer

Richard J. Daly was appointed President and Chief Executive Officer of Catalyst Pharmaceuticals on January 1, 2024, and has served as a member of its Board of Directors since February 2015. With over three decades of pharmaceutical experience, Mr. Daly previously held the role of President at CARsgen Therapeutics Corporation from January 2022 to August 2023 and Chief Operating Officer at BeyondSpring Pharmaceuticals from July 2018 through January 2022. He also served as CEO, President, and Chairman of Neuralstem, Inc. from 2016 to 2018. Earlier in his career, he was President of AstraZeneca's U.S. diabetes subsidiary, where he was responsible for all commercial and medical plans and objectives for a $1.2 billion division and led the successful launch of an orphan rare disorder drug, Myalept. He also held senior leadership positions at Takeda and TAP Pharmaceuticals, a joint venture between Takeda and Abbott Laboratories.

Michael W. Kalb, Executive Vice President, Treasurer and Chief Financial Officer

Michael W. Kalb, CPA, joined Catalyst Pharmaceuticals in January 2024, bringing over 30 years of experience in the pharmaceutical and financial service industries. From May 2023 through December 2023, he served as Chief Financial Officer of Impel Pharmaceuticals, Inc. Prior to that, Mr. Kalb was the Executive Vice President & Chief Financial Officer of CinCor Pharma, Inc. from November 2022 through March 2023, where he played an integral role in the company's acquisition by AstraZeneca for up to $1.8 billion.

Patrick J. McEnany, Non-Executive Chairman of the Board

Patrick J. McEnany co-founded Catalyst Pharmaceuticals in January 2002 and has been a director since its formation. He served as Chairman, President, and Chief Executive Officer from 2006 until 2023, when he stepped down as President and CEO but remained Chairman. From 1991 to April 1997, Mr. McEnany was Chairman and Chief Executive Officer of Royce Laboratories, Inc., a generic pharmaceutical manufacturer. He built Royce Laboratories' sales to over $100 million annually and arranged its sale to Watson Pharmaceuticals, Inc., where he then served as President of the Royce Laboratories subsidiary and Vice President of Corporate Development from 1997 to 1998.

Steven R. Miller, PhD, Executive Vice President, Chief Operating Officer and Chief Scientific Officer

Dr. Steven R. Miller is responsible for Catalyst's product development program, encompassing chemistry and manufacturing controls, quality systems, contract operations, and development planning. He has 25 years of experience in the pharmaceutical and healthcare industries and joined Catalyst in 2007. Dr. Miller's previous roles include Executive Director for Research and Development Operations at Watson Pharmaceuticals and Vice President of Research and Product Development at Royce Laboratories. He also managed medical device development groups at Baxter Diagnostics and worked as an analytical chemist for the U.S. Food and Drug Administration.

Dr. Gary Ingenito, M.D., Ph.D., Chief Medical and Regulatory Officer

Dr. Gary Ingenito joined Catalyst Pharmaceuticals as Chief Medical Officer in June 2015 and currently serves as Chief Medical and Regulatory Officer. He oversees all clinical development programs, medical affairs, regulatory, and other related functions. With over 25 years of experience in pharmaceutical development, Dr. Ingenito has held executive responsibilities for clinical research, regulatory, drug safety, and medical affairs at various pharmaceutical companies and contract research organizations. His prior positions include Head of Medical Affairs at Sandoz Pharmaceuticals and eight years at Otsuka Pharmaceuticals, where he oversaw the approval of several anti-infective, cardiovascular, and central nervous system products. Most recently before joining Catalyst, he was Head of Regulatory Affairs North America for biosimilars at Boehringer-Ingelheim Pharmaceuticals.

The most significant clear emerging threat to Catalyst Pharmaceuticals (CPRX) is the potential market entry of rimegepant (developed by Pfizer) for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults. In March 2024, Pfizer announced positive top-line results from a Phase 3 clinical study evaluating rimegepant in adult patients with LEMS, with plans to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2024. Firdapse (amifampridine) is currently CPRX's primary revenue-generating product for adult LEMS. The potential approval and commercialization of rimegepant, backed by Pfizer's resources, could introduce significant direct competition and market share erosion for CPRX in its core therapeutic area.

Catalyst Pharmaceuticals (CPRX) has two main products with identified addressable markets:

• FIRDAPSE (amifampridine phosphate): The total addressable market for FIRDAPSE is estimated to be approximately $1.2 billion in the U.S.. This market encompasses both idiopathic and cancer-associated Lambert-Eaton myasthenic syndrome (LEMS) patient segments.
• AGAMREE (vamorolone): AGAMREE is approved for the treatment of Duchenne Muscular Dystrophy (DMD). The market for DMD is estimated to be $2 billion per year in the U.S. and is projected to reach $27 billion per year by 2030. AGAMREE alone could potentially achieve peak U.S. sales of $1 billion per year.

Catalyst Pharmaceuticals (CPRX) is expected to drive future revenue growth over the next 2-3 years through several key strategies:

1. Continued Growth of Firdapse (amifampridine): Firdapse, the company's leading product for Lambert-Eaton myasthenic syndrome (LEMS), is a primary revenue driver with sustained organic sales growth, increasing prescription rates, and ongoing diagnosis of new LEMS patients. A significant patent settlement in August 2025 blocks generic competition in the United States until February 2035, securing its market exclusivity for nearly another decade. Catalyst also aims to expand Firdapse's market penetration by focusing on education for healthcare professionals and accelerating diagnosis, particularly for the potentially 90% undiagnosed cancer-associated LEMS (c-LEMS) patients. The company reaffirmed its full-year 2025 Firdapse revenue guidance, projecting 15-20% annual growth moving forward.
2. Accelerated Uptake and Expansion of Agamree (vamorolone): Recently launched in the U.S. in March 2024 for Duchenne Muscular Dystrophy (DMD), Agamree has demonstrated strong market momentum, outperforming expectations with significant year-over-year revenue increases. This growth is attributed to high patient retention rates and growing adoption across DMD centers of excellence. Catalyst has also raised its full-year 2025 revenue guidance for Agamree, reflecting continued demand and early penetration. Furthermore, in October 2025, Health Canada accepted a New Drug Submission to commercialize Agamree in Canada, indicating potential international market expansion.
3. Sustained Demand for Fycompa (perampanel): Despite facing generic competition, Fycompa, an epilepsy drug, has shown stronger-than-originally-forecasted sales. Catalyst has raised its full-year 2025 revenue guidance for Fycompa, driven by continued demand and patient retention.
4. Strategic Business Development and Portfolio Expansion: Catalyst Pharmaceuticals actively evaluates strategically aligned opportunities to further strengthen its portfolio and expand its reach to serve more patients with rare diseases. This includes potential acquisitions or in-licensing of new products or therapies, as highlighted by their robust cash position with no debt, which supports strategic investments for long-term growth. The company's business model involves acquiring and developing FDA-approved products.
5. Geographic Expansion: Catalyst is pursuing initiatives to expand the geographic footprint of its rare disease portfolio in key territories outside the U.S. An example of this is the progress made with Firdapse in Canada, including favorable court rulings concerning the protection of Firdapse data and the potential for commercialization in the Canadian market.

Share Repurchases

• Catalyst Pharmaceuticals authorized a new $200 million share repurchase program, effective from October 1, 2025, through December 31, 2026.
• As of November 5, 2025, the company had repurchased 405,092 shares for approximately $8.4 million under this program.

Share Issuance

• In January 2024, Catalyst Pharmaceuticals completed an underwritten public offering of 10,000,000 shares of common stock at $15.00 per share, generating total gross proceeds of $150,000,000.
• As of November 3, 2025, the number of outstanding common shares was 122,912,387.
• During the three and nine months ended September 30, 2025, the company granted options to purchase 82,500 and 773,903 shares of common stock, respectively, to employees.

Capital Expenditures

• Capital expenditures for Catalyst Pharmaceuticals peaked in December 2023 at $198.5 million.
• The company's capital expenditures averaged $42.028 million from fiscal years ending December 2020 to 2024.
• Capital expenditures significantly increased in 2021 ($1.021 million), 2022 ($10.029 million), and 2023 ($198.5 million), after a low of $11 thousand in 2020.