CATALYST PHARMACEUTICALS Business Summary

Catalyst Pharmaceuticals, Inc. (CPRX) is a commercial-stage biopharmaceutical company that generates revenue primarily through the sale of proprietary therapies for rare neuromuscular and neurological diseases. Its business model centers on the strategic in-licensing and commercialization of orphan drugs, with a portfolio anchored by three key assets: Firdapse (amifampridine) for Lambert-Eaton myasthenic syndrome (LEMS), Agamree (vamorolone) for Duchenne muscular dystrophy (DMD), and the anti-seizure medication Fycompa (perampanel). As of early 2026, the company continues to see robust growth, having raised its 2025 revenue guidance to a range of $565 million to $585 million, driven by the rapid market adoption of Agamree and the sustained dominance of Firdapse, which remains the only FDA-approved treatment for LEMS. Catalyst operates in a specialized niche alongside competitors such as Jazz Pharmaceuticals, BridgeBio Pharma, and Spark Therapeutics (Roche), maintaining a competitive edge through its high-touch patient support programs and a fortified patent estate that protects its core LEMS franchise through 2035. The leadership team is led by President and CEO Richard J. Daly, a seasoned executive who assumed the role in January 2024 after serving as President of CARsgen Therapeutics and President of AstraZeneca’s US Diabetes division. He is supported by Executive Vice President and CFO Michael W. Kalb, who joined in 2024 with over 30 years of experience, including previous CFO roles at Amarin Corporation and CinCor Pharma. In June 2025, the company further strengthened its clinical expertise by appointing Dr. William T. Andrews as Chief Medical Officer, bringing a 24-year pedigree from rare disease leaders like Akcea Therapeutics and Flexion Therapeutics. The company was co-founded by Patrick J. McEnany, who currently serves as Non-Executive Chairman of the Board, and Hubert E. Huckel. Catalyst is backed by a strong institutional investor base, including BlackRock, The Vanguard Group, State Street Global Advisors, and Deerfield Management, reflecting its status as a highly profitable, small-cap leader in the orphan drug sector.