Catalyst Pharmaceuticals
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Catalyst Pharmaceuticals (CPRX) investor relations material

Catalyst Pharmaceuticals 44th Annual J.P. Morgan Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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44th Annual J.P. Morgan Healthcare Conference summary12 Jan, 2026

Company overview and strategy

  • Focuses on rare diseases with a commercial-stage model, leveraging in-licensing, acquisition, and development of therapies for underserved conditions.

  • Operates a patient-first approach with bespoke support programs, high compliance rates, and a specialized pharmacy hub.

  • Maintains profitability, over $700 million in cash, no funded debt, and capacity for $1 billion in acquisitions.

  • Pursues both organic and inorganic growth, seeking immediately or nearly immediately accretive opportunities.

  • Expanding mandate to include products in development, targeting peak sales up to $500 million and global rights.

Product portfolio and market performance

  • FIRDAPSE is the only evidence-based treatment for Lambert-Eaton myasthenic syndrome (LEMS), with a $1 billion addressable market and 15%-20% quarterly growth.

  • AGAMREE, a differentiated steroid for Duchenne muscular dystrophy (DMD), launched in March 2024, showing 224% growth through September 2025.

  • FYCOMPA, for epilepsy, continues to outperform competitors despite generic entry, supporting cash flow.

  • FIRDAPSE market split is nearly 50/50 between cancer-associated and idiopathic LEMS, with the former slightly larger.

  • AGAMREE is gaining rapid adoption, with 90% of top centers using it and high conversion from existing steroids.

Patient support and access initiatives

  • Offers free bridge medication, insurance navigation, and broad co-pay programs to ensure therapy access.

  • Patient advocacy liaisons and ambassador programs support education and peer engagement.

  • High-touch specialty pharmacy and direct-to-patient outreach drive 90% retention and compliance.

  • Focused on reducing diagnostic delays, especially for LEMS, and leveraging machine learning for patient identification.

  • Collaborates with independent advocacy groups to expand patient support.

Impact of expanded BD on future pipeline?
FIRDAPSE IP confidence for Hetero trial?
AGAMREE's path to $1B market amid competition?
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Next Catalyst Pharmaceuticals earnings date

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Q4 202526 Feb, 2026
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Frequently asked questions

Catalyst Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing therapies for rare neurological and neuromuscular diseases. The company's portfolio includes treatments designed to address conditions such as Lambert-Eaton Myasthenic Syndrome (LEMS) and other disorders that have limited treatment options. The company is headquartered in Coral Gables, Florida, and its shares are listed on the NASDAQ.

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