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

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.