Catalyst Pharmaceuticals (CPRX) investor relations material

Business development and licensing strategy

• Focus on acquiring rare disease assets with immediate or near-term accretion, minimal clinical and regulatory risk, and peak sales potential around $500 million, but open to upside beyond that.

• Over 100 opportunities assessed since the start of the year, with a shift to consider Phase III assets as capital markets evolve.

• Strong cash position ($689 million, no funded debt) enables competitive bidding, but avoids large-scale deals against bigger players.

• Not waiting for Firdapse IP resolution to pursue deals; prioritizing growth and patient impact.

• Will consider leverage only for on-market products with long-term potential and near-certain commercial success.

Product lifecycle management and expansion

• Lifecycle management focuses on earlier patient initiation, longer duration, and dose optimization for Agamree and Firdapse.

• Agamree's orphan status ends in 2029, with patent protection expected through 2034; Phase I studies underway to guide future indications.

• Decision on Agamree's broader rare disease applications expected in the first half of next year, pending Phase I data.

• Label expansion and new indications for Agamree are prioritized, especially in rare, long-term steroid use conditions.

• SUMMIT and GUARDIAN trials are key to demonstrating Agamree's benefits and market potential.

Market dynamics and patient sourcing

• Agamree patient sourcing remains balanced: 45% from Emflaza, 45% from prednisone, 10% naive, with high payer approval rates (85-90%).

• U.S. addressable market for steroids in DMD estimated above $1 billion, with potential to grow as dissatisfaction with current therapies persists.

• Gene therapy has not significantly reduced steroid use; steroids remain foundational therapy.

• Payer coverage approval times have improved, now under two weeks.