Catalyst Pharmaceuticals (CPRX) Barclays 28th Annual Global Healthcare Conference summary

Company overview and product portfolio

• Focuses on ultra-rare diseases with three main commercial products: Firdapse (Lambert-Eaton myasthenic syndrome), Agamree (Duchenne muscular dystrophy), and Fycompa (epilepsy).

• Firdapse has shown consistent 15%-20% annual growth over 8 years.

• Agamree launched in March 2024, showing strong uptake and differentiation in clinical profile.

• Fycompa, acquired from Eisai, is declining due to generic competition but remains above expectations.

Recent performance and growth drivers

• Achieved 20% revenue growth in 2025, with expectations for even faster growth in 2026.

• Specialty pharmacy initiatives improved patient retention and brand performance for Firdapse.

• New programs and guideline inclusion for cancer-associated LEMS are expanding Firdapse's reach.

• Agamree sourced patients from both generic prednisone and Emflaza, weathering market challenges well.

Firdapse: Market opportunity and strategy

• Penetration remains at 30% for idiopathic LEMS and less than 10% for cancer-associated LEMS, indicating significant growth potential.

• NCCN guideline inclusion and partnerships with GPOs are expected to drive adoption, especially in oncology.

• Commercial strategy includes targeted education and a dedicated field force for GPO engagement.

• Intellectual property secured through settlements with Teva and Lupin, with protection until February 2035.