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 and is positioned as a differentiated steroid.

• Fycompa, acquired from Eisai, is experiencing expected revenue decline due to generic competition but remains a solid performer.

Recent performance and operational highlights

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

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

• Agamree launch weathered market challenges, sourcing patients from both generic prednisone and Emflaza, and maintained strong momentum.

• Fycompa's revenue is declining post-generic entry, but promotional efforts have sustained performance above expectations.

Firdapse: Market dynamics and growth drivers

• 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 increased diagnosis and adoption, especially in oncology.

• Commercial strategy includes a dedicated field force to educate and align with GPOs and oncologists.

• Intellectual property secured through settlements with Teva and Lupin, with protection until February 2035; one litigation with Hetero remains.