Catalyst Pharmaceuticals (CPRX) investor relations material

Business model and patient support

• Focuses on acquiring and commercializing rare disease products in the US, emphasizing risk mitigation and operational excellence.

• Offers intensive patient support programs, including patient access liaisons and ambassadors, to ensure therapy access and adherence.

• Maintains therapy for patients regardless of ability to pay, demonstrated during the Change Healthcare cyberattack, ensuring uninterrupted drug supply.

• Provides healthcare provider education and free diagnostic testing to improve rare disease diagnosis and treatment.

• Business development is largely inbound due to strong reputation and financial flexibility.

Product portfolio and commercial performance

• Launched Firdapse in 2019, followed by Agamree and Fycompa, achieving a 37% compound annual growth rate.

• Firdapse is the only evidence-based US treatment for Lambert-Eaton myasthenic syndrome (LEMS), with a $1 billion market opportunity.

• Agamree targets Duchenne muscular dystrophy (DMD) with superior safety data; rapid uptake in top centers.

• Fycompa, for epilepsy, outperformed expectations despite generic competition.

• Net product revenue grew 17.4% year-over-year, with updated guidance of $565–585 million in top-line revenue.

Market strategy and growth drivers

• Uses AI and machine learning to identify and prioritize rare disease patients, improving diagnosis and market penetration.

• Enhanced pharmacy programs optimize dosing and accelerate LEMS diagnosis, with 30% penetration in idiopathic LEMS and 10% in cancer-associated LEMS.

• Collaborates with group purchasing organizations and national labs to streamline testing and embed updated NCCN guidelines.

• Agamree benefits from robust European and Canadian labels; ongoing Summit trial aims to strengthen US label and demonstrate long-term benefits.

• Pursues immediately accretive business development opportunities and maintains disease area agnosticism.