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Catalyst Pharmaceuticals (CPRX) investor relations material

Catalyst Pharmaceuticals Baird Global Healthcare Conference 2025 summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Baird Global Healthcare Conference 2025 summary9 Sep, 2025

Business model and strategic approach

  • Focuses on acquiring de-risked assets in orphan and rare diseases, leveraging partnerships with companies lacking commercialization infrastructure.

  • Operates with a lean internal structure, supported by specialty pharmacy, patient hub, and 3PL services.

  • Prioritizes immediately or nearly immediately accretive assets, with a strong emphasis on differentiated products and cultural fit in partnerships.

  • Maintains a strong balance sheet with $652 million in cash and no debt, enabling flexibility for future acquisitions.

  • Targets opportunities with peak sales up to $500 million, focusing on markets unattractive to larger players.

FIRDAPSE: Market dynamics and growth strategy

  • FIRDAPSE addresses Lambert-Eaton Myasthenic Syndrome (LEMS), split evenly between idiopathic and cancer-associated cases, with about 3,600 U.S. patients.

  • Business has grown 15% quarter over quarter, driven by expansion into oncology and improved testing and guideline updates.

  • Recent NCCN guideline inclusion and frictionless testing are expected to drive significant growth, especially in oncology practices.

  • Oncology market penetration is currently under 10%, with a long-term goal to exceed 50% diagnosis and treatment rates.

  • Full-year 2025 guidance is $355–$360 million in sales, with a $1.2 billion market potential.

AGAMREE: Launch progress and future plans

  • AGAMREE is positioned as a safer corticosteroid for Duchenne Muscular Dystrophy (DMD), with better bone health, stature, and reduced behavioral side effects.

  • Outreach to 100+ centers of excellence has resulted in broad adoption, with a focus now on deepening engagement and data dissemination.

  • The Summit Study, enrolling over five years, aims to generate long-term safety data to support label updates.

  • Early feedback highlights rapid behavioral improvements, benefiting patients and families.

  • 2025 sales guidance is $100–$110 million, with a $1 billion market opportunity as steroid use expands from 70% to 95% of DMD patients.

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Q3 20256 Nov, 2025
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Frequently asked questions

Catalyst Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing therapies for rare neurological and neuromuscular diseases. The company's portfolio includes treatments designed to address conditions such as Lambert-Eaton Myasthenic Syndrome (LEMS) and other disorders that have limited treatment options. The company is headquartered in Coral Gables, Florida, and its shares are listed on the NASDAQ.

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