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Visual22 Oct 2025
Epiroc's cash spent on M&A
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.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.
FIRDAPSE oncology LEMS: How to reach 50% diagnosis?
AGAMREE: How to gain 'unfair share' before label update?
M&A: Why is 'social element' a deal-breaker?
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Next Catalyst Pharmaceuticals earnings date
Q3 20256 Nov, 2025
Next Catalyst Pharmaceuticals earnings date
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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