Catalyst Pharmaceuticals, Inc. · NASDAQ:CPRX
Catalyst Pharmaceuticals — J.P. Morgan Healthcare Conference 2025
20 slides from Catalyst Pharmaceuticals's conference presentation, Jan 13, 2025
Slide 1: Improving the Lives of Patients with Rare Diseases
Exhibit 99.1 Improving the Lives of Patients with Rare Diseases NASDAQ: CPRX J.P. Morgan Healthcare Conference January 2025
Slide 2: Safe Harbor
Safe Harbor This presentation contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are outside our control. All statements regarding our strategy, future operations, financial position, estimated revenues or losses, projected costs, prospects, pl
Slide 3: Patients are at the center of every decision we make
Patients are at the center of every decision we make Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases 3
Slide 4: Growth-Oriented, Rare Disease-Focused
Growth-Oriented, Rare Disease-Focused What we are doing Commercial-Focused Business Model Maximizing the value of our innovative and differentiated commercial portfolio to serve patients with rare diseases How we will continue Ongoing Lifecycle Management Strategic Portfolio Expansion to grow Drivin
Slide 5: Building a Leading Orphan Drug Portfolio
Building a Leading Orphan Drug Portfolio Organic-Driven Growth Acquisition-Focused Growth Market penetration and Geographic expansion Focus on immediate or Strong financial and business label expansion represent prioritizing strategic near-term accretive position enables flexible significant opportu
Slide 6: Prioritizing Patient Access to Rare Disease Treatments
Prioritizing Patient Access to Rare Disease Treatments Patient-Centric Support Additional Patient Support Initiatives • Patient Access Liaisons (PALs) support throughout the patient journey which may lead to improved outcomes • Free LEMS testing 1 (VGCC ) provided for • Patient ambassador programs t
Slide 7: Diversified U.S. Commercial Portfolio of Innovative Assets
Diversified U.S. Commercial Portfolio of Innovative Assets with Multiple Expansion Opportunities 37% CAGR since launch of FIRDAPSE Catalyst’s The only evidence-based flagship commercial product approved product in the U.S. in 2019 for Lambert Eaton myasthenic syndrome (LEMS) A novel corticosteroid t
Slide 8: LEMS is a Debilitating Nerve-Muscle Communication Disorder
LEMS is a Debilitating Nerve-Muscle Communication Disorder Causing Progressive Weakness and Fatigue ~3,600 ~500 ~50% U.S. patient LEMS patients Currently identified prevalence potential believed to have patients in LEMS of up to 5,400 cancer-associated diagnostic journey not 1 patients LEMS yet trea
Slide 9: FIRDAPSE: The Only Evidence Based Approved Product in the U.S. for LEMS
FIRDAPSE: The Only Evidence Based Approved Product in the U.S. for LEMS 14 quarters of 15%+ growth with similar continued growth anticipated Key Growth Drivers – 100 mg maximum daily dose label expansion – Misdiagnosed patients in need of LEMS treatment – Growth opportunity in cancer- 1 Orphan drug
Slide 10: FIRDAPSE Well Positioned for Sustained Growth
FIRDAPSE Well Positioned for Sustained Growth >$1 Billion 1 Total Addressable Market for LEMS Population • The only evidence-based approved product in the U.S. for LEMS • Education campaigns increasing usage of LEMS diagnostic testing (VGCC) • Prescription approval rates exceed 90% across payers • H
Slide 11: DMD is a Rare and Life Threatening Neuromuscular Disorder
DMD is a Rare and Life Threatening Neuromuscular Disorder 1 ~95% ~11k-13k DMD diagnosis rate; U.S. DMD patient typically occurs at ages prevalence 2-5 years 2 ~70% ~90% DMD patients currently DMD patients are receiving concomitant treated with steroid treatment; an corticosteroids at opportunity to
Slide 12: AGAMREE: A Novel Corticosteroid Treatment for DMD
AGAMREE: A Novel Corticosteroid Treatment for DMD 84% of DMD Centers of Excellence writing prescriptions Conversions from existing treatments account for ~90% of patients Key Growth Drivers – Current standard of care is associated with behavioral and safety issues – Differentiated product profile su
Slide 13: AGAMREE Differentiated Profile Uniquely Positioned to Address Unmet Need in DMD
AGAMREE Differentiated Profile Uniquely Positioned to Address Unmet Need in DMD Treatment Landscape >$1 Billion 1 Total Addressable Market for DMD Population 2 • Clinically proven to improve muscle strength and function • Equivalent efficacy to prednisone • Potential for significant reduction of ste
Slide 14: SUMMIT Study Seeks to Demonstrate Potential Clinical Superiority of AGAMREE
SUMMIT Study Seeks to Demonstrate Potential Clinical Superiority of AGAMREE Open label, five-year follow up of ~250 DMD patients 1 across ~25 Centers of Excellence Real-world data to assess potential long-term benefits of AGAMREE to current standard of care • Behavioral improvements • Bone health (e
Slide 15: FYCOMPA: The Only Non-Competitive AMPA Receptor Antagonist for Epilepsy
FYCOMPA: The Only Non-Competitive AMPA Receptor Antagonist for Epilepsy Key Value Drivers – Patient preference to remain on existing epilepsy treatment expected to support commercial durability following anticipated generic entry May 2025 – Strong product profile: Well tolerated 1 Epilepsy is 4th mo
Slide 16: Clear and Focused Acquisition Growth Strategy
Clear and Focused Acquisition Growth Strategy Exploring all synergistic opportunities inclusive of Orphan, rare-disease products across therapeutic areas Differentiated therapies that address significant medical needs Immediate / near-immediate accretive acquisitions 16
Slide 17: Proven Partnership Model that Maximizes Value of Orphan Products
Proven Partnership Model that Maximizes Value of Orphan Products Established track Exceptional Best-in-class patient Deep expertise in record of identifying commercial support programs serving rare disease and integrating capabilities and communities orphan products expanding therapeutic footprint 1
Slide 18: 2024 Total Net Revenues Slightly Exceed Upper End of Latest Guidance
2024 Total Net Revenues Slightly Exceed Upper End of Latest Guidance 2024 Full Year Total Revenue Guidance 500 $475M - $485M 400 2024 Net Product Revenue Guidance 300 FIRDAPSE $300M - $310M 200 FYCOMPA $130M - $135M 100 AGAMREE $40M - $45M 0 Cash position of $500+M, no debt 2019 2020 2021 2022 2023
Slide 19: Accelerating Momentum: Strategic, Disciplined Execution
Accelerating Momentum: Strategic, Disciplined Execution 2024 Achievements 2025 Priorities Third quarter net product revenue growth of Continue to protect FIRDAPSE intellectual 23.2% YoY property portfolio Settled with Teva on January 8, 2025 AGAMREE Continuous pursuit of new assets Commenced U.S. co
Slide 20: AN) YD
AN) YD -2-Catalyst pharmaceuticals NASDAQ: CPRX
Catalyst Pharmaceuticals — J.P. Morgan Healthcare Conference 2025 on 6ix