Catalyst Pharmaceuticals, Inc. · NASDAQ:CPRX
Catalyst Pharmaceuticals — Q4 2025 Earnings Presentation
21 slides from Catalyst Pharmaceuticals's earnings presentation, Feb 26, 2026
Slide 1: Improving the Lives of Patients Living with Rare Diseases
Improving the Lives of Patients Living with Rare Diseases NASDAQ: CPRX March 2026
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 acquiring, in- licensing, developing, and commercializing novel medicines for patients living with rare diseases 3
Slide 4: A Profitable Leader in Rare Disease Therapeutics
A Profitable Leader in Rare Disease Therapeutics Forging New Paths for • the only evidence-based approved treatment for LEMS Rare Disease Patients • a differentiated corticosteroid treatment for DMD Growth Through • Bespoke patient programs supporting identification Patient-First Approach through ev
Slide 5: Catalyst Pathways: Personalized Patient Support
Catalyst Pathways: Personalized Patient Support Accelerating access, ensuring compliance, and optimizing care Industry Leading Patient HUB Accelerating treatment initiation • Free bridge medication • Personalized insurance navigation Best-in-Class Patient • Broad copay programs Support Long-term com
Slide 6: Clear and Focused Acquisition Strategy to Supplement Organic Growth
Clear and Focused Acquisition Strategy to Supplement Organic Growth Financial flexibility to deploy >$1B toward: Rare disease products across diverse therapeutic areas, agnostic to treatment modality Immediate / near-term accretive acquisitions and clinical- stage opportunities (Ph 3) with robust an
Slide 7: Proven Business Development Engine Maximizes Rare Disease Assets
Proven Business Development Engine Maximizes Rare Disease Assets Established rare disease infrastructure Product-dedicated rare disease field forces and back-office resources to drive commercial success Demonstrated success integrating and launching products Current portfolio comprised of both in-ho
Slide 8: Diversified U.S. Commercial Portfolio of Innovative Assets with Multiple Expansion Opportunities
Diversified U.S. Commercial Portfolio of Innovative Assets with Multiple Expansion Opportunities The only evidence-based A novel corticosteroid The first non-competitive approved product in the treatment for DMD with AMPA receptor agonist for U.S. for LEMS a differentiated profile epilepsy Frontline
Slide 9: LEMS is a Debilitating Nerve-Muscle Communication Disorder
LEMS is a Debilitating Nerve-Muscle Communication Disorder Causing Progressive Weakness and Fatigue ~3,600 ~50% >600 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 patients1 LEMS yet treat
Slide 10: FIRDAPSE: One Molecule, Two Growing Markets
FIRDAPSE: One Molecule, Two Growing Markets Idiopathic LEMS CA-LEMS (~50%) (~50%) NEUROLOGY ONCOLOGY Estimated Prevalence ~1,800 ~1,800 Life expectancy unaffected Shorter life expectancy1 Up to 5,400 patients in U.S. Newly Diagnosed Two distinct Patients each Year ~150 ~900 markets representing Addr
Slide 11: FIRDAPSE: Expanding Market Penetration in Idiopathic LEMS
FIRDAPSE: Expanding Market Penetration in Idiopathic LEMS Ongoing patient Converting misdiagnosed Increasing LEMS Growing testing sourcing gMG patients diagnoses rates awareness Tracking >600 patients; Generalized myasthenia Layering VGCC testing Driving awareness of the 50% of new FIRDAPSE gravis (
Slide 12: FIRDAPSE: Unlocking the Next Phase of Growth in Cancer-Associated LEMS
FIRDAPSE: Unlocking the Next Phase of Growth in Cancer-Associated LEMS Streamlining Driving NCCN Embedding change in Forging strategic VGCC testing clinical practice the oncology care relationships access guidelines awareness pathways Contracted with a National Updated NCCN SCLC Collaborating with D
Slide 13: DMD is a Rare and Life-Threatening Neuromuscular Disorder
DMD is a Rare and Life-Threatening Neuromuscular Disorder ~11k-13k1 ~95% U.S. DMD patient DMD diagnosis typically prevalence occurs between ages 2 and 5 ~90%2 ~70% DMD patients are DMD patients currently treated with receiving concomitant corticosteroids at steroid treatment; an some point opportuni
Slide 14: 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 Total Addressable Market for DMD Population1 • Clinically proven to improve muscle strength and function2 • Data shows equivalent efficacy to prednisone • Potential for significant reduct
Slide 15: AGAMREE: Strategy to Achieve Full Potential
AGAMREE: Strategy to Achieve Full Potential A multi-faceted approach to lifecycle management Strong Profile and Outstanding ~90% penetration across all DMD Centers of Excellence >85% patients are reimbursed ~90% patient retention rate Acceptance Strengthening Expanding Realizing Full Differentiation
Slide 16: AGAMREE: SUMMIT Study Evaluating Potential Long-term Safety Benefits
AGAMREE: SUMMIT Study Evaluating Potential Long-term Safety Benefits Open label, five-year follow up on ~250 DMD patients across ~25 Centers of Excellence1 Real-world data to assess potential long-term benefits of AGAMREE over standard of care • Behavioral changes • Cardiovascular health (e.g., redu
Slide 17: FYCOMPA: The First Non-Competitive AMPA Receptor Antagonist for Epilepsy
FYCOMPA: The First Non-Competitive AMPA Receptor Antagonist for Epilepsy Market Dynamics – Strong product profile: Well- tolerated with minimal drug-drug interactions and no contraindications2 – Seizure freedom rate of ~72% Epilepsy is 4th most common neurological disorder1 when used adjunctively3 –
Slide 18: Notable Commercial Performance: Sustained Momentum
Notable Commercial Performance: Sustained Momentum Q4’24 & Q4’25 Years ended December 31, 2024 & 2025 Total Net Product Revenue Growth +7.5% Total Net Product Revenue Growth +20.3% $500.0 $700.0 $588.8 $600.0 $400.0 $489.3 $113.3 (in USD, millions) (in USD, millions) $500.0 $300.0 $400.0 $137.3 $117
Slide 19: Promoted Products Continue to Drive Total Revenue Growth
Promoted Products Continue to Drive Total Revenue Growth Promoted Product Revenue, Net Expected 700 to Grow by 20% or More in 2026 FIRDAPSE $435M - $450M 600 AGAMREE $140M - $150M Promoted Products Revenue 500 2026 Full Year Promoted Product Revenue Guidance $575M - $600M (USD millions) 400 300 200
Slide 20: Accelerating Momentum: Strategic, Disciplined Execution
Accelerating Momentum: Strategic, Disciplined Execution 2025 Achievements 2026 Priorities Full year net product revenue growth of 20.3% YoY BUSINESS DEVELOPMENT Pursue immediate/near-term accretive acquisitions, Purchased >1M shares through December 31, 2025, under including clinical-stage opportuni
Slide 21: NASDAQ: CPRX
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Catalyst Pharmaceuticals — Q4 2025 Earnings Presentation on 6ix