Catalyst Pharmaceuticals, Inc. · NASDAQ:CPRX
Catalyst Pharmaceuticals — Investor Presentation (January 2026)
21 slides from Catalyst Pharmaceuticals's investor presentation, Jan 12, 2026
Slide 1: Improving the Lives of Patients Living with Rare Diseases
NASDAQ: CPRX Improving the Lives of Patients Living with Rare Diseases January 2026 Exhibit 99.1
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: Commercial-stage biopharmaceutical company focused on rare diseases
Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on acquiring, in-licensing, developing, and commercializing novel medicines for patients living with rare diseases Patients are at the center of every decision we make
Slide 4: Growth Through Patient-First Approach
Growth Through Patient-First Approach Educating rare disease communities on access to care and adherence A Profitable Leader in Rare Disease Therapeutics Forging New Paths for Rare Disease Patients Patient-driven Strategy 1) Nine months ended Sept. 30, 2025; 2) Based on preliminary unaudited 2025 fi
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 Broad copay programs Best-in-Class Patient Support Long-term commitmen
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 Opportunities with up to ~$500M in peak sales Immediate / near-term accretive acquisitions Clinical-
Slide 7: Proven Business Development Engine Maximizes Rare Disease Assets
Proven Business Development Engine Maximizes Rare Disease Assets 100+ BD assessments in 2025 Adept at extending asset value Integrated development and commercial capabilities to enable lifecycle management Established rare disease infrastructure Product-dedicated rare disease field forces and back-o
Slide 8: Diversified U.S. Commercial Portfolio of Innovative Assets
The only evidence-based approved product in the U.S. for LEMS Diversified U.S. Commercial Portfolio of Innovative Assets with Multiple Expansion Opportunities Frontline therapy for idiopathic and cancer-associated LEMS markets $1B+ total addressable market A novel corticosteroid treatment for DMD wi
Slide 9: LEMS is a Debilitating Nerve-Muscle Communication Disorder
LEMS is a Debilitating Nerve-Muscle Communication Disorder Causing Progressive Weakness and Fatigue LEMS Patients Often Experience a Long Diagnostic Journey ~3,600 >500 ~50% U.S. patient prevalence potential of up to 5,400 patients1 Currently identified patients in LEMS diagnostic journey not yet tr
Slide 10: FIRDAPSE: One Molecule, Two Growing Markets
Two distinct markets representing a combined $1B+ opportunity Up to 5,400 patients in U.S. 1) Estimated life expectancy for CA-LEMS patients ~17mos FIRDAPSE: One Molecule, Two Growing Markets Estimated Prevalence Addressable Patient Population NEUROLOGY Idiopathic LEMS (~50%) ONCOLOGY CA-LEMS (~50%)
Slide 11: FIRDAPSE: Expanding Market Penetration in Idiopathic LEMS
FIRDAPSE: Expanding Market Penetration in Idiopathic LEMS Leverage existing Catalyst Pathways program, which provides easy-to-understand disease, treatment, and financial support to help patients navigate their healthcare journey. Ongoing patient sourcing Converting misdiagnosed gMG patients Growing
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 VGCC testing access Driving NCCN clinical practice guidelines awareness Forging strategic relationships Embedding change in the oncology care pathways Contracted with a National Testing Lab to conduct frictionless VG
Slide 13: DMD is a Rare and Life-Threatening Neuromuscular Disorder
~11k-13k1 ~95% ~70% U.S. DMD patient prevalence DMD diagnosis typically occurs between ages 2 and 5 DMD is a Rare and Life-Threatening Neuromuscular Disorder DMD leads to loss of mobility, respiratory failure, heart issues, and premature death DMD patients currently receiving concomitant steroid tre
Slide 14: AGAMREE: Differentiated Profile Uniquely Positioned in DMD
AGAMREE: Differentiated Profile Uniquely Positioned to Address Unmet Need in DMD Treatment Landscape Total Addressable Market for DMD Population1 >$1 Billion Clinically proven to improve muscle strength and function2 Data shows equivalent efficacy to prednisone Potential for significant reduction of
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 Acceptance ~90% patient retention rate ~90% penetration across all DMD Centers of Excellence >85% patients are reimbursed 85% patient conversion from branded / generic agents C
Slide 16: AGAMREE: SUMMIT Study Evaluating Potential Long-term Safety Benefits
AGAMREE: SUMMIT Study Evaluating Potential Long-term Safety Benefits Real-world data to assess potential long-term benefits of AGAMREE over standard of care Behavioral improvements (e.g., reduced aggression) Stature/growth parameters Bone health Open label, five-year follow up on ~250 DMD patients a
Slide 17: 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% when used adjunctively3 Generic entry (Q2 ’25, tablets; Q4’25 oral solution) less significant in the epilepsy space FYCOMPA: The First Non-Competitive AMP
Slide 18: Notable Commercial Performance: Sustained Momentum
$148.4 $126.4 (in USD, millions) Notable Commercial Performance: Sustained Momentum Q3’24 & Q3’25 Record Q3’25 and Year-to-Date 2025 Net Product Revenues $436.3 $347.5 (in USD, millions) Nine months ended September 30, 2024 & Nine months ended September 30, 2025 Total Net Product Revenue Growth +17.
Slide 19: Updated 2025 Net Product Revenue Guidance
Updated 2025 Net Product Revenue Guidance FIRDAPSE$355M - $360M Within guidance AGAMREE$105M - $115M At or slightly exceed upper end of guidance FYCOMPA$100M - $110M Upper end of guidance 2025 Full Year Total Revenue Guidance Increased to be Between $565M - $585M Upper end of guidance Total revenue
Slide 20: 2025 Achievements and 2026 Priorities
Third quarter net product revenue growth of 17.4% YoY Purchased >1M shares through December 31, 2025, under current share repurchase program FIRDAPSE NCCN guidelines updated to include amifampridine (FIRDAPSE) as LEMS treatment recommendation Settled patent litigation with Teva Pharmaceuticals and L
Slide 21: Catalyst Pharmaceuticals
AA, -2-Catalyst pharmaceuticals NASDAQ: CPRX
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