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NASDAQ: CPRX Improving the Lives of Patients with Rare Diseases J.P. Morgan Healthcare Conference January 2025
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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, plans, and objectives, other than statements of historical fact included in our filings with the U.S. Securities and Exchange Commission (“SEC”), are forward-looking statements. The language reflected in these statements only speaks as of the date that appears on the front cover of the presentation; the words “may,” “will,” “could,” “would,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “potential,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward- looking statements contain these identifying words. You should not place undue reliance on forward-looking statements. While we believe that we have a reasonable basis for each forward-looking statement that we make, we caution you that these statements are based on a combination of facts and factors currently known by us and projections of future events or conditions about which we cannot be certain. Forward-looking statements in this presentation should be evaluated together with the many uncertainties that affect our business, particularly those mentioned in the “Risk Factors” section of our Annual Report on Form 10-K filed with the SEC, reporting our financial position and results of operations as of and for the year ended December 31, 2023, as well as our subsequent reports filed with the SEC. In addition, market and industry statistics contained in this presentation are based on information available to us that we believe is accurate. This information is generally based on publications that are not produced for purposes of securities offerings or economic analysis. All forward-looking statements speak only as of the date that appears on the front cover of the presentation or the date of this presentation. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future. 2
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Catalyst Pharmaceuticals is a commercial-stage biopharmaceutical company focused on in-licensing, developing, and commercializing novel medicines for patients living with rare diseases Patients are at the center of every decision we make 3
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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 to grow Ongoing Lifecycle Management Driving organic growth and expanding patient access to best-in-class rare disease therapies Strategic Portfolio Expansion Buy-and-build strategy acquiring and integrating high-value, orphan designated, synergistic assets Why we will continue to succeed Operational Excellence Continued emphasis on efficiency, innovation, and execution Proven Capabilities Successful track record of launching products and delivering value Financial Flexibility Record revenue and sustainable profitability enable future investments Growth-Oriented, Rare Disease-Focused 4
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Organic-Driven Growth Market penetration and label expansion represent significant opportunities Acquisition-Focused Growth Focus on immediate or near-term accretive orphan opportunities Strong financial and business position enables flexible decision-making Building a Leading Orphan Drug Portfolio 5 Geographic expansion prioritizing strategic markets through licensing opportunities
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Prioritizing Patient Access to Rare Disease Treatments 6 Access to Therapy • Accelerating treatment initiation with free bridge medication, insurance navigation, and copay programs Patient-Centric Support • Patient Access Liaisons (PALs) support throughout the patient journey which may lead to improved outcomes • Patient ambassador programs to foster connections within the rare disease patient community Additional Patient Support Initiatives • Free LEMS testing (VGCC1) provided for potential patients who may be eligible for FIRDAPSE • Partnerships with independent advocacy groups to deepen understanding of patient unmet needs and experiences Healthcare Provider Education • Provide disease education and outreach enabling providers to make more informed diagnostic decisions Facilitating seamless care through comprehensive personalized treatment support and patient assistance 1) Voltage-Gated Calcium Channel antibody test is a definitive diagnostic tool for Lambert-Eaton myasthenic syndrome, and Catalyst Pharmaceuticals, Inc. offers the test for free to uninsured patients or those whose insurance does not cover it.
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2019 2020 2021 2022 2023 2024 Diversified U.S. Commercial Portfolio of Innovative Assets with Multiple Expansion Opportunities The only evidence-based approved product in the U.S. for Lambert Eaton myasthenic syndrome (LEMS) The only non-competitive AMPA receptor antagonist for epilepsy A novel corticosteroid treatment for Duchenne Muscular Dystrophy (DMD) 7 37% CAGR since launch of FIRDAPSE Catalyst’s flagship commercial product in 2019 CAGR calculation includes 2019 total revenues and upper end of 2024 total revenue guidance provided on November 6, 2024.
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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 treated with FIRDAPSE LEMS patients believed to have cancer-associated LEMS 8 1) Lambert Eaton Myasthenic Syndrome is Underrecognized in Small Cell Lung Cancer: An Analysis of Real-World Data; presented IASLC 2023 World Conference on Lung Cancer; authors: David Morrell, Benjamin Drapkin, Guy Shechter, Regina Grebla.
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Key Growth Drivers – 100 mg maximum daily dose label expansion – Misdiagnosed patients in need of LEMS treatment – Growth opportunity in cancer- associated LEMS1Orphan drug exclusivity until November 2025; IP estate out to 2037; 6 Orange Book listed patents 1) Long-term follow-up, quality of life, and survival of patients with Lambert-Eaton myasthenic syndrome; authors: Alexander F Lipka, Marion I Boldingh, Erik W van Zwet, Marco WJ Schreurs, Jan BM Kuks, Chantal M Tallaksen, Maarten J Titulaer, Jan JGM Verschuuren FIRDAPSE: The Only Evidence Based Approved Product in the U.S. for LEMS 9 14 quarters of 15%+ growth with similar continued growth anticipated
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10 Total Addressable Market for LEMS Population1 >$1 Billion 10 FIRDAPSE Well Positioned for Sustained Growth • 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 • High patient response and retention • Enhanced prescriber engagement to support adoption 1) Based on internal Company estimates and anticipated 2025 market dynamics, represents future market potential and does not represent the Company’s 2025 product forecast
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~11k-13k1 ~95% ~70% U.S. DMD patient prevalence DMD diagnosis rate; typically occurs at ages 2-5 years DMD is a Rare and Life Threatening Neuromuscular Disorder Leads to loss of mobility, respiratory failure, heart issues, and premature death 11 DMD patients currently receiving concomitant steroid treatment; an opportunity to re-engage lapsed patients DMD patients are treated with corticosteroids at some point ~90%2 1) Neuroepidemiology. 2023;57(2):90-99. doi: 10.1159/000528962. Epub 2023 Jan 9. 2) Neurology. 2016 Feb 2;86(5):465-72. doi: 10.1212/WNL.0000000000002337 Steroids will continue to be the standard of care for the treatment of DMD
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Key Growth Drivers – Current standard of care is associated with behavioral and safety issues – Differentiated product profile supports earlier treatment adoption and longer treatment duration – Investing in SUMMIT study in an effort to demonstrate potential enhanced clinical benefits of AGAMREE, including behavior, bone growth, and stature 1 12 AGAMREE: A Novel Corticosteroid Treatment for DMD 84% of DMD Centers of Excellence writing prescriptions Conversions from existing treatments account for ~90% of patients Orphan drug designation offers market exclusivity until October 2030; Patent protection to 2040; 6 Orange Book listed patents 1) Clinicaltrials.gov
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1313 13 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 • Equivalent efficacy to prednisone • Potential for significant reduction of steroid-associated side effect burden when compared with other corticosteroids 1) Based on internal Company estimates and anticipated 2025 market dynamics, represents future market potential and does not represent the Company’s 2025 product forecast. 2) AGAMREE (vamorolone) [package insert]. Coral Gables, FL: Catalyst Pharmaceuticals, Inc.; 2023.
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SUMMIT Study Seeks to Demonstrate Potential Clinical Superiority of AGAMREE Real-world data to assess potential long-term benefits of AGAMREE to current standard of care • Behavioral improvements (e.g., reduced aggression) • Stature / growth parameters 14 Open label, five-year follow up of ~250 DMD patients across ~25 Centers of Excellence1 SUMMIT [SUpplemental assessMents of dMd patients Investigating ouTcomes] Evaluate AGAMREE as a monotherapy and in conjunction with other treatment options • Bone health • Ophthalmological status • Cardiovascular health . 1) Clinicaltrials.gov
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15 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 with minimal drug-drug interactions and no contraindications 2 – Seizure freedom rate of ~72% when used adjunctively 3 FYCOMPA: The Only Non-Competitive AMPA Receptor Antagonist for Epilepsy Epilepsy is 4th most common neurological disorder1 AMPA = α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid. 1) England MJ, Liverman CT, Schultz AM, Strawbridge LM, eds. Epilepsy Across the Spectrum: Promoting Health and Understanding. Washington, DC: National Academies Press (US); 2012. 2) CDC Epilepsy Data and Statistics; Epilepsy Prevalence in the US (data as of 2015); 3) Examining the Economic Impact and Implications of Epilepsy, AJMC (US); 2020.
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16 Clear and Focused Acquisition Growth Strategy Exploring all synergistic opportunities inclusive of Orphan, rare-disease products across therapeutic areas Immediate / near-immediate accretive acquisitions Differentiated therapies that address significant medical needs
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Proven Partnership Model that Maximizes Value of Orphan Products Established track record of identifying and integrating orphan products Exceptional commercial capabilities and expanding therapeutic footprint Best-in-class patient support programs Deep expertise in serving rare disease communities 17
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0 100 200 300 400 500 2019 2020 2021 2022 2023 2024 Net Revenues (USD millions) FIRDAPSE FYCOMPA AGAMREE 18 Cash position of $500+M, no debt as of 12/31/2024 2024 Net Product Revenue Guidance FIRDAPSE $300M - $310M FYCOMPA $130M - $135M AGAMREE $40M - $45M 2024 Full Year Total Revenue Guidance $475M - $485M The total revenue also included net product revenue as represented above. 2024 Total Net Revenues Slightly Exceed Upper End of Latest Guidance
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Third quarter net product revenue growth of 23.2% YoY AGAMREE Commenced U.S. commercial launch for DMD Initiated SUMMIT study Expanded in Canada through licensing (Kye Pharmaceuticals) FIRDAPSE Received U.S. approval for 100 mg maximum daily dose enhancing dosing flexibility MHLW Japan approval of NDA (DyDo Pharma) 2024 Achievements Accelerating Momentum: Strategic, Disciplined Execution 19 2025 Priorities Continue to protect FIRDAPSE intellectual property portfolio Settled with Teva on January 8, 2025 Continuous pursuit of new assets Sustain organic growth of FIRDAPSE Continue to drive the success of the U.S. AGAMREE launch Increase patient access and health equity FIRDAPSE launch in Japan (DyDo Pharma) AGAMREE application submission filing to Health Canada (Kye Pharmaceuticals)
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