Biomarin Pharmaceutical Inc. · NASDAQ:BMRN

Biomarin Pharmaceutical — 42nd Annual J.P. Morgan Healthcare Conference (January 2024)

16 slides from Biomarin Pharmaceutical's conference presentation, Jan 8, 2024

  1. Slide 1: 42nd Annual J.P. Morgan Healthcare Conference

    42nd Annual J.P. Morgan Healthcare Conference BioMarin Pharmaceutical Inc. Alexander Hardy President and Chief Executive Officer January 8, 2024

  2. Slide 2: Forward-Looking Statements

    Forward-Looking Statements This non-confidential presentation contains “forward-looking statements” about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the profitability opportunities and expected financial performance of BioMarin

  3. Slide 3: My Background, Why I Chose BioMarin, Key Priorities Ahead

    1 My Background and Relevant Experience 2 Why I Chose BioMarin 3 Key Priorities Ahead 3

  4. Slide 4: A bit about me

    Track record of innovation, accelerating new product growth A bit about me…. and delivering operational efficiencies  Responsible for Global Product Strategy Lifecycle Management  Led late-stage strategy, launch and LCM across Experience oncology, benign hematology, neuroscience, immunology and op

  5. Slide 5: Strong Revenue Growth Potential, Significant Profitability Opportunities

     Significant multi-year growth driver with VOXZOGO® in achondroplasia and new indications Strong Revenue Growth Potential  ROCTAVIAN™ for severe hemophilia A as a potential standard of care gene therapy  Durable, profitable, “IRA-insulated” annuity-like base business  8 approved products driving

  6. Slide 6: Products Approved in the Last Years

    5 Products Approved in the Last 9 Years Enzyme Replacement Therapies Achondroplasia Over xx,xxx Patients Patient lives benefitting impacted by our from our therapies therapies Phenylketonuria Hemophilia A Gene Therapy 6

  7. Slide 7: VOXZOGO patient stories

    Eve Annika Kennedy Kaye, “KK” Age 3, started VOXZOGO at age 1.5 At age 14, started VOXZOGO at age 8 Age 10, started VOXZOGO at age 8 on therapy for 1.5+ years on therapy for 6+ years on therapy for 2+ years Label Expansion For Long-Term Efficacy Impact On An Earlier Start And Safety Quality of Life

  8. Slide 8: Linking our Foundational Core Four to the Highest Potential Commercial Medicines

    Linking our Foundational “Core Four” to the Highest Potential Early-stage Pipeline Optionality: Commercial Medicines  7 candidates in clinical/IND-enabling development  Dozens of assets in research  Multiple modalities and therapeutic areas Thoughts on R&D Framework:  Focus on assets with highes

  9. Slide 9: Key priorities: Accelerate and maximize the VOXZOGO opportunity

    ACCELERATE AND MAXIMIZE THE  VOXZOGO indication expansion is the top priority for BioMarin VOXZOGO  Maintain momentum on achondroplasia launch and leverage broadened labels OPPORTUNITY ESTABLISH  Important progress made in market access and readiness ROCTAVIAN OPPORTUNITY  2024 and 2025 will fur

  10. Slide 10: Leveraging 1000+ patient years of experience to accelerate new indications

    Leveraging more than 1000 patient years of experience in achondroplasia to accelerate new indications Label expansion has increased HCP intent to prescribe across all ages, Pivotal programs to start 1H24 * including infants and toddlers Ped Endos are taking ownership of VOXZOGO and coordinating mult

  11. Slide 11: VOXZOGO's commercial launch has outperformed rare disease analogs

    VOXZOGO’s commercial launch has outperformed numerous rare disease analogs Benchmarking Orphan Drug Post Launch Revenue Trajectories REVENUE (USD, M) QUARTERS POST LAUNCH 11

  12. Slide 12: Ensuring VOXZOGO Supply Capacity to Serve Patients

    Capacity to Serve VOXZOGO Patients Ensuring VOXZOGO Supply Secured additional near-term 1 fill/finish capacity with CMO to expand VOXZOGO supply 8,000 ~50% 1 VOXZOGO / 6,000 ROCTAVIAN Plan for fully unconstrained supply 2 by mid-2024 2,800 1,200 3 Continued capacity expansion to supply long-term gro

  13. Slide 13: ROCTAVIAN: Value Proposition Remains Strong — Ramp Taking Time

    ROCTAVIAN: Value Proposition Remains Strong — Ramp Taking Time Durable, One-time Treatment Offering Superior Outcomes Versus Standard of Care Cost Effective Solution Global Commercialization Strategy Utilizing Existing Global Infrastructure Targeted Marketing Strategy Significant reduction in treate

  14. Slide 14: Maturing Guided Line Items and More Consistent Cadence of Updates

    Maturing Guided Line Items More Consistent Cadence Investor Day 2024 (4Q23 Earnings Call) and Content of Updates (3Q24)  Total Revenues  Communicate business updates  New leadership perspectives on and objectives each quarter company strategy and long-term  Non-GAAP Operating Margin aspirations

  15. Slide 15: Priorities: VOXZOGO, ROCTAVIAN, R&D Assets, Profitability

    1 Accelerate VOXZOGO Expansion 2 Execute on ROCTAVIAN 3 Prioritize R&D Assets 4 Increase Profitability Faster 15

  16. Slide 16: Thank You

    Thank You Transforming Lives Through Genetic Discovery

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