Scholar Rock Holding Corporation · NASDAQ:SRRK

Scholar Rock — Cure SMA 2020

10 slides from Scholar Rock's conference presentation, Jun 12, 2020

  1. Slide 1: Clinical Development of SRK-015 for Later-Onset Spinal Muscular Atrophy

    Clinical Development of SRK-015, a Fully Human Anti-proMyostatin Monoclonal Antibody, for the Treatment of Later-Onset Spinal Muscular Atrophy George Nomikos, MD, PhD VP, Head of Medical Research Scholar Rock, Inc. Cure SMA June 12th, 2020 1 | CONFIDENTIAL AND PROPRIETARY

  2. Slide 2: Disclaimer

    Disclaimer • SRK-015 is an investigational product candidate that is currently being evaluated in a clinical trial • SRK-015 has not been approved by the U.S. Food and Drug Administration (FDA), the European Commission, or any other health authority, and the safety and effectiveness of this molecule

  3. Slide 3: SRK-015 is a Fully Human Monoclonal Antibody that Specifically Inhibits Myostatin Activation

    SRK-015 is a Fully Human Monoclonal Antibody that Specifically Inhibits Myostatin Activation 3 | CONFIDENTIAL AND PROPRIETARY

  4. Slide 4: SRK-015 Blocks Cleavage of the Myostatin Prodomain

    SRK-015 Blocks Cleavage of the Myostatin Prodomain 4 | CONFIDENTIAL AND PROPRIETARY

  5. Slide 5: SRK-015 Displays a Favorable PK Profile in Nonhuman Primates with Robust PD

    SRK-015 Displays a Favorable PK Profile in Nonhuman Primates with Robust PD (Target Engagement) 5 | CONFIDENTIAL AND PROPRIETARY

  6. Slide 6: muSRK-015P Robustly Engages Latent Myostatin in a Mouse Model of SMA

    muSRK-015P Robustly Engages Latent Myostatin in a Mouse Model of SMA (SMNΔ7) 6 | CONFIDENTIAL AND PROPRIETARY

  7. Slide 7: SRK-015 Phase 1 Study in Healthy Volunteers: PK Data Support Dosing Every 4 Weeks

    SRK-015 Phase 1 Study in Healthy Volunteers: PK Data Support Dosing Every 4 Weeks 7 | CONFIDENTIAL AND PROPRIETARY

  8. Slide 8: SRK-015 Phase 1 Study in Healthy Volunteers: PD Data Demonstrate Robust Target Engagement

    SRK-015 Phase 1 Study in Healthy Volunteers: PD Data Demonstrate Robust Target Engagement 8 | CONFIDENTIAL AND PROPRIETARY

  9. Slide 9: SRK-015 Phase 2 Trial (TOPAZ) Design

    SRK-015 Phase 2 Trial (TOPAZ) Design Cohort 1 Cohort 2 Cohort 3 • N= 20; ages 5-21 • N= 20; ages ≥2 • N= 15; ages 5-21 • Double-blind, randomized (1:1) to 2 • Open-label, single-arm • Open-label, single-arm Design • 20 mg/kg SRK-015 IV Q4W • 20 mg/kg SRK-015 IV Q4W mg/kg or 20 mg/kg • 12-month treat

  10. Slide 10: Acknowledgements

    Acknowledgements 10 | CONFIDENTIAL AND PROPRIETARY

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