Scholar Rock Holding Corporation · NASDAQ:SRRK

Scholar Rock — Corporate Update (September 2026)

22 slides from Scholar Rock's corporate update, Sep 14, 2026

  1. Slide 1: ISEMBYLD (apitegromab-mstn) FDA Approval Conference Call

    ISEMBYLD (apitegromab-mstn) FDA Approval Conference Call September 14, 2026 © 2026 Scholar Rock, Inc. All rights reserved.

  2. Slide 2: Forward-Looking Statements

    2 Forward-Looking Statements Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, “Scholar Rock”), including without limitation, Scholar Rock’s expectations regarding the U.S. commerc

  3. Slide 3: ISEMBYLD FDA Approval

    ISEMBYLD FDA Approval David L. Hallal Chairman & Chief Executive Officer © 2026 Scholar Rock, Inc. All rights reserved.

  4. Slide 4: First and only muscle-targeted therapy for SMA: FDA approved

    4 SMA, Spinal Muscular Atrophy. FIRST AND ONLY muscle-targeted therapy for adults and children ≥2 years of age with SMA FDA APPROVED NOW © 2026 Scholar Rock, Inc. All rights reserved.

  5. Slide 5: Ushering in the next phase of innovation for the SMA community

    5 Ushering in the next phase of innovation for the SMA community 1. ISEMBYLD prescribing information. HMFSE, Hammersmith Functional Motor Scale-Expanded. *Nominal p-value ISEMBYLD (apitegromab-mstn) is indicated for the treatment of spinal muscular atrophy (SMA) in adults and children 2 years of age

  6. Slide 6: Working to bring to SMA patients globally

    6 WORKING TO BRING TO SMA PATIENTS GLOBALLY Building a 50-country operating platform to serve patients with rare, severe neuromuscular diseases UNITED STATES EUROPE REST OF WORLD Pending regulatory approvals outside the United States. © 2026 Scholar Rock, Inc. All rights reserved. 6 JAPAN

  7. Slide 7: ISEMBYLD FDA Approval and Clinical Program

    ISEMBYLD FDA Approval and Clinical Program Akshay Vaishnaw, M.D., Ph.D. President of R&D © 2026 Scholar Rock, Inc. All rights reserved.

  8. Slide 8: First and only FDA-approved myostatin inhibitor

    8 FIRST AND ONLY FDA-approved myostatin inhibitor FDA APPROVED NOW © 2026 Scholar Rock, Inc. All rights reserved.

  9. Slide 9: Motor neuron loss and muscle atrophy in SMA lead to progressive muscle weakness

    9 Motor neuron loss and muscle atrophy in SMA lead to progressive muscle weakness SMA, Spinal muscular atrophy; SMN, Survival motor neuron. Figure adapted from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf. HEALTHY MOTOR UNIT MOTOR UNIT IN SMA Hea

  10. Slide 10: ISEMBYLD has been rigorously studied in children and adults with SMA

    10 SMA, Spinal Muscular Atrophy; SMN, Survival Motor Neuron. 1. Barrett D, et al. Adv Ther 2021;38;3203-3222. 2. Crawford TO, et al. Front Neurol 2024: 15:1419791. 3. Crawford TO, et al. Presented at World Muscle Society, Oct 8-12, 2024. 4. Crawford, TO, et al. Lancet Neurol. 2025 Sep; 24(9):727-739

  11. Slide 11: Positive Phase 3 trial demonstrates clinically meaningful benefit in children and adults

    11 Positive Phase 3 trial demonstrates robust, clinically meaningful benefit of ISEMBYLD in children and adults living with SMA HFMSE, Hammersmith Functional Motor Scale-Expanded; SMA, Spinal Muscular Atrophy; SMN, Survival Motor Neuron. *Nominal p-value; Based on ISEMBYLD 10 mg/kg + SMN-targeted th

  12. Slide 12: ISEMBYLD well-established safety profile

    12 *Occurring in ≥20% of ISEMBYLD-treated patients and more frequently than in placebo. An increased risk of fractures, including serious fractures, was observed with ISEMBYLD. All AEs were coded using the MedDRA (Medical Dictionary for Regulatory Activities Terminology) version 26.1. ISEMBYLD well-

  13. Slide 13: ISEMBYLD U.S. Launch

    ISEMBYLD U.S. Launch Keith Woods Chief Operating Officer © 2026 Scholar Rock, Inc. All rights reserved.

  14. Slide 14: Persistent and progressive muscle atrophy is a critical unmet need in SMA

    14 Persistent and progressive muscle atrophy is a critical unmet need in SMA despite SMN-targeted treatments 1 Parsons JA, et al. Ann Clin Transl Neurol. 2025;0:1–16. © 2026 Scholar Rock, Inc. All rights reserved. Continue to experience persistent and progressive muscle atrophy that limits both func

  15. Slide 15: U.S. launch underway

    15 SMA, Spinal Muscular Atrophy; SMN, Survival Motor Neuron. © 2026 Scholar Rock, Inc. All rights reserved. U.S. LAUNCH UNDERWAY 6,600 Children and adults with SMA in the U.S. receiving an SMN2-targeted treatment 2,600 PRESCRIBING PHYSICIANS 140 TREATMENT CENTERS COMPELLING CLINICAL BENEFIT EXPECT S

  16. Slide 16: Scholar Rock Supports patient support program

    16 Scholar Rock Supports patient support program designed to offer personalized, comprehensive services to patients and caregivers *For eligible patients © 2026 Scholar Rock, Inc. All rights reserved. Disease & Treatment Education Insurance Navigation Infusion Day Logistics Caregiver Support Financi

  17. Slide 17: Establishing broad and reliable access to ISEMBYLD

    17 Establishing broad and reliable access to ISEMBYLD SMA, Spinal Muscular Atrophy. SMN, Survival Motor Neuron. COMMERCIAL GOVERNMENT ~50% ~50% EXTREME RARITY: ~6,600 patients with SMA on an SMN2-targeted treatment in the U.S. SEVERITY: devastating and progressive disease, despite the use of chronic

  18. Slide 18: Ready to serve the SMA community in the U.S. with ISEMBYLD

    18 Ready to serve the SMA community in the U.S. with ISEMBYLD Available for infusion in coming days Centers of Excellence, infusion centers, or the patient’s home COMMERCIAL SUPPLY SITE OF CARE © 2026 Scholar Rock, Inc. All rights reserved. Optionality for patients, caregivers, providers, and payers

  19. Slide 19: Financial Update

    Financial Update Vikas Sinha Chief Financial Officer © 2026 Scholar Rock, Inc. All rights reserved.

  20. Slide 20: Strong financial position

    20 Strong financial position Additional $150 million available under debt facility 1 Rare Pediatric Disease Priority Review Voucher Scholar Rock intends to monetize 2 3 Begin to recognize revenue from ISEMBYLD $492M IN CASH, CASH EQUIVALENTS, AND MARKETABLE SECURITIES AS OF JUNE 30, 2026 PRIORITIZED

  21. Slide 21: First and only muscle-targeted therapy for SMA: FDA approved

    21 SMA, Spinal Muscular Atrophy. FIRST AND ONLY muscle-targeted therapy for adults and children ≥2 years of age with SMA FDA APPROVED NOW © 2026 Scholar Rock, Inc. All rights reserved.

  22. Slide 22: Thank You

    ir@scholarrock.com media@scholarrock.com www.scholarrock.com THANK YOU © 2026 Scholar Rock, Inc. All rights reserved.

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