Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q1 2026 Earnings Presentation
22 slides from Scholar Rock's earnings presentation, May 7, 2026
Slide 1: Q1 2026 Earnings Call
Q1 2026 Earnings Call MAY 7, 2026 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 2: Q1 2026 Earnings Call agenda and speakers
TO P I C SPEAKER Q1 2026 1 BUSINESS UPDATE David L. Hallal Chairman & Chief Executive Officer EARNINGS Akshay Vaishnaw, M.D., Ph.D. CALL 2 R&D PROGRESS President of R&D 3 Keith Woods COMMERCIAL READINESS Chief Operating Officer Vikas Sinha 4 COMPANY FINANCIALS Chief Financial Officer 5 Q&A SESSION 2
Slide 3: Forward-Looking Statements
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 its growth, strate
Slide 4: Business Update
Business Update David L. Hallal Chairman and Chief Executive Officer 4 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 5: Scholar Rock is positioned for a pivotal year ahead
Scholar Rock is positioned for a pivotal year ahead Our top priority: Bringing apitegromab to children and adults living with SMA Apitegromab PDUFA Action Date Two Fill-Finish BLA Accepted of September 30th Facilities by FDA U.S. Commercial team Included in BLA resubmission, is ready for launch prov
Slide 6: Two independent paths to apitegromab FDA approval by September 30th PDUFA date
Two independent paths to apitegromab FDA approval by September 30th PDUFA date Within 90 days of end of Early Q1 2026 Early Q2 2026 reinspection, per FDA guidelines FDA/Catalent FDA Completes FDA Classifies Indiana Meeting Reinspection Facility CATALENT INDIANA Apitegromab BLA Accepted FACILITY POTE
Slide 7: Scholar Rock is poised for a transformative year
Scholar Rock is poised for a transformative year S H A P I N G T H E F U T U R E O F T R E AT M E N T FOR PATIENTS LIVING WITH RARE NEUROMUSCULAR DISEASES A P I T E G R O M A B F O R PAT I E N T S W I T H S M A U N D E R W AY ONGOING PHASE 2 OPAL SC APITEGROMAB STUDY for apitegromab development Myos
Slide 8: R&D Progress
R&D Progress Akshay Vaishnaw, M.D., Ph.D. President of R&D 8 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 9: Advancing Scholar Rock's world-leading anti-myostatin pipeline
Advancing Scholar Rock’s world-leading anti-myostatin pipeline Program Indication Milestone Spinal Muscular PDUFA action date of CHMP opinion mid-2026 Atrophy ≥2 YoA September 30 Apitegromab Spinal Muscular Phase 2 OPAL study in infants Atrophy <2 YoA and toddlers ongoing Facioscapulohumeral Phase 2
Slide 10: Furthering our commitment to broad SMA community
Furthering our commitment to broad SMA community Ongoing Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA Focused on addressing the needs of children <2 years of age with SMA to reach patients earlier Expanding our potential impact including evaluation of apitegromab in pat
Slide 11: FSHD: Rare, devastating NMD with significant unmet need
FSHD: Rare, devastating NMD with significant unmet need >30,000 patients diagnosed in U.S. and Europe1-3; no approved therapies FSHD FLExDUX4 Mouse Model Support for apitegromab therapeutic hypothesis in FSHD Robust Increase in Muscle Randomized studies of exercise programs suggest muscle has Mass (
Slide 12: Advancing innovation with subcutaneous apitegromab and SRK-439
Advancing innovation with subcutaneous apitegromab and SRK-439 Leveraging world-leading expertise to drive continued innovation Subcutaneous Apitegromab SRK-439 Phase 1 study comparing Novel, highly potent myostatin intravenous (IV) and subcutaneous inhibitor (SC) apitegromab in healthy volunteers O
Slide 13: Commercial Readiness
Commercial Readiness Keith Woods Chief Operating Officer 13 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 14: Muscle strength and motor function remain the top unmet need in children and adults living with SMA
Muscle strength and motor function remain the top unmet need in children and adults living with SMA Scholar Rock’s disease education program continues to focus on a broader understanding of SMA 95% O F PAT I E N T S continue to experience persistent and progressive muscle atrophy that limits functio
Slide 15: U.S commercial team operating with urgency to prepare for launch
U.S commercial team operating with urgency to prepare for launch TARGETING TARGETING SMA Treatment Centers, SMA Prescribing Physicians, Multi- 140 >2,600 SMA SMA Disciplinary Care Teams Centers Prescribers Patient Engagement & Community Activation National & Regional Payers Medicare & Medicaid 15 SM
Slide 16: Strong momentum with apitegromab launch readiness in Europe in advance of mid-2026 CHMP opinion
Strong momentum with apitegromab launch readiness in Europe in advance of mid-2026 CHMP opinion 1 Building world-class team 2 Engaging the SMA community 3 Establishing access 16 SMA, Spinal Muscular Atrophy; CHMP, Committee for Medicinal Products for Human Use. © 2026 Scholar Rock, Inc. All rights r
Slide 17: Global apitegromab opportunity in SMA alone offers potential for many years of sustainable growth
Global apitegromab opportunity in SMA alone offers potential for many years of sustainable growth ~35,000 SMA patients have received an approved SMN-targeted therapy1-3 Powering Scholar Rock through the end of this decade and into the next 17 1. Biogen Q4 2023 Report; 2. Roche Q3 2024 report; 3. Nov
Slide 18: Company Financials
Company Financials Vikas Sinha Chief Financial Officer 18 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 19: Operating with financial discipline to achieve our ambitions
Operating with financial discipline to achieve our ambitions Prioritized investments focused on: Apitegromab commercial launch readiness 1 in the U.S. and Europe $480M 2 Strengthening supply chain to support expanding pipeline and anticipated growing global in cash and equivalents commercial demand
Slide 20: Closing Remarks
Closing Remarks David L. Hallal Chairman and Chief Executive Officer 20 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 21: Q&A
Q&A 21 © 2026 Scholar Rock, Inc. All rights reserved.
Slide 22: For more information, please contact
For more information, please contact: ir@scholarrock.com media@scholarrock.com Or visit us at www.scholarrock.com 22 © 2026 Scholar Rock, Inc. All rights reserved.