Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q1 2025 Earnings Presentation
21 slides from Scholar Rock's earnings presentation, May 14, 2025
Slide 1: Dedicated to Dramatically Improving the Lives of Patients with SMA
Dedicated to Dramatically Improving the Lives of Patients with SMA Q1 2025 Business Update May 14, 2025 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 2: Q1 Earnings Call Topics and Speakers
Q1 Earnings Call TO P I C SPEAKER Scholar Rock David L. Hallal Next Phase of Growth Chief Executive Officer Akshay Vaishnaw, M.D., Ph.D R&D Progress President of R&D Keith Woods Commercial Readiness Chief Operating Officer Vikas Sinha Company Financials Chief Financial Officer Q&A Session 2 © 2025 S
Slide 3: Forward-Looking Statements
Forward-Looking Statements This presentations contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, S
Slide 4: Scholar Rock Next Phase of Growth
Scholar Rock Next Phase of Growth David L. Hallal Chief Executive Officer 4 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 5: Strong Q1: Advances Scholar Rock Toward Apitegromab US Commercial Launch
Strong Q1: Advances Scholar Rock Toward Apitegromab US Commercial Launch & Series of Country Launches in Years Ahead SPINAL MUSCULAR ATROPHY Detailed Positive Phase 3 BLA Granted Priority Review MAA Validated SAPPHIRE data PDUFA date September 22 anticipated 2026 Presented at MDA approval PIPELINE A
Slide 6: Advancing Apitegromab for Patients and Families with SMA
Advancing Apitegromab for Patients and Families with SMA Apitegromab in SMA alone is opportunity to build sustainable growth through the end of this decade and into the next Commercial launch Global expansion, SMA expansion with anticipated in SMA starting w/ Europe, Ph 2 OPAL trial for LATAM, APAC
Slide 7: R&D Progress
R&D Progress Akshay Vaishnaw, M.D., Ph.D President of R&D 7 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 8: Apitegromab: Positive Phase 3 Trial with Hammersmith Motor Function Scale in SMA
APITEGROMAB Positive Phase 3 Trial with Gold Standard Hammersmith Motor Function Scale in SMA CONSISTENT 30% vs 12.5% FAVORABLE 1.8 SAFETY POINT IMPROVEMENT clinically meaningful of apitegromab patients profile consistent with (p=0.0192) benefit observed ACHIEVED ≥3PT >48 months experience across al
Slide 9: FDA Accepted BLA Under Priority Review
APITEGROMAB FDA Accepted BLA Under Priority Review (PDUFA Date September 22) By definition, a priority review designation by the FDA conveys the Potential clinical benefits of capacity of apitegromab to potentially apitegromab, as demonstrated impact unmet need in SMA by either: by our Phase 3 trial
Slide 10: Growing Our Innovative Anti-myostatin Platform: SRK-439
SRK-439 Growing Our Innovative Anti-myostatin Platform: SRK-439 Optimized for Strong Scientific Advancing to 1 Subcutaneous 2 Validation 3 Clinic Formulation Novel, highly Preclinical data On track to file the selective myostatin demonstrated IND application for inhibitor favorable muscle SRK-439 in
Slide 11: Goals of the Exploratory EMBRAZE Proof-of-Concept Study
CARDIOMETABOLIC PROGRAM Goals of the Exploratory EMBRAZE Proof-of-Concept Study PRESERVE LEAN MASS COMPARABLE WEIGHT LOSS REDUCE FAT REGAIN Exploratory program to COMPARABLE SAFETY (no added toxicities) generate insights 11 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 12: Remain Focused & On Track to Deliver R&D Priorities
Remain Focused & On Track to Deliver R&D Priorities 1 Advance toward anticipated US approval of apitegromab in Q3 2025; EU approval in 2026 2 Initiate Ph 2 OPAL Trial in Q3 2025 3 File IND for SRK-439 in Q3 2025 4 Complete clinical development plans: apitegromab in additional neuromuscular disorders
Slide 13: Commercial Readiness
Commercial Readiness Keith Woods Chief Operating Officer 13 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 14: Compelling Phase 3 Results in Patients with SMA
Compelling Phase 3 Results in Patients with SMA Apitegromab is the first and only muscle-targeted treatment to show clinically meaningful and statistically significant motor function Preparing for improvement in SMA. commercial launch of Apitegromab has the potential to apitegromab establish a new s
Slide 15: Progressive, Debilitating Muscle Degeneration Remains a Critical Unmet Need in SMA
COMMERCIAL PREPAREDNESS Progressive, Debilitating Muscle Degeneration Remains a Critical Unmet Need in SMA Despite SMN-Targeted Therapies Personal hygiene, using the toilet and the shower on my own would be huge. My four-year-old can do it on her own. It’s degrading. US Patient 90% >80% of of patien
Slide 16: The Future of SMA is in Treating the Muscle and the Motor Neuron
COMMERCIAL PREPAREDNESS The Future of SMA is in Treating the Muscle and the Motor Neuron SMA Today IN THE US WORLDWIDE 74% of neurologists agree that in the future, multiple modalities ~10,000 2/3 ~35,000 are necessary living SMA patients patients have received an to treat SMA5 with SMA1 received an
Slide 17: Scholar Rock is Positioned for a Successful Launch in SMA
COMMERCIAL PREPAREDNESS Scholar Rock is Positioned for a Successful Launch in SMA Preparing patient services and home infusion for launch* Building a customer-facing team of sales, reimbursement, and patient support personnel in mid-2025 Initiated payer outreach to top US commercial and federal paye
Slide 18: Scholar Rock Financials
Scholar Rock Financials Vikas Sinha Chief Financial Officer 18 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 19: Strong Cash Position Supports Launch & Additional Priorities
Strong Cash Position Supports Launch & Additional Priorities • US launch expected in 2025 and EU launch in 2026 1 Financial Discipline • SMA: <2 years old study initiation planned for Q3 2025 2025 2 Capital Efficient Commercial Build Priorities • Exploring additional neuromuscular indications with a
Slide 20: Delivering on 2025 Priorities Supports Long-Term Growth
Delivering on 2025 Priorities Supports Long-Term Growth 1 2 3 Regulatory Expand in Additional Rare, Disciplined Capital Approvals & Severe & Debilitating Allocation Commercialization Neuromuscular Diseases Execute Successful Apitegromab Development Efficient Commercial Build US Commercial Launch* Pr
Slide 21: Q&A
Q&A © 2025 Scholar Rock, Inc. All rights reserved. 21