Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q2 2025 Earnings Presentation
24 slides from Scholar Rock's earnings presentation, Aug 6, 2025
Slide 1: Dedicated to Dramatically Improving the Lives of Children and Adults with SMA
Dedicated to Dramatically Improving the Lives of Children and Adults with SMA Q2 2025 BUSINESS UPDATE August 6, 2025 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 2: Q2 Earnings Call
TO P I C SPEAKER David L. Hallal SCHOLAR ROCK NEXT PHASE OF GROWTH Chairman and Chief Executive Officer Akshay Vaishnaw, M.D., Ph.D R&D PROGRESS President of R&D Keith Woods COMMERCIAL READINESS Chief Operating Officer Q2 COMPANY FINANCIALS Vikas Sinha Chief Financial Officer EARNINGS Q&A SESSION CA
Slide 3: Forward-Looking Statements
Forward-Looking Statements © 2025 Scholar Rock, Inc. All rights reserved. This presentation 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
Slide 4: Next Phase of Growth
SCHOLAR ROCK NEXT PHASE OF GROWTH David L. Hallal Chairman and Chief Executive Officer 4 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 5: 2025 Priorities Support Long-Term Growth
2025 Priorities Support Long-Term Growth Apitegromab in SMA Regulatory Approvals & Commercialization • Execute Successful US Commercial Launch* • Advance EU Launch Preparedness Expand into Additional Rare, Severe & Debilitating Neuromuscular Diseases • Apitegromab Development Program: Building a Pip
Slide 6: Progress at a Glance: Our Key Milestones
Progress at a Glance: Our Key Milestones SPINAL MUSCULAR ATROPHY FDA Target Action Date MAA Validated Positive SAPPHIRE Sept 22, 2025 Anticipated 2026 approval Data Presented at Cure SMA Research Meeting PIPELINE EMBRAZE Achieved File IND for SRK-439 Initiate OPAL Trial in Q3 Primary Endpoint in 2H
Slide 7: Apitegromab in SMA
Apitegromab in SMA Fueling Scholar Rock’s long-term sustainable growth 7 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 8: R&D Progress
R&D PROGRESS Akshay Vaishnaw, M.D., Ph.D President of R&D 8 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 9: Apitegromab
APITEGROMAB Positive Phase 3 Trial with Gold Standard Hammersmith Functional Motor Scale-Expanded in SMA 1.8 30% vs 12.5% CONSISTENT FAVORABLE SAFETY POINT HFMSE of apitegromab patients clinically meaningful profile consistent with IMPROVEMENT ACHIEVED ≥3PT benefit (1.8 points) >48 months experience
Slide 10: FDA Accepted BLA Under Priority Review – Target Action Date September 22
APITEGROMAB FDA Accepted BLA Under Priority Review – Target Action Date September 22 Potential clinical benefits of apitegromab, as demonstrated by our Phase 3 trial, are underscored by the FDA’s priority review designation By definition, a priority review Being a treatment Providing a significant d
Slide 11: Expanding Our Impact: Initiating Phase 2 OPAL Trial in Q3
APITEGROMAB Expanding Our Impact: Initiating Phase 2 OPAL Trial in Q3 Studying apitegromab in patients under 2 years old ADDRESSING THE NEEDS OF INFANTS & TODDLERS WITH SMA Potential to alter the course of SMA in children under 2 EXPANDING OUR IMPACT Including evaluation of apitegromab in patients w
Slide 12: Apitegromab: Pipeline in a Product
APITEGROMAB Apitegromab: Pipeline in a Product Potential to address additional rare, severe and debilitating neuromuscular disorders MuSRK-015P Increased Muscle Force and Dystrophin Protein MuSRK-015P Increased Muscle Force in Combination With an Exon Skipper in a Mouse Model of DMD1 in a Model of F
Slide 13: EMBRAZE Proof-of-Concept Study Achieved Goals
EMBRAZE EMBRAZE Proof-of-Concept Study Achieved Goals placebo + apitegromab tirzepatide + tirzepatide Combining apitegromab 10 mg/kg with tirzepatide 54.9 % over 24 weeks 4.2 lbs lean mass REDUCTION in lean mass loss versus tirzepatide alone (p=0.001) HIGHER QUALITY 30 % % of Mass Loss from Lean Mas
Slide 14: Growing Our Innovative Anti-myostatin Platform: SRK-439
SRK-439 Growing Our Innovative Anti-myostatin Platform: SRK-439 OPTIMIZED FOR SUBCUTANEOUS Percent Change in Whole Body Lean Mass from Baseline ADMINISTRATION 25 * ** * Novel, highly selective myostatin inhibitor 20 % Change from Baseline 15 STRONG SCIENTIFIC VALIDATION 10 Preclinical data demonstra
Slide 15: Remain Focused On Delivering R&D Priorities
Remain Focused On Delivering R&D Priorities Advance toward anticipated US approval of 1 apitegromab in Q3 2025; anticipated EU approval in 2026 2 Initiate Ph 2 OPAL Trial in Q3 2025 3 File IND for SRK-439 in 2H 2025 Complete clinical development plans: apitegromab 4 in additional rare, severe, and d
Slide 16: Commercial Readiness
COMMERCIAL READINESS Keith Woods Chief Operating Officer 16 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 17: Ushering in a New Treatment Era for SMA
Ushering in a New Treatment Era for SMA Preparing for a SMA is a progressive Apitegromab is the first global launch of and devastating and only muscle-targeted disease that leads to treatment to show clinically apitegromab in loss of mobility, meaningful and statistically SMA* limited activities of
Slide 18: Progressive, Debilitating Muscle Degeneration Remains a Critical Unmet Need in SMA
C COC OOM MM EE R MM EC R C IC R IA AI L L LR A PERA P ED R EP P IANA RER ESD ESD NNE SE S S 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 90% O F PAT I E N T S be hu
Slide 19: Scholar Rock is Positioned for a Successful U.S. Launch of Apitegromab in SMA
COMMERCIAL READINESS Scholar Rock is Positioned for a Successful U.S. Launch of Apitegromab in SMA* Preparing patient services and home infusion for launch Deploying customer-facing teams Continued expansion of U.S. payer outreach in Q2/Q3 Ongoing disease education and SMA stakeholder engagement 19
Slide 20: The Future of SMA is in Treating the Muscle and the Motor Neuron
C COOM MMM EE R RCC II A A LL R P ER A EDP IANREESDSN E S S The Future of SMA is in Treating the Muscle and the Motor Neuron S M A TO D AY Many researchers IN THE US WORLDWIDE believe that it will take a combination of SMN-dependent ~10,000 2/3 ~35,000 and SMN-independent treatments to provide Livin
Slide 21: Scholar Rock Financials
Scholar Rock FINANCIALS Vikas Sinha Chief Financial Officer 21 © 2025 Scholar Rock, Inc. All rights reserved.
Slide 22: Strong Cash Position Supports Launch & Additional Priorities
Strong Cash Position Supports Launch & Additional Priorities 1 Financial • US launch expected in Discipline 2025 and EU launch in 2026 • SMA: <2 years old study initiation planned for Q3 2025 2025 2 Capital Efficient Commercial Build PRIORITIES • Expand in additional rare, severe, and debilitating n
Slide 23: 2025 Priorities Support Long-Term Growth
2025 Priorities Support Long-Term Growth Apitegromab in SMA Regulatory Approvals & Commercialization • Execute Successful US Commercial Launch* • Advance EU Launch Preparedness Expand into Additional Rare, Severe & Debilitating Neuromuscular Diseases • Apitegromab Development Program: Building a Pip
Slide 24: Q&A
Q&A 24 © 2025 Scholar Rock, Inc. All rights reserved.