Scholar Rock Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Key Business Updates

Executive Summary

• Apitegromab Biologics License Application (BLA) resubmission and U.S. launch anticipated in 2026, pending FDA approval
• Positive FDA feedback on Catalent Indiana facility remediation; no further corrective actions requested
• New debt facility secured: up to \$550 million in non-dilutive capital
• European launch expected in H2 2026, starting in Germany (pending EMA approval)
• Cash, cash equivalents, and marketable securities at \$367.6 million as of December 31, 2025
• Multiple pipeline advancements in SMA and FSHD programs; new fill-finish facility and subcutaneous formulation developments
• Significant financial losses reported, but company remains well-funded for commercialization and R&D

Business and Regulatory Updates

Apitegromab – SMA Program

Scholar Rock’s lead candidate, apitegromab, is an investigational monoclonal antibody targeting myostatin activation in skeletal muscle, representing a novel approach in treating spinal muscular atrophy (SMA). It is the first muscle-targeted therapy to demonstrate statistically significant and clinically meaningful benefits in the pivotal Phase 3 SAPPHIRE trial.

• BLA Resubmission & U.S. Launch: Scholar Rock plans to resubmit the Biologics License Application for apitegromab following a successful FDA reinspection of the Catalent Indiana facility, which underwent remediation by Novo Nordisk. A recent meeting with the FDA was constructive, and no further corrective actions were requested, suggesting a positive regulatory outlook. The U.S. launch is expected in 2026, subject to regulatory approval.
• Commercial Preparation: The U.S. commercial team is expanding its reach and deepening relationships with SMA treatment centers and payers, focusing on educating stakeholders about the importance of treating the full motor unit (motor neuron and muscle).
• EMA Review: The European Medicines Agency (EMA) is reviewing the apitegromab Marketing Authorisation Application (MAA), with a decision anticipated in mid-2026. European launch is expected in the second half of 2026, beginning with Germany.
• Supply Chain & Manufacturing: Technology transfer continues at a second U.S.-based fill-finish facility to ensure supply continuity and support future commercial demand. Scholar Rock plans to submit a supplemental BLA (sBLA) for this facility in 2026. Engineering and manufacturing runs are ongoing.
• Phase 2 OPAL Study: Enrollment and patient dosing continue in the OPAL trial, designed to evaluate apitegromab in infants and toddlers (under two years old) with SMA who have received approved SMN1-targeted gene therapy or are receiving SMN2-targeted therapy.
• Subcutaneous Formulation: Scholar Rock is advancing a subcutaneous formulation of apitegromab, intended for self- or caregiver administration via autoinjector. Phase 1 study in healthy volunteers has been completed, with further regulatory engagement planned with FDA and EMA.

FSHD Program

• Scholar Rock will initiate the Phase 2 FORGE trial for apitegromab in facioscapulohumeral muscular dystrophy (FSHD) in mid-2026. The IND application is cleared, and the trial will be randomized, double-blind, and placebo-controlled.

SRK-439 Pipeline Update

• SRK-439, a novel subcutaneous myostatin inhibitor, is in Phase 1 trials in healthy volunteers. The compound shows high affinity and selectivity for pro- and latent myostatin and does not bind GDF11 or Activin A. Topline data from the ongoing trial is expected in the second half of 2026.

Financial Summary and Capital Position

• Net Loss: Scholar Rock reported a net loss of \$91.0 million for Q4 2025 (including \$19.4 million in stock-based compensation), up from \$66.5 million in Q4 2024. Net loss per share was \$0.76 for Q4 2025, compared to \$0.61 in Q4 2024.
• Full Year Results: Net loss for the full year 2025 was \$377.9 million (including \$75.6 million in stock-based compensation), compared to \$246.3 million in 2024. Net loss per share was \$3.29 for 2025, versus \$2.47 in 2024.
• R&D Expenses: R&D expense was \$46.9 million in Q4 2025 and \$208.4 million for the full year, up from \$50.4 million and \$184.5 million in 2024, respectively.
• G&A Expenses: General and administrative expenses were \$45.0 million in Q4 2025 and \$176.2 million for the full year, significantly higher than \$19.0 million and \$67.5 million in 2024, reflecting increased stock-based compensation.
• Cash Position: As of December 31, 2025, Scholar Rock had \$367.6 million in cash, cash equivalents, and marketable securities. This includes \$60.4 million from the exercise of outstanding warrants in Q4 2025.

Debt Facility

• Scholar Rock secured a new debt facility with Blue Owl Capital, providing up to \$550 million in non-dilutive capital. The facility matures in February 2032 and is structured as follows:
  • \$100 million available at closing, used to retire prior debt with Oxford Finance
  • \$100 million to be drawn in Q1 2026
  • Up to \$150 million available upon FDA approval of apitegromab
  • Option for up to \$200 million in additional incremental facilities at mutual consent

Investor Highlights & Potential Share Price Impact

• Apitegromab BLA resubmission and potential U.S. launch in 2026 are major catalysts. Positive FDA feedback on facility remediation increases probability of approval.
• European approval and launch could open new revenue streams in H2 2026.
• New debt facility strengthens financial position, supports commercialization and pipeline development, and reduces dilution risk for shareholders.
• Advancements in pipeline and manufacturing bolster future growth prospects.
• Significant financial losses and increased expenses may concern investors, but strong cash position and capital access mitigate risk.
• Any regulatory delays, negative trial results, or unresolved manufacturing issues could negatively impact share price.
• Successful commercialization and expansion into new indications (FSHD) would be strong positive drivers.

Conference Call

Scholar Rock will host a conference call and webcast on Tuesday, March 3, at 8:00 a.m. ET to review its financial results and business updates. Registration is required for telephone participation, and a replay will be available on the company website for approximately 90 days.

About Scholar Rock

Scholar Rock is a late-stage biopharmaceutical company focusing on developing and commercializing apitegromab for SMA and other rare neuromuscular diseases. The company leverages its proprietary platform in myostatin biology to develop novel antibody therapies. For more information, visit ScholarRock.com and follow @ScholarRock on X and LinkedIn.

Disclaimer

This article contains forward-looking statements regarding Scholar Rock’s business, pipeline, regulatory outlook, and financial position. Actual results may differ materially due to risks and uncertainties, including regulatory decisions, clinical outcomes, manufacturing issues, competition, and other factors detailed in Scholar Rock’s SEC filings. Investors should conduct their own due diligence before making investment decisions. This article is for informational purposes only and does not constitute investment advice.

View Scholar Rock Holding Corp Historical chart here