- Apitegromab Biologics License Application (BLA) resubmission and U.S. launch, following FDA approval, are anticipated in 2026 for the treatment of children and adults with spinal muscular atrophy (SMA)
- FDA has scheduled meeting with Catalent Indiana, LLC (part of Novo Nordisk) in early 2026 to discuss progress of remediation efforts
- Apitegromab Investigational New Drug (IND) application cleared for facioscapulohumeral muscular dystrophy (FSHD); Phase 2 study initiation and patient dosing expected mid-2026
- Subcutaneous apitegromab advancing; Phase 1 study demonstrated favorable bioavailability, with pharmacodynamic profile comparable to IV administration
- SRK-439 Phase 1 trial ongoing in healthy volunteers; topline data expected in H2 2026
- Approximately $365 million1 in cash as of December 31, 2025; cash runway expected to support operations into 2027
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to dramatically improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today provided a corporate update and highlighted its strategic priorities for 2026.
“We believe 2026 will be a transformative year for Scholar Rock. We are executing with urgency, and we have strong momentum across our rare, severe neuromuscular disease programs as we build our foundation to become the next global biotech powerhouse,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “Our foremost objective remains bringing apitegromab to children and adults with SMA as quickly as possible, and we are confident in the steps we are taking to support a BLA resubmission and a U.S. commercial launch, following approval, this year.”
2026 Strategic Priorities
Scholar Rock is focused on three core strategic priorities in 2026 to create sustainable long-term value and maximize the potential of its innovative anti-myostatin platform to serve patients:
- FDA and European Medicines Agency (EMA) regulatory approvals and commercialization of apitegromab for children and adults with SMA.
- Develop apitegromab for patients with SMA (