On September 11, 2026, the U.S. Food and Drug Administration approved ISEMBYLD (apitegromab-mstn) for the treatment of spinal muscular atrophy in adults and children two years of age and older currently receiving SMN2-targeted treatment. The approval was based on Phase 3 SAPPHIRE study results showing a 2.2-point improvement in Hammersmith Functional Motor Scale-Expanded scores compared to placebo. Scholar Rock is launching ISEMBYLD commercially in the U.S., with product available for shipment immediately, and received a Rare Pediatric Disease Priority Review Voucher. The company reported $492 million in cash, cash equivalents, and marketable securities as of June 30, 2026, and intends to monetize the voucher. Scholar Rock Holding Corporation is a global biopharmaceutical company focused on developing treatments for rare neuromuscular diseases.

Scholar Rock filed a prospectus supplement to offer up to $200 million in additional common stock under its at-the-market (ATM) program with Jefferies. This filing replaces and supersedes the prior prospectus supplement dated November 14, 2025, terminating that previous offering. The company has raised approximately $257.7 million in net proceeds from shares sold under the ATM Program to date. Scholar Rock is a biopharmaceutical company focused on discovering and developing therapies for serious diseases.

Scholar Rock Holding Corporation reported a net loss of $109.9 million for the quarter ended June 30, 2026, compared to $110.0 million in the prior year period. The company held cash and marketable securities of $492.1 million as of June 30, 2026, which included $62.8 million in net proceeds from its at-the-market equity program. Apitegromab's Biologics License Application for spinal muscular atrophy remains on track for a potential FDA approval by the September 30, 2026 PDUFA date, with review of a second fill-finish facility progressing and ample commercial supply secured. The company also initiated the Phase 2 FORGE study for facioscapulohumeral muscular dystrophy and continues engagement with the European Medicines Agency regarding its Marketing Authorisation Application. Scholar Rock is a biopharmaceutical company focused on developing treatments for rare neuromuscular diseases.