Scholar Rock Holding Corporation disclosed its 2026 strategic priorities, outlining a focused plan to bring its lead candidate apitegromab to children and adults with spinal muscular atrophy (SMA) while expanding the program to younger patients and other rare neuromuscular diseases. The company also announced plans to advance its anti‑myostatin antibody SRK‑439 into Phase 1 and to leverage early cardiometabolic data from the EMBRAZE study.
The company’s launch timeline for apitegromab has been adjusted to 2026 following a Complete Response Letter (CRL) issued by the FDA on September 22, 2025. The CRL highlighted manufacturing compliance issues that require remediation at the Catalent Indiana facility. Scholar Rock has scheduled a meeting with the FDA in early 2026 to discuss the remediation plan and is preparing a BLA resubmission that is expected to be filed later that year.
In addition to the SMA focus, Scholar Rock is expanding its pipeline to include a Phase 2 program for patients under two years old and a Phase 2 program for facioscapulohumeral muscular dystrophy (FSHD). The SRK‑439 anti‑myostatin antibody is in a healthy‑volunteer Phase 1 study that began dosing in Q4 2025, with topline data anticipated in the second half of 2026. The EMBRAZE Phase 2 trial, which evaluated apitegromab in adults with obesity, is expected to provide cardiometabolic insights that could broaden the drug’s therapeutic scope.
Financially, the company reported approximately $365 million in cash as of December 31, 2025, giving it a runway into 2027. CEO David L. Hallal, who took the helm in April 2025, emphasized that the cash position supports the company’s aggressive development and commercialization agenda while it transitions toward a commercial‑stage biopharmaceutical model.
Management expressed confidence that 2026 will be a transformative year, noting that the company is “executing with urgency” and has “strong momentum across our rare, severe neuromuscular disease programs.” The focus on regulatory remediation, pipeline expansion, and robust financial footing positions Scholar Rock to capitalize on the growing market for SMA and other neuromuscular therapies.
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