Scholar Rock Holding Corporation

Scholar Rock Holding Corporation is a clinical-stage biopharmaceutical company dedicated to discovering, developing, and delivering innovative medicines for serious diseases where protein growth factors play a fundamental role. The company's mission is to create new possibilities for patients by translating cutting-edge scientific understanding into life-changing therapies. Headquartered in Cambridge, Massachusetts, Scholar Rock utilizes a proprietary platform to selectively target latent, inactive growth factors, aiming to regulate their activity at the cellular level.

The company's pipeline of investigational biologic candidates focuses on various therapeutic areas, including rare disease therapeutics, neuromuscular diseases, cardiometabolic disorders, immuno-oncology, and fibrotic conditions. Its lead product candidate, apitegromab, is a myostatin-directed biologic in pivotal Phase 3 development for spinal muscular atrophy (SMA). Scholar Rock is also advancing SRK-181, a selective inhibitor of latent TGFβ1, to address resistance to checkpoint inhibitors in solid tumors, and SRK-439, an investigational myostatin antibody for rare neuromuscular diseases, obesity, and cardiometabolic disorders.

In April 2025, David Hallal was appointed CEO, succeeding Jay Backstrom, as part of a leadership transition that also saw the appointment of Vikas Sinha as CFO, Akshay Vaishnaw as President of R&D, and R. Keith Woods as COO. Scholar Rock is strategically transitioning from a clinical-stage to a commercial-stage biopharmaceutical company, with apitegromab for SMA as its flagship asset, which has received priority review for its Biologics License Application (BLA) and is anticipated for launch later in 2025. The company reported positive topline data from the SAPPHIRE clinical trial for apitegromab in October 2024 and has a collaboration with Gilead focused on latent TGFβ-targeting approaches in fibrotic disease.