Scholar Rock Holding Corp

I. Overview We are a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy ( SMA ) and additional rare, severe and debilitating neuromuscular diseases. As a leader in the biology of the transforming growth factor beta ( TGF ) superfamily, our novel understanding of the molecular mechanisms of growth factor activation enabled the development of a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. Based on our innovative, proprietary, and scalable technology platform, we are building a world-leading anti-myostatin pipeline. During 2025, we made significant progress in advancing our product candidates. We believe 2026 could be a transformational year for Scholar Rock as we anticipate the potential to become a commercial-stage biotech company. Our lead pipeline product candidates include apitegromab, a subcutaneous formulation of apitegromab, and SRK-439. Apitegromab is a novel, investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Myostatin is a catabolic agent that functions as a negative regulator of muscle mass, therefore inhibition of myostatin results in increased muscle mass and strength. Apitegromab is in development for the treatment of people with SMA and for the treatment of people with facioscapulohumeral muscular dystrophy ( FSHD ).