Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced investigational product candidate, apitegromab (SRK-015), a selective inhibitor of the activation of the growth factor myostatin in skeletal muscle, is in development for the treatment of spinal muscular atrophy, or SMA. Apitegromab was evaluated in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA and we shared positive 12-month top-line data demonstrating apitegromab’s therapeutic potential in April 2021. We plan to initiate the Phase 3 registrational trial by end of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a selective inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies.
Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
October 3, 2021
Scholar Rock to Present Apitegromab TOPAZ Phase 2 Pharmacologic Data at the 2021 World Congress of Neurology
September 30, 2021
Scholar Rock Presents Exploratory Responder Analysis on Efficacy Data from the Apitegromab TOPAZ Phase 2 Trial at the Child Neurology Society Annual Meeting
September 28, 2021
Scholar Rock Announces Issuance of U.S. Patent Protecting SRK-181, an Inhibitor of TGFβ1 Activation