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 initiated our SAPPHIRE Phase 3 registrational trial in patients with later-onset Type 2 and Type 3 SMA in November 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 iron disorders.
May 16, 2022
Scholar Rock Reports First Quarter 2022 Financial Results and Updates Strategic Priorities
April 21, 2022
Scholar Rock Presents Data Analysis of Multiple Efficacy Endpoints from the Apitegromab TOPAZ Phase 2 Trial at the 2022 European Paediatric Neurology Society Congress
March 22, 2022
Scholar Rock Presents Data Analysis of Multiple Efficacy Endpoints from the Apitegromab TOPAZ Phase 2 Trial at the American Academy of Neurology 2022 Annual Meeting