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.
June 11, 2021
Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 Spinal Muscular Atrophy (SMA) at the 2021 Virtual SMA Research & Clinical Care Meeting
June 7, 2021
Scholar Rock to Present Apitegromab Clinical Data from the TOPAZ Phase 2 Trial in Patients with Type 2 and 3 Spinal Muscular Atrophy at the 2021 Virtual SMA Research & Clinical Care Meeting
May 27, 2021
Scholar Rock to Present at Upcoming Healthcare Conferences in June