Company Profile
Scholar Rock
Company Overview
Scholar Rock is a clinical-stage biopharmaceutical company focused on creating a robust pipeline of 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. Our proprietary platform is built upon newly elucidated understanding of the molecular mechanisms of growth factor activation, which has led to the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level.
Our lead product candidate, SRK-015, is a highly specific inhibitor of myostatin activation and is currently being evaluated in a Phase 2 proof-of-concept trial for the treatment of patients with Type 2 and Type 3 Spinal Muscular Atrophy (SMA). SRK-015 has received Orphan Drug Designation in both the US and EU.
We are also advancing SRK-181, a highly specific inhibitor of TGF?1 activation, into clinical development with plans to initiate a Phase 1 trial in patients with solid tumors in mid-2020. We believe SRK-181 has the potential to treat tumors resistant to checkpoint blockade therapy, such as anti-PD(L)1 antibodies.