Scholar Rock
Scholar Rock is a biopharmaceutical company headquartered in Cambridge, MA, focused on developing innovative treatments for neuromuscular disorders, fibrosis, and oncology by targeting latent growth factors.
Apitegromab Development
Scholar Rock is developing apitegromab, a selective inhibitor of the activation of latent myostatin. This innovative treatment aims to improve motor function in patients with spinal muscular atrophy (SMA). Apitegromab targets the precursor form of myostatin to potentially enhance muscle mass and strength, and is currently being investigated in the SAPPHIRE Phase 3 clinical trial for efficacy and safety in non-ambulatory individuals with Type 2 and Type 3 SMA.
SRK-181 and Oncology Research
Scholar Rock is actively researching SRK-181, a selective inhibitor of latent TGFβ1 activation. This drug is being tested in the DRAGON Phase 1 clinical trial. SRK-181 is aimed at patients with locally advanced or metastatic solid tumors. The goal is to target the latent form of TGFβ1 to potentially overcome resistance to existing cancer therapies and improve patient outcomes.
Proprietary Platform for Drug Development
Scholar Rock utilizes a proprietary platform to develop drugs that selectively target the precursor forms of growth factors. This approach aims to minimize toxicity and improve therapeutic efficacy. By focusing on latent growth factors, Scholar Rock is able to innovate treatments that can more precisely intervene in disease processes, offering potential benefits in areas like neuromuscular disorders, fibrosis, and oncology.
Company Headquarters and Mission
Scholar Rock is headquartered in Cambridge, MA. The company's mission is to transform patient lives through the discovery and development of innovative medicines. By focusing on the latent forms of growth factors, Scholar Rock seeks to create therapies that can substantially change treatment paradigms across various serious illnesses, emphasizing neuromuscular disorders, fibrosis, and oncology.
Clinical Trials and Research Focus
Scholar Rock is involved in multiple clinical trials, including the SAPPHIRE Phase 3 trial for apitegromab in SMA and the DRAGON Phase 1 trial for SRK-181 in solid tumors. The company is focused on developing treatments that target latent growth factors, which play a role in disease processes. Their efforts are aimed at addressing unmet medical needs in neuromuscular disorders, oncology, and fibrosis.