Business
Scholar Rock Reports Third Quarter 2020 Financial Results and Highlights Business Progress
- Positive interim analysis data from the TOPAZ Phase 2 trial demonstrated proof-of-concept for apitegromab (SRK-015) in patients with Type 2 and Type 3
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Positive interim analysis data from the TOPAZ Phase 2 trial demonstrated proof-of-concept for apitegromab (SRK-015) in patients with Type 2 and Type 3 Spinal Muscular Atrophy\n\n- Progress update on dose escalation in Part A of the SRK-181 DRAGON Phase 1 trial to be presented at the Society for Immunotherapy of Cancer (SITC) Congress; no dose-limiting toxicities observed as of the data cutoff \n\n- Raised $230 million in gross proceeds through a public offering to advance apitegromab, SRK-181 and preclinical programs\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the third quarter ended September 30, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs.\n\n“We achieved a significant milestone with the TOPAZ interim analysis proof-of-concept results. The data support apitegromab’s potential to improve motor function for patients with Type 2 and Type 3 SMA and importantly, highlight the therapeutic potential of our platform targeting the latent forms of growth factors, which includes SRK-181 in immuno-oncology,” said Tony Kingsley, President and CEO of Scholar Rock. “As we look to the remainder of 2020 and into 2021, I am very excited for the potential of the company as we continue to advance and expand our pipeline across a wide range of serious diseases where growth factors may play a role, including other neuromuscular disorders, cancer, and fibrosis.”\n\n\nCompany Updates and Upcoming Milestones\n\nApitegromab (SRK-015) Program for Spinal Muscular Atrophy (SMA): \n\nApitegromab is a highly selective inhibitor of latent myostatin activation being developed as the potential first muscle-directed therapy for the treatment of SMA.\n\n\nSix-Month Interim Data from TOPAZ Demonstrate Meaningful Potential for Apitegromab to Improve Motor Function for Patients with SMA. In October 2020, Scholar Rock announced positive proof-of-concept data from a pre-planned six-month interim analysis of the TOPAZ Phase 2 trial, which enrolled 58 patients with Type 2 and Type 3 SMA across three parallel cohorts. Key findings included:\n\n\nTreatment with apitegromab led to improvements in the Hammersmith scale sco...