Business
Scholar Rock Provides Corporate Update and Highlights Priorities for 2021
- TOPAZ Phase 2 trial evaluating apitegromab in spinal muscular atrophy on-track to report top-line results from the 12-month treatment period in 2Q21 -
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- TOPAZ Phase 2 trial evaluating apitegromab in spinal muscular atrophy on-track to report top-line results from the 12-month treatment period in 2Q21\n\n- SRK-181 DRAGON Phase 1 trial enrolling patients with solid tumors in Part A2; initial clinical response and safety data anticipated 2H21\n\n- Ended 2020 with approximately $340 million in cash, cash equivalents, and marketable securities \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 provided a corporate update and highlighted priorities for 2021.\n\n“2020 was a transformative year for Scholar Rock with significant progress made both clinically and operationally with positive interim data from the TOPAZ trial providing initial proof-of-concept of apitegromab’s potential in SMA and showing the potential benefit of inhibiting the latent forms of growth factors,” said Tony Kingsley, President and CEO of Scholar Rock. “In 2021, we will further elucidate the potential of our product candidates through data from our TOPAZ Phase 2 trial in SMA and our DRAGON Phase 1 trial in cancer immunotherapy as well as build upon our scientific platform towards additional indications.”\n\n2021 Expected Milestones: \n\nApitegromab is a highly selective inhibitor of latent myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).\n\n\nTop-line Efficacy and Safety Data from TOPAZ Phase 2 Trial in SMA Anticipated in 2Q21. A total of 58 patients were enrolled across the three cohorts of the TOPAZ clinical trial with one discontinuation to date. Positive six-month interim analysis data were announced in October 2020, demonstrating apitegromab’s potential to improve motor function in patients with Type 2 and Type 3 SMA. The top-line 12-month efficacy and safety data are expected in the second quarter of 2021.\n\n\nAs of January 8, 2021, 56 patients have completed the 12-month study and all 56 have opted into the extension period.\n\n\nIdentification of Second Indication for Apitegromab Planned for 2021. With the demonstration of initial proof-of-concept from the TOPAZ interim analysis, Scholar Rock is actively evaluating multiple other disorders for wh...