Business
Scholar Rock Reports Full Year 2021 Financial Results and Highlights Business Progress
- Progressing Phase 3 SAPPHIRE clinical trial evaluating apitegromab in patients with non-ambulatory Type 2 and 3 Spinal Muscular Atrophy (SMA) - Two-year
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Progressing Phase 3 SAPPHIRE clinical trial evaluating apitegromab in patients with non-ambulatory Type 2 and 3 Spinal Muscular Atrophy (SMA)\n\n- Two-year data from open label extension of TOPAZ Phase 2 trial in SMA expected by mid-2022\n\n- Data from DRAGON Part B proof-of-concept trial evaluating SRK-181 to overcome checkpoint inhibitor resistance in cancer patients anticipated in 2022\n\n- Ended 2021 with approximately $253 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 reported financial results for the full year ended December 31, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs.\n\n“We are excited by the momentum and engagement around our ongoing Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy, as well as our DRAGON Phase 1 trial of SRK-181 in solid tumors. These programs have significant potential to address unmet patient need in a differentiated way, as well as demonstrate the tremendous potential of Scholar Rock’s platform, which focuses on inhibiting the activation of latent growth factors,” said Nagesh Mahanthappa, Interim Chief Executive Officer of Scholar Rock. “While we progress these clinical programs, we also continue to build upon our world-leading expertise in selectively targeting the activation mechanisms of the TGFβ-superfamily growth factors and advance our preclinical pipeline focused on diseases such as fibrosis and regulation of iron metabolism. We look forward to providing more details about these programs at scientific meetings and through publications in the coming months.”\n\nCompany Updates and Upcoming Milestones\n\nApitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).\n\n\nConducting SAPPHIRE Phase 3 Clinical Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 SMA. The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam. The study design plans for approximately...