Business
Scholar Rock Reports Second Quarter 2021 Financial Results and Highlights Business Progress
- Phase 3 trial to evaluate apitegromab in patients with non-ambulatory Type 2 and Type 3 spinal muscular atrophy (SMA) anticipated to initiate by end of
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Phase 3 trial to evaluate apitegromab in patients with non-ambulatory Type 2 and Type 3 spinal muscular atrophy (SMA) anticipated to initiate by end of 2021\n\n- DRAGON Phase 1 trial evaluating SRK-181’s ability to overcome resistance to checkpoint inhibitors continues to progress and on-track to advance to Part B dose expansion mid-year 2021\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 second quarter ended June 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs.\n\n“Scholar Rock has had significant momentum in the first half of the year with further demonstration of apitegromab’s transformative potential to improve motor function for patients with SMA, as well as continued progression of dose escalation in the DRAGON trial evaluating SRK-181’s potential to overcome resistance to checkpoint inhibitors in solid tumors,” said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. “I look forward to working closely with this exceptional team as we continue to advance our research and clinical programs to further elucidate the potential of our scientific platform and help make a difference in the lives of patients suffering from serious diseases.”\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\n\nPositive Top-line Results and Additional Supportive Exploratory Analyses from the TOPAZ Trial were Presented at the Cure SMA Annual Conference. In June 2021, positive top-line data from the TOPAZ Phase 2 trial (NCT03921528) were presented by the lead principal investigator, Thomas Crawford, M.D. of Johns Hopkins Medicine. Treatment with apitegromab in conjunction with nusinersen in patients with Type 2 and 3 SMA led to meaningful motor function improvements of up to 20 points as measured by Hammersmith Functional Motor Scale Expanded (HFMSE). The majority (74%, 23/31) of non-ambulatory patients showed a clinical improvement, as defined by at least a 1-point increase in HFMSE. Further evidence that improvements in...