Business
Scholar Rock Reports First Quarter 2020 Financial Results and Highlights Business Progress
- Progressing TOPAZ Phase 2 clinical trial of SRK-015 in patients with Spinal Muscular Atrophy; interim efficacy and safety results delayed by approximately
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Progressing TOPAZ Phase 2 clinical trial of SRK-015 in patients with Spinal Muscular Atrophy; interim efficacy and safety results delayed by approximately one quarter to 4Q20 due to impact of COVID-19 pandemic on clinical trial sites\n\n\n- Commenced dosing of patients with solid tumors that exhibit primary resistance to anti-PD(L)1 therapy in the DRAGON Phase 1 clinical trial of SRK-181; update on dose escalation expected in 4Q20\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 first quarter ended March 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs.\n\n\n“We are humbled by the tenacity and commitment of the patients and clinicians with whom we have the privilege to work as we continue to adjust to the challenges of the COVID-19 pandemic,” said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. “It is with a primary focus on the safety and well-being of our patients, clinical investigators and employees, that we are committed to continuing the execution of the SRK-015 TOPAZ Phase 2 and the SRK-181 DRAGON Phase 1 trials. Though we may continue to experience uncertainty during these unprecedented times, Scholar Rock is planning for additional updates on both programs later this year.”\n\n\nCompany Updates and Upcoming Milestones\n\n\nSRK-015 Program for Spinal Muscular Atrophy (SMA):\n\n\n\nAnticipate Interim Efficacy and Safety Data from the TOPAZ Phase 2 Trial in the Fourth Quarter of 2020. COVID-19-related restrictions have impacted patient access to clinical trial sites, which have led to missed or delayed doses or assessments (including efficacy) in some patients and has affected Scholar Rock’s ability to monitor trial data collected by sites. As a result, Scholar Rock now plans to report interim efficacy, safety, pharmacokinetic (PK), and pharmacodynamic (PD) results in the fourth quarter of 2020 for patients across the three cohorts who have progressed through at least six months of the treatment period. This is an approximately one quarter delay compared to the previous guidance of interim 6-month results in mid-2020.\n\nAs of May 1, 2020, the majority of patie...