Business
Scholar Rock Reports Second Quarter 2023 Financial Results and Highlights Business Progress
- Recently presented 36-month apitegromab extension data from Phase 2 TOPAZ trial demonstrate long-term substantial and sustained improvement of motor
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Recently presented 36-month apitegromab extension data from Phase 2 TOPAZ trial demonstrate long-term substantial and sustained improvement of motor function and further strengthen conviction in pivotal Phase 3 SAPPHIRE trial\n\n\n- Pivotal Phase 3 SAPPHIRE trial evaluating apitegromab on track to complete enrollment in Q3 2023 with top-line data expected in 2H 2024\n\n\n- Clinical and biomarker update from the Part B portion of the Phase 1 DRAGON trial of SRK-181 in solid tumors anticipated in 2H 2023\n\n\n- Approximately $249 million in cash, cash equivalents, and marketable securities as of June 30, 2023, expected to fund operations into 2025\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results and corporate updates for the second quarter ended June 30, 2023.\n\n\n“We continue to advance our apitegromab program, notably with the recent presentation of 36-month data from the Phase 2 TOPAZ trial which supports apitegromab’s therapeutic potential to meaningfully improve motor function for patients with SMA,” said Jay Backstrom, M.D., M.P.H., President and Chief Executive Officer of Scholar Rock. “The continued success of TOPAZ increases our conviction in SAPPHIRE and we look forward to completing enrollment this quarter and sharing a topline data readout in 2024.”\n\n\nRecent Company Highlights and Upcoming Milestones\n\n\nSpinal Muscular Atrophy (SMA) Program\n\n\nApitegromab is an investigational fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA.\n\n\n\nPresented 36-month extension data from Phase 2 TOPAZ trial at Cure SMA Research & Clinical Care Meeting in June. The company shared new data evaluating outcomes after 36 months of treatment with apitegromab, which showed substantial and sustained improvement in motor function, as well as improvements in patient-reported outcome measures in patients with nonambulatory Types 2 and 3 SMA receiving survival motor neuron (SMN) therapy. The company also hosted a virtual investor day event in...