Business
Scholar Rock Reports First Quarter 2023 Financial Results and Highlights Business Progress
- Enrollment of pivotal Phase 3 SAPPHIRE trial evaluating apitegromab on track for completion in 2023 - Upcoming presentations of 36-month apitegromab data
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Enrollment of pivotal Phase 3 SAPPHIRE trial evaluating apitegromab on track for completion in 2023\n\n\n- Upcoming presentations of 36-month apitegromab data from Phase 2 TOPAZ trial at Cure SMA’s Annual Research & Clinical Conference\n\n\n- Continued advancement of Phase 1 DRAGON trial of SRK-181; biomarker and clinical data update planned in the second half of 2023\n\n\n- Approximately $275 million in cash, cash equivalents, and marketable securities as of March 31, 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 first quarter ended March 31, 2023.\n\n\n“We are executing across our business and leveraging Scholar Rock’s differentiated platform, which selectively targets latent forms of growth factors, such as myostatin and TGFβ1, to develop highly selective and potentially transformative therapies for patients who have high unmet medical need,” said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. “We continue to advance our two clinical programs in spinal muscular atrophy and oncology, and we look forward to sharing updates on our progress as we approach several key milestones, including presenting 36-month data on apitegromab from the Phase 2 TOPAZ trial, completing enrollment of our apitegromab Phase 3 SAPPHIRE trial, and providing biomarker and clinical updates from our SRK-181 Phase 1 DRAGON trial.”\n\n\nRecent Company Highlights and Upcoming Milestones\n\n\nApitegromab is an investigational antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin and is being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).\n\n\n\nContinued progress towards completion of enrollment for Phase 3 SAPPHIRE clinical trial. The randomized double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam, is actively enrolling SMA patients across sites in the U.S. and Europe. Enrollment completion is expected in 2023, with the top-line d...