Business
Scholar Rock Reports Full Year 2022 Financial Results and Highlights Business Progress
- Enrollment of pivotal Phase 3 SAPPHIRE trial evaluating apitegromab on track for completion in 2023, and 36-month extension data from Phase 2 TOPAZ trial
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, and 36-month extension data from Phase 2 TOPAZ trial expected in mid-2023\n\n- Phase 1 DRAGON trial of SRK-181 continues to advance with biomarker and clinical data updates planned in 2023\n\n- Company expanded commercial experience with recent appointment of Chief Commercial Officer\n\n- Ended 2022 with approximately $315 million in cash, cash equivalents, and marketable securities, which is expected to fund operations into 2025\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 full year ended December 31, 2022.\n\n“2023 is off to a great start as we continue to build upon our momentum from last year. Importantly, we are on track to complete the enrollment of our pivotal SAPPHIRE trial of apitegromab and are looking forward to providing 36-month extension data from the Phase 2 TOPAZ trial of apitegromab in the coming months. Additionally, we look forward to sharing biomarker and clinical updates throughout the year from the SRK-181 Phase 1 DRAGON trial in patients with relapsed and/or refractory cancers resistant to checkpoint inhibitor therapy,” said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock.\n\nCompany Highlights and Upcoming Milestones\n\nApitegromab is a selective inhibitor of latent myostatin activation being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).\n\n\nContinue to progress and complete enrollment of Phase 3 SAPPHIRE clinical trial. The last patient is expected to be enrolled in SAPPHIRE in 2023, with the top-line data readout expected in 2024. If successful, and if approved, the company expects to initiate a commercial product launch in 2025. SAPPHIRE is a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating apitegromab in patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency ...