Business
Scholar Rock Reports Second Quarter 2024 Financial Results and Highlights Business Progress
- Remains on track to report topline data from pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy (SMA) in 4Q 2024 - New data from Phase
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n- Remains on track to report topline data from pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy (SMA) in 4Q 2024\n\n\n- New data from Phase 2 TOPAZ extension study in patients with nonambulatory SMA showed sustained clinical benefit over 48 months, a continued favorable safety profile with no new safety findings; patient retention rate of over 90%\n\n\n- Phase 2 EMBRAZE proof-of-concept trial enrolling ahead of schedule, topline data expected in 2Q 2025\n\n\n- Presented new SRK-439 preclinical data at American Diabetes Association’s 84th Scientific Sessions (ADA) supporting the potential to contribute to a favorable body composition; increased lean mass and reduced fat mass regain following withdrawal from GLP-1 receptor agonist treatment\n\n\n- Management to host update call today at 8:15 a.m. ET\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today reported financial results and corporate updates for the second quarter ended June 30, 2024.\n\n\n“Scholar Rock continues to execute across our portfolio of highly selective myostatin inhibition programs, further cementing our position as the global leader in harnessing the life-changing potential of TGF-beta superfamily biology,” said Jay Backstrom, M.D., MPH, President & Chief Executive Officer of Scholar Rock. “With the only muscle-targeted program to demonstrate clinical proof-of-concept in SMA, our confidence in our lead program apitegromab continues to be supported by the clinical data generated over the past four years. At 48 months, over 90% of TOPAZ patients with nonambulatory SMA remained on apitegromab treatment on top of SMN therapy and we continued to observe sustained clinical benefit. We look forward to reporting topline data from the Phase 3 SAPPHIRE trial of apitegromab in SMA in the fourth quarter of this year.”\n\n\nDr. Backstrom continued, “In addition, we are pleased with the progress of our cardiometabolic program. The enrollment of the Phase 2 proof-of-concept EMBRAZE study evaluating apitegromab in obesity has been advancing ahead of schedule and as a result, we are updating our guidance fo...