Scholar Rock Announces FDA Clearance of IND Application to Initiate Phase 2 Proof-of-Concept Trial with Apitegromab to Treat Obesity

Scholar Rock (NASDAQ: SRRK) (the Company), 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 announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its Phase 2 proof-of-concept trial of apitegromab to treat obesity in patients taking a GLP-1 receptor agonist (GLP-1 RA).

The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective myostatin inhibitor, to safely preserve lean muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Trial initiation is on track for mid-2024, and data from the apitegromab Phase 2 trial are expected in mid-2025. In parallel, Scholar Rock is developing SRK-439, a novel investigational selective myostatin inhibitor, optimized for the treatment of obesity. The Company plans to file an IND for SRK-439 in 2025.

“The FDA’s acceptance of our IND application to study apitegromab in obesity allows us to assess the effect of our highly selective myostatin inhibitor on preserving lean muscle mass, and safety and tolerability of our approach when combined with a GLP-1 RA. The IND builds on our encouraging apitegromab clinical and safety data to date and allows us to inform the development of our cardiometabolic program with SRK-439, a novel preclinical selective myostatin inhibitor optimized for development in cardiometabolic disorders,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “Maintaining lean mass during weight loss is important to overall metabolic health, and we look forward to initiating the Phase 2 proof-of-concept trial in apitegromab to validate our differentiated approach of selectively targeting only the pro- and latent forms of myostatin to retain muscle mass.”

About SRK-439

SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass and enhancing fat mass loss. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency.