Scholar Rock Completes Phase 2 Apitegromab Trial Enrollment for Obesity

Scholar Rock, a late-stage biopharmaceutical firm listed on NASDAQ under the ticker SRRK, has successfully completed participant enrollment for its Phase 2 EMBRAZE trial. The trial aims to demonstrate the proof-of-concept for apitegromab, a selective myostatin inhibitor antibody, which is designed to maintain lean muscle mass in patients undergoing GLP-1 receptor agonist therapy for obesity. The results from this study are anticipated in the second quarter of 2025. These findings will contribute to the development of SRK-439, another investigational selective myostatin inhibitor, aimed at treating cardiometabolic disorders, including obesity.

The EMBRAZE trial is a randomized, double-blind, placebo-controlled study assessing the effectiveness, safety, and pharmacokinetics of apitegromab in adults aged 18-65 who are overweight or obese but do not have diabetes. Participants in the trial are also receiving GLP-1 receptor agonist treatments. The primary endpoint of the study is to measure the change in lean muscle mass using DEXA scans over a 24-week period.

Chief Medical Officer Jing Marantz, M.D., Ph.D., emphasized the importance of the trial, stating, "The rapid enrollment in the EMBRAZE trial highlights the urgent need to preserve lean muscle mass in patients experiencing significant weight loss on GLP-1 therapies." Marantz noted that preserving muscle could enhance the quality of weight loss and reduce the weight regain often observed after discontinuing GLP-1 therapy.

Additionally, Scholar Rock announced that it would present new preclinical data on SRK-439 during The Obesity Society’s Annual Meeting at ObesityWeek in San Antonio, Texas, from November 3-6. Earlier preclinical studies demonstrated that SRK-439 preserved lean mass and facilitated fat mass loss when used alongside a GLP-1 receptor agonist in diet-induced obesity mice. These studies also suggested that SRK-439 could help maintain lean mass and reduce fat regain after stopping GLP-1 therapy. Scholar Rock aims to file an Investigational New Drug (IND) application for SRK-439 in 2025.

Apitegromab is also being explored as a potential treatment for spinal muscular atrophy (SMA), marking it as the first muscle-targeted therapy to show clinical proof-of-concept in this condition. Scholar Rock plans to release top-line data from the Phase 3 SAPPHIRE trial in the fourth quarter of 2024. This study evaluates apitegromab's safety and efficacy in nonambulatory patients with Types 2 and 3 SMA who are on SMN-targeted therapies. Successful results and subsequent FDA approval could lead to a commercial launch in 2025.

Apitegromab is a fully human monoclonal antibody that selectively inhibits the activation of myostatin, a protein involved in muscle growth regulation. Scholar Rock believes that targeting myostatin could lead to significant improvements in motor function for SMA patients. The U.S. Food and Drug Administration (FDA) has granted various designations to apitegromab, including Fast Track, Orphan Drug, and Rare Pediatric Disease, while the European Medicines Agency (EMA) has given it Priority Medicines (PRIME) and Orphan Medicinal Product designations.

Scholar Rock continues to focus on developing life-changing therapies for serious diseases with high unmet needs. With its proprietary platform, the company has developed a pipeline of innovative treatments targeting protein growth factors, aiming to transform the standard of care in neuromuscular disease, cardiometabolic disorders, and other conditions.

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