Scholar Rock to Advance Antimyostatin Program to Develop Novel Therapies for Cardiometabolic Disorders

Scholar 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 announced it plans to advance SRK-439, a novel investigational myostatin inhibitor for the treatment of obesity towards an investigational new drug application (IND) submission in 2025. As part of the Company’s strategy to advance the development of SRK-439, it plans to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in 2024, subject to IND acceptance. Data from the clinical trial are expected in mid-2025 and will be used to inform further clinical development of SRK-439.

“As a long-standing leader in targeting myostatin with a highly selective antibody platform, we are excited to advance our cardiometabolic program. We have discovered multiple, highly selective myostatin inhibitors and see potential with SRK-439 to retain lean muscle mass, a concern that is commonly associated with weight loss strategies, including treatments currently on the market for obesity, a common, serious and costly public health issue affecting adults and children globally. As apitegromab has shown to date in spinal muscular atrophy (SMA), muscle-targeted therapies that are well tolerated have the potential to represent a new class of treatments across a wide range of diseases,” said Jay Backstrom, M.D., M.P.H., President and Chief Executive Officer of Scholar Rock. “While we continue to expand the development of our pipeline, our top priority remains advancing the apitegromab program in SMA. We recently completed full enrollment of the pivotal Phase 3 SAPPHIRE trial of apitegromab in SMA, for which we expect to read out topline data in Q4 2024.”

Cardiometabolic program

Muscle plays a key role in metabolic functions and energy homeostasis. Leveraging proven expertise in antimyostatin and its effect on increasing muscle mass, the Company has been developing myostatin-selective inhibitors to address cardiometabolic disorders, including obesity. Scholar Rock’s platform has generated multiple antibody candidates, including apitegromab, that selectively target pro- and latent forms of myostatin. The Company believes the selectivity of these antibody candidates enables a favorable risk-benefit profile for patients with cardiometabolic disorders.