FDA Approves Scholar Rock's Phase 2 Trial for Obesity Treatment with Apitegromab

Scholar Rock, a biopharmaceutical firm dedicated to developing therapies for spinal muscular atrophy (SMA) and other serious conditions, has received approval from the FDA for its Investigational New Drug (IND) application. This approval is for a Phase 2 clinical trial of apitegromab, a drug designed to treat obesity in individuals who are also receiving GLP-1 receptor agonist (GLP-1 RA) therapy. The trial, which is set to begin in mid-2024, is a randomized, double-blind, placebo-controlled study that will assess the efficacy of apitegromab in preserving lean muscle mass in overweight and obese adults.

The study's objective is to demonstrate the significance of selective myostatin inhibition in maintaining lean muscle mass during weight loss, a key aspect of healthy weight management. Apitegromab is a highly selective inhibitor of myostatin, a protein that plays a crucial role in muscle growth and development. Scholar Rock is also working on SRK-439, another selective myostatin inhibitor that is being developed specifically for obesity treatment, with plans to file an IND for this drug in 2025.

Jay Backstrom, Scholar Rock's President and CEO, emphasized the importance of maintaining lean muscle mass during weight loss for metabolic health and expressed enthusiasm for the upcoming trial. He highlighted the potential of apitegromab to selectively target the pro- and latent forms of myostatin, which could lead to significant improvements in muscle function.

Apitegromab is an innovative, fully human monoclonal antibody that inhibits myostatin activation by binding to its pro- and latent forms in skeletal muscle. It has shown promise in clinical trials for SMA, a condition characterized by loss of motor neurons and muscle weakness. The FDA has granted apitegromab several designations, including Fast Track, Orphan Drug, and Rare Pediatric Disease, for its potential use in treating SMA.

Scholar Rock is renowned for its leadership in TGFβ superfamily biology and its proprietary platform for developing monoclonal antibodies with exceptional selectivity. The company's mission is to create transformative treatments for serious diseases with significant unmet needs, focusing on conditions where protein growth factors are pivotal.