Rhythm, a Boston-based biotechnology company working on peptide therapeutics for metabolic diseases, said Tuesday that it is starting clinical testing of a drug designed to treat obesity in people with a genetic deficiency.
The company’s drug candidate is currently dubbed RM-493, and this new study expands on Rhythm’s ongoing Phase 2 clinical program for RM-493 for the treatment of obesity.
RM-493 is a melanocortin 4 receptor (MC4R) agonist, and the hope is that it can treat obesity in individuals with a genetic deficiency in the MC4R pathway. The MC4 receptor mediates a key pathway in humans that regulates energy homeostasis and food intake, and MC4R deficiency may undermine the effects of diet and exercise for weight loss, limiting treatment options for patients with the deficiency, Rhythm’s press release said.
The company’s release included a statement from Sadaf Farooqi, a professor of metabolism and medicine at the University of Cambridge. Farooqi is working with Rhythm on the project.
‘‘Genes with loss of function in the MC4 pathway result in severe obesity, with a prevalence that may exceed one million people in the US,’’ Farooqi said. ‘‘This RM-493 clinical trial is a new approach that targets the root cause of obesity in the MC4 heterozygote population.’’
In a separate statement, Rhythm chief executive Keith Gottesdiener, MD, added: ‘‘RM-493 has demonstrated impressive efficacy on weight loss in both preclinical studies and early clinical trials in subjects with normal, fully functioning MC4R genes. This new clinical study is a personalized medicine approach to the treatment of obesity in people with a poorly functioning MC4R gene and has the potential to restore function in this pathway and improve weight regulation.’’
The company’s release noted that more than 78 million US adults and 12.5 million US children and adolescents are obese.
Rhythm investors include MPM Capital, New Enterprise Associates, Third Rock Ventures, Ipsen, and Pfizer Ventures.