Early data show beloranib improved hunger-related behaviors in Prader-Willi syndrome

An investigational selective inhibitor of methionine aminopeptidase 2, beloranib, has demonstrated significant reductions in body fat, body weight, hunger-related behaviors, and body composition in patients with Prader-Willi syndrome in a phase 2a study, according to a press release.

“The medication had positive effects on body composition in adults with Prader-Willi syndrome by increasing muscle mass and decreasing fat mass, but it also allowed them to eat 50% more calories without gaining weight, which is better than anything else we have for patients with PWS, including growth hormone,” Jennifer Miller, MD, associate professor of pediatric endocrinology at the University of Florida, told Endocrine Today.

Jennifer Miller

Miller and colleagues conducted a randomized, double blind, placebo-controlled trial to assess the safety and efficacy of beloranib (Zafgen Inc.) 1.8-mg twice-weekly subcutaneous injections compared with placebo for 4 weeks, followed by a 4-week open label extension.

The trial included 17 patients with PWS (11 women; 6 men) with a mean BMI of 31.4. The investigational agent reduced body fat content by 8.6% vs. placebo in 4 weeks. It appeared to be well-tolerated and safe, according to the press release. Complete data are not available at this time.

Disclosure: Miller reports no relevant financial disclosures.

Jennifer Miller, MD, can be reached at the University of Florida, Department of Pediatrics, 1600 SW Archer Rd., Gainesville, FL 32607; email: millejl@peds.ufl.edu.