Subcutaneous beloranib: Preliminary Phase IIa data

Zafgen reported preliminary data from a double-blind, placebo-controlled, U.S. Phase IIa trial in 17 obese patients with genetically confirmed Prader-Willi syndrome showing that "a trend towards overall improvement" in body weight from baseline to week 4, the primary endpoint, was observed in patients who received twice-weekly subcutaneous beloranib. The company said the trial was not powered for statistical significance.

Additionally, Zafgen said high-dose beloranib reduced body fat content by 8.1% vs. placebo, despite a 50% increase in daily caloric allowance. Patients received placebo or twice-weekly 1.2 or 1.8 mg beloranib for 4 weeks. Zafgen said beloranib improved hunger-related behaviors and treatment appeared to be well tolerated. All patients completed the 4-week trial as well as a 4-week open-label expansion. ...