Zafgen Shares Surge On Positive Phase 3 Trial

Shares of Zafgen Inc ZFGN gained more than 14 percent early Monday morning after the company announced positive data from a Phase 3 trial evaluating the safety and efficacy of beloranib.

The study evaluated the safety and efficacy of beloranib, a MetAP2 inhibitor, in patients with Prader-Wili syndrome (PWS) over six months. The data showed beloranib was associated with improvement in total cholesterol, LDL cholesterol and other cardio-metabolic risk factors, and a reduction of fat mass when compared to a placebo.

Beloranib is also the first investigational drug to demonstrate a positive impact on reducing both body weight and hyperphagia-related behaviors.

"These data further support the efficacy profile of beloranib in PWS, and advance our understanding of the potential of our MetAP2 inhibitor platform to impact metabolic disorders," stated Thomas Hughes, Ph.D., Chief Executive Officer of Zafgen. "We believe these data also provide greater perspective on the benefit/risk relationship of beloranib in this high-risk patient population and we look forward to discussing these results with the FDA."

"Prader-Willi syndrome is a life-limiting and life-threatening condition with no available treatment options that significantly impact affected individuals and their families," said Merlin G. Butler, M.D., Ph.D., FFACMG, Professor of Psychiatry, Behavioral Sciences and Pediatrics, Director, Division of Research and Genetics, Departments of Psychiatry & Behavioral Sciences and Pediatrics at the University of Kansas Medical Center. "In addition to the reduction in body weight and decrease in excessive eating behaviors previously reported from this study, the data presented today demonstrate important reductions in cardiometabolic risk factors and further support a strong rationale for continued evaluation of beloranib as a potential treatment for PWS."