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Prader-Willi syndrome trial

Zafgen begins Phase 3 Prader-Willi syndrome trial

Patients enrolled will be randomized to receive placebo, 1.8mg or 2.4mg of twice weekly subcutaneous injections during the randomized treatment period of six months.
Zafgen has been granted orphan drug status in both the US and the European Union for beloranib for the treatment of PWS.

Zafgen has initiated the first Phase 3 clinical trial in the US States with beloranib in the treatment of Prader-Willi syndrome (PWS). PWS is a rare disease and the most common known genetic cause of life-threatening obesity. It is a complex metabolic syndrome characterized by hyperphagia (unrelenting hunger) and obesity resulting from impaired functioning of the hypothalamus.

"PWS is a devastating condition that leads to life-threatening obesity and affects patients and caretakers alike," said Dr. Jennifer Miller, Pediatric Endocrinologist and Associate Professor of Medicine at University of Florida Health. "This Phase 3 trial is a significant step in meeting the urgent need for novel and effective treatment options that have the potential to positively impact the lives of patients and families living with this disease."

The bestPWS trial (Beloranib Efficacy Safety and Tolerability in PWS), is a randomised, double-blind, placebo-controlled trial in obese adolescents (12 years of age and older) and adults with Prader-Willi syndrome to evaluate food-related behaviours, total body fat mass, and safety of beloranib. The trial is expected to enroll 84 patients at 14 sites across the US.

Patients enrolled will be randomized to receive placebo, 1.8mg or 2.4mg of twice weekly subcutaneous injections during the randomized treatment period of six months. Patients who complete study treatment will have the option to enter a six month open label extension study where all participants will receive beloranib.

The two primary efficacy endpoints are planned to be changes in hyperphagia-related behaviours and/or total body fat mass from baseline to the end of randomized treatment. Key secondary endpoints include changes in lipid parameters and body weight. Additional assessments include quality of life (QoL) impact for patients and caregivers.

"The initiation of the bestPWS Phase 3 trial is a major milestone for Zafgen as we work toward our ultimate goal of improving the lives of patients affected by PWS and bringing beloranib to market," said Dr Thomas Hughes, CEO. "We are committed to the development of beloranib, which has the potential to be the first therapy to help improve hyperphagia and body fat content, for which no treatment options are currently available. We currently expect to report on initial six-month data from this study in the fourth quarter of 2015."

Zafgen has been granted orphan drug status in both the US and the European Union for beloranib for the treatment of PWS.

Beloranib is a novel, first-in-class injectable small molecule therapy with a unique mechanism of action that reduces hunger while stimulating the use of stored fat as an energy source. Beloranib is a potent inhibitor of MetAP2, an enzyme that modulates the activity of key cellular processes that control metabolism. MetAP2 inhibitors work, at least in part, by directing MetAP2 binding to cellular stress mediators, and, thus, reducing the tone of signals that drive lipid synthesis by the liver and fat storage throughout the body. In this manner, MetAP2 inhibition increases metabolism of fat as an energy source. Zafgen holds exclusive worldwide rights (exclusive of South Korea) for the development and commercialization of beloranib. Zafgen exclusively licensed beloranib from Chong Kun Dang (CKD) Pharmaceutical Corp. of South Korea.

According to Zafgen's license agreements with CKD Pharma and other licensors, the initiation of the first Phase 3 trial with beloranib will trigger milestone payments of US$6.7 million (primarily to CKD).

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