Rare Disease Report

Obesity Drug Being Tested in Prader-Willi Syndrome

OCTOBER 02, 2014
James Radke

One week after announcing the initiation of their phase 2 obesity study in patients with hypothalamic injury-associated obesity, Zafgen, Inc has announced the initiation of a phase 3 clinical trial to test the safety and efficacy of beloranib in the treatment of another rare obesity condition - Prader-Willi syndrome.

Prader-Willi syndrome is a rare disease that begins at birth and results in a many physical, mental, and behavioral problems. A key symptom of Prader-Willi is hyperphagia (unrelenting hunger) that leads to obesity.

Prader-Willi syndrome is due to mutations present in chromosome 15 that impairs hypothalamic function. The resulting obesity in turn leads to a plethora of problems as the children develop.  It is hopeful that if the hunger in these children can be better controlled, some of the other problems these children develop will be easier to manage.

Zafgen’s beloranib is a potent inhibitor of MetAP2, an enzyme that modulates the activity of key cellular processes that control metabolism which may reduce hunger while stimulating the use of stored fat as an energy source. The drug is being tested in a number of obesity trials, including obese patients with Prader-Willi syndrome and those with hypothalamic injury.

Trial Design

The "bestPWS" trial (Beloranib Efficacy Safety and Tolerability in PWS), is a randomized, double-blind, placebo-controlled trial in obese adolescents (12 years of age and older) and adults with Prader-Willi syndrome to evaluate food-related behaviors, total body fat mass, and safety of beloranib. The trial is expected to enroll 84 patients at 14 sites in the United States. Patients will be randomized to receive placebo, 1.8mg or 2.4mg of twice weekly subcutaneous injections of beloranib during the randomized treatment period of s6 months. Patients who complete study treatment will have the option to enter a 6  month open label extension study.

The two planned primary efficacy endpoints are 1) changes in hyperphagia-related behaviors, 2) total body fat mass from baseline to the end of randomized treatment.

Dr. Thomas Hughes, Ph.D., Chief Executive Officer of Zafgen said:

"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.”
”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."

Dr. Jennifer Miller, Pediatric Endocrinologist and Associate Professor of Medicine at University of Florida Health added:

"PWS is a devastating condition that leads to life-threatening obesity and affects patients and caretakers alike."
 "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."

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

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