Beloranib Being Tested in Rare Form of Obesity

James Radke

Zafgen, Inc. announced they have completed enrollment of a Phase 2a clinical trial testing beloranib for the treatment of hypothalamic injury-associated obesity (HIAO). The study will evaluate the efficacy and safety of beloranib in HIAO patients over four weeks of randomized treatment in 14 patients with HIAO, followed by an optional four-week open-label extension. 

More specifically,  the ZAF-221 trial is a randomized, double-blind, placebo controlled, trial in which patients will receive twice-weekly subcutaneous injections of 1.8 mg beloranib or placebo.  The primary outcome measure is change in body weight from baseline to the end of the randomized dosing period of four weeks. Secondary outcomes include changes in the patient's lipid profile, hs-CRP (a marker of systemic inflammation), sense of hunger, and quality of life. The trial patients are enrolled at 1 of  4 trial centers,  2 in the United States and 2 in Australia. The study is expected to be completed by the fourth quarter of this year. For more information about this trial, please visit

Hypothalamic Injury-Associated Obesity (HIAO)

HIAO is a rare condition.  The incidence is estimated to be 0.13 to 0.17 per 100,000 per year, or approximately 400 to 500 new cases per year in the United States. HIAO may develop following the removal of a rare benign tumor (craniopharyngioma). In about half the cases in which these tumors are removed, hypothalamic dysfunction can lead to hyperphagia and obesity. HIAO may also develop from less common types of hypothalamic injury such as a strokes, brain trauma, or radiation therapy to the brain.

Dennis Kim, M.D., Chief Medical Officer of Zafgen said:

"Currently, there is no marketed treatment available for hypothalamic injury-associated obesity. Patients often experience uncontrollable hunger, much like patients with Prader-Willi syndrome, which can result in hyperphagia and significant obesity."
"We have seen the therapeutic benefits of beloranib demonstrated in five clinical trials for multiple obesity-related conditions to date, and look forward to learning more from this study regarding beloranib's prospects as a potential treatment for HIAO patients."


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.

 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.

Image of craniopharyngioma courtesy wikimedia commons.

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