US-based biopharmaceutical firm Zafgen has completed patient enrolment in its Phase IIa clinical trial (ZAF-221) of Beloranib to treat hypothalamic injury-associated obesity (HIAO).
The trial is designed to assess the efficacy and safety of beloranib in HIAO patients over four weeks of randomised treatment, followed by an optional four-week open-label extension.
HIAO is caused by damage incurred during removal of a central nervous system (CNS) tumour called Craniopharyngioma, a rare form of benign brain tumour that occurs most commonly during childhood and infiltrates near the optic nerve, pituitary gland and hypothalamus.
This disease can also be caused as a result from less common types of hypothalamic injury.
A total of 14 obese patients with radiographically confirmed hypothalamic damage have been enrolled at four trial centres, two each in the US and Australia.
Zafgen chief executive officer Thomas Hughes said: "The completion of enrollment for the ZAF-221 trial represents an important step forward for both Zafgen and beloranib.
"ZAF-221 will provide us with a first look in patients with HIAO to determine if beloranib has the potential to be a significant new treatment for patients afflicted with this life altering form of obesity.
"Zafgen expects to complete this study in the fourth quarter of this year."
The randomised, double-blind, placebo controlled Phase 2a trial includes twice-weekly subcutaneous injections of 1.8mg Beloranib or placebo in 14 obese patients with radiographically confirmed hypothalamic damage.
The trial’s primary outcome measure is change in body weight from baseline to the end of the randomised dosing period of four weeks, while secondary outcomes include changes in the patient’s lipid profile, hs-CRP (a marker of systemic inflammation), sense of hunger, and quality of life.
Zafgen chief medical officer Dennis Kim 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."