Zafgen Quietly Making Progress in Severe Obesity

Zafgen — a young biotech company “dedicated to severe obesity treatment” — quietly continues to make progress in efficacy studies of beloranib. This week, the company announced positive results of a third phase II study in a third distinct population of patients severely affected by obesity.

This study involved patients with obesity resulting from injuries to the hypothalamus, a part of the brain that plays a key role in regulating body weight. Hypothalamic injury-associated obesity (HIAO) most often results from surgery to remove a brain tumor. A very small, but severely affected segment of people with obesity — about 500 each year — makes this an orphan drug indication. Though not many people are affected, the impact is quite severe.

In a randomized, double-blind, placebo-controlled study of just 14 patients with HIAO, patients who received the drug lost 3.4 kg on average after 4 weeks of treatment, significantly more than 0.3 kg lost by people receiving the placebo. Beloranib and the placebo were given twice weekly as an injection.

Beloranib is thought to work by blocking an enzyme called methionine aminopetidase 2 (MetAP2), leading to less synthesis of fat and more metabolism of it.

Earlier trials targeted other small populations of people severely affected by obesity: Prader-Willi Syndrome and severe obesity in people with an average BMI of 38. Zafgen has now reported positive results in all three of populations they’ve studied that are typically resistant to treatment.

Some caveats are worth noting. These are all early-stage (phase II) trials and the results have not yet been fully published in peer-reviewed journals. Results have a way of looking different under the harsh glare of peer review and in phase III studies with larger populations.

Regardless, the innovative strategy of pursuing indications for people severely affected by obesity is impressive. It takes us away from some of the petty debates about the merits of treating obesity.

Click here to read more from Fierce Biotech and here to read a publication of one of their phase II studies.

Injection, photograph © Markus Grossalber / flickr

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