CUMC offers Clinical Trial of
Promising, new Obesity Drug

A promising, experimental obesity drug called setmelanotide, is now being tested in a clinical trial at Columbia University Medical Center. The drug, developed by Rhythm, a Boston-based biotech company, targets the protein melanocortin 4 receptor, or MC4R, which plays a key role in appetite and weight regulation in the brain.

This clinical trial, says geneticist Wendy Chung, MD, the Kennedy Family Professor of Pediatrics and Medicine at Columbia, is for people who suffer from one of two different rare, genetic disorders—pro-opiomelanocortin (POMC) deficiency obesity, and leptin receptor (LepR) deficiency obesity—that manifest in unrelenting hunger. “This drug is not for everybody,” said Dr. Chung. “But for the people for whom it has worked, the results have been dramatic and impressive.”

In 2016, Rhythm (which is focused on developing drugs for the treatment of rare genetic disorders that result in life-threatening metabolic disorders) released data from small, Phase 2 trials for both LepR and POMC indicating that setmelanotide was able to meet goals of reducing patients’ hunger and weight. One LepR patient lost 56.4 lbs. over 22 weeks, from a baseline weight of 288 lbs.—a 19.6 per cent decrease. The same patient experienced a substantial reduction in hunger from a baseline score of 9 (on a hunger scale with 10 representing extreme hunger) to a score of between 1 and 2.

(The U.S. Food and Drug Administration has acknowledged the importance of setmelanotide by granting it breakthrough therapy designation enabling an expedited path to approval for use in patients with LepR and POMC.)

For now, most people who have weight problems don’t have the genetic forms of obesity for which the medication is currently being tested. (The drug is also being tested on other genetic obesity syndromes including Prader Willi).  “What we don’t know is if it would work for people who don’t have a defined genetic cause,” said Dr. Chung. “That’s what we don’t know. Over time, once the Rhythm tests it on individuals who have a specific genetic reason for why it should work, I think they’ll expand it to other people with weight problems. It often starts out small and ripples out."

This clinical trial is exciting for multiple reasons. For one thing, today there is a scarcity of effective medications to treat obesity. “We don’t currently have great treatments for people with weight problems,” said Dr. Chung. “The most effective treatment we have right now is surgical. This is one of a few medications that looks promising.”

A short questionnaire and a blood test screens candidates for this trial. If you’re interested in participating, please contact Wendy Chung, MD PhD at wkc15@columbia.edu or 212-851-5313.