americanpharmaceuticalreviewMay 18, 2017
Rhythm announced the expansion of the company's Phase 2 clinical trials of setmelanotide to the treatment of Bardet-Biedl syndrome (BBS) obesity. Setmelanotide is a melanocortin-4 receptor (MC4R) agonist in development for the treatment of obesity caused by genetic deficiencies in the MC4 pathway, a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite.
"Bardet-Biedl syndrome affects vision, kidney function, and other organs—but the obsessive appetite without relief, along with the burden of obesity, are often the most important issues for people living with this condition, including many of our patients," Robert Haws, MD, Medical Director of Clinical Research at the Marshfield Clinic Research Foundation and an investigator for the study said. "We are therefore very excited about the setmelanotide study as a potential treatment for this condition."
This initial study is an open-label, Phase 2 clinical trial of setmelanotide for the treatment of BBS obesity. The clinical trial will evaluate the safety and efficacy of setmelanotide administered once daily by subcutaneous injection in patients with BBS obesity.
Bardet-Biedl syndrome (BBS) is a life-threatening, orphan disease with a prevalence of approximately one in one hundred thousand in North America. BBS is a monogenic disorder that causes severe obesity and hyperphagia as well as vision loss, polydactyly, kidney abnormalities, and other signs and symptoms. For these patients, hyperphagia and obesity can have significant health consequences for which there is currently no approved treatment. Recent scientific studies identify deficiencies affecting the MC4 pathway as a potential cause of the obesity and hyperphagia associated with BBS.
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