Rhythm Starts Phase 3 Clinical Trial of Setmelanotide

Rhythm announced the initiation of a Phase 3 clinical trial to evaluate the safety and efficacy of setmelanotide, the company's melanocortin-4 receptor (MC4R) agonist, for the treatment of pro-opiomelanocortin (POMC) deficiency obesity, a genetic disorder associated with severe, early-onset obesity and unrelenting, abnormally increased appetite (hyperphagia).

Rhythm is developing setmelanotide 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.

This Phase 3 registration trial is an open-label, single-arm, multinational trial to evaluate the safety and efficacy of setmelanotide in patients with POMC deficiency obesity, with setmelanotide administered once daily by subcutaneous injection for 12 months.

"In POMC deficiency obesity, setmelanotide has the potential to restore lost function in the MC4 pathway to treat the life-threatening obesity and unrelenting hunger that afflicts people with this genetic disorder," Keith Gottesdiener, CEO of Rhythm said. "We are working closely with the FDA under the Breakthrough Therapy designation and with investigators to advance setmelanotide clinical development with urgency."

POMC deficiency obesity is a life-threatening orphan disease that affects between 100 and 500 patients in the U.S. Patients with POMC deficiency have unrelenting hyperphagia that begins in infancy, and they develop severe, early-onset obesity. POMC deficiency obesity results from two different homozygous genetic defects, both upstream of the MC4 receptor.

Setmelanotide is a C4R 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. The critical role of the MC4 pathway in weight regulation was validated with the discovery that single genetic defects along this pathway result in early-onset and severe obesity.