Rhythm Pharmaceuticals Announces Progress in Clinical Development of Setmelanotide for Treatment of Rare Genetic Disorders of Obesity

BOSTON – Rhythm (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced recent progress in the clinical development of the company’s lead product candidate, setmelanotide, a first-in-class melanocortin-4 receptor (MC4R) agonist.

“We are pleased to announce important progress across our clinical development programs for setmelanotide, as we work to deliver a first-in-class therapy for the treatment of monogenic MC4 pathway defects that result in life-threatening obesity,” said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm. “We continue to work closely with the U.S. Food and Drug Administration to advance our ongoing Phase 3 trial in POMC deficiency obesity, and recently opened enrollment in a second pivotal trial in LEPR deficiency obesity, while also enrolling patients to establish proof-of-concept in Alström Syndrome and POMC epigenetic disorder. We look forward to advancing these efforts in 2018, as we evaluate setmelanotide’s ability to reestablish weight and appetite control in patients with genetic, MC4 pathway deficiencies.”

Following recent discussions with the U.S. Food & Drug Administration (FDA), Rhythm has finalized the protocol for its ongoing, open-label, single-arm, multinational pivotal Phase 3 clinical trial evaluating setmelanotide in patients with POMC deficiency obesity, an ultra-rare orphan disease that results in hyperphagia and severe, early-onset obesity. The FDA previously granted Breakthrough Therapy Designation for setmelanotide in this indication.

The study originally positioned mean percentage change in weight from baseline as the primary endpoint, and a categorical analysis of responders for weight, defined as patients achieving a 10 percent change from baseline, as the first secondary endpoint. Following recent discussions with regulatory authorities, the study’s primary endpoint will now be the responder analysis, with mean percentage change in weight as the first secondary endpoint, in a change that increases the power of the trial.  Other key secondary endpoints on hunger remain unchanged.