Rhythm Pharmaceuticals announces progress in development of setmelanotide
Rhythm Pharmaceuticals announced recent progress in the clinical development of the company's lead product candidate, setmelanotide, a first-in-class melanocortin-4 receptor 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 Alstrom 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 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.