Rhythm Pharmaceuticals Completes Pivotal Enrollment in Two Ongoing Phase 3 Clinical Trials

Rhythm Pharmaceuticals (NASDAQ:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity, today announced that it has completed enrollment of the pivotal cohorts of 10 patients in two separate, ongoing, registration-enabling Phase 3 clinical trials evaluating setmelanotide in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity.

As quoted in the press release:

POMC and LEPR deficiency obesity are two ultra-rare genetic disorders that result in excess hunger, also known as hyperphagia, and severe, early-onset obesity. Setmelanotide is a first-in-class melanocortin-4 receptor (MC4R) agonist, for which the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation and Orphan Drug Designation in POMC and LEPR deficiency obesity.

Rhythm expects to report initial data from the Phase 3 trials of both POMC and LEPR deficiency obesity in the third quarter of 2019. Rhythm then plans to submit concurrent New Drug Application (NDA) filings to the FDA for setmelanotide in patients with these indications based on one-year data from these pivotal cohorts of 10 patients. In addition, the Company plans to continue enrolling supplemental patients in both trials who may not complete one year of treatment at the time of NDA filing, including patients between six and 11 years of age, to provide additional data regarding the use of setmelanotide in people living with POMC and LEPR deficiency obesity.

Click here to read the full press release.