Rhythm reports Phase II data of setmelanotide to treat Bardet-Biedl Syndrome

In October, Rhythm Pharmaceuticals Inc. (NASDAQ:RYTM) reported preliminary data from 5 Bardet-Biedl Syndrome patients with morbid obesity and hyperphagia in the Phase II portion of a Phase II/III trial showing that setmelanotide (RM-493) reduced body weight, the primary endpoint, in 4 patients by 12.1%, 7.9%, 9.7% and 9.7%, respectively. One patient had no weight loss, but achieved weight stabilization. Hunger scores improved in all 5 patients. Setmelanotide was well tolerated. Data were presented at the ObesityWeek meeting in Washington D.C.

The open-label, U.S. and U.K. trial is evaluating up to 3 mg subcutaneous setmelanotide daily for 52 weeks in about 25 patients with rare genetic obesity disorders, including proopiomelanocortin (POMC) deficiency obesity, leptin receptor (LEPR; CD295) deficiency obesity, Alstrom syndrome and Bardet-Biedl Syndrome. Secondary endpoints include safety, body fat mass, hunger, insulin sensitivity, waist circumference and reversal of weight during the off treatment withdrawal phase...