Rhythm Pharmaceuticals (NASDAQ:RYTM) and its collaborators presented new data at the Endocrine Society annual meeting from the company’s long-term extension trial with setmelanotide, which show continued body mass index and weight reductions in patients with SH2B1 or SRC1 deficiency obesity, or with POMC or LEPR insufficiency obesity.
“Consistent with our experience in other rare genetic diseases of obesity, these data demonstrate that ongoing treatment with setmelanotide delivers continued improvement in weight-related measures,” David Meeker, M.D., chairman, president and CEO of Rhythm, said in a statement.
He said these data also “reinforce our confidence in the design of the placebo-controlled Phase 3 EMANATE clinical trial, for which we are enrolling patients with each of these genetic variants, in hopes of focusing on those patient populations with impaired signaling in the melanocortin-4 receptor pathway who have highest probability of responding to our precision therapy, setmelanotide.”
Rhythm plans to enroll approximately 400 patients with hyperphagia and obesity that began in early childhood in the Phase 3 trial. The first patient was enrolled in April 2022 and Rhythm anticipates 12-to-18 months to enroll the trial.
Separately, Rhythm presented new data at ENDO from its long-term extension trial, which show continued body mass index and weight reductions in patients with Bardet-Biedl syndrome (BBS) or POMC or LEPR deficiency obesity receiving between 18 months and three years of setmelanotide therapy.
“As we approach the June 16 PDUFA goal date for BBS, we are eager to deliver setmelanotide to the waiting community, and we look forward to offering patients and their families a safe, effective medicine that can provide lasting benefit,” Dr. Meeker said.