Rhythm Pharmaceuticals Inc (NASDAQ:RYTM) has announced interim data from its long-term extension study evaluating setmelanotide in patients with Bardet-Biedl Syndrome (BBS).
- BBS is a genetic disorder characterized by rod/cone dystrophy, polydactyly, central obesity, hypogonadism, and kidney dysfunction in some cases.
- Of the patients enrolled in Rhythm's long-term extension trial, 19 individuals with BBS have reached 24 months on therapy.
- The mean percent reduction in body mass index (BMI) from pivotal trial baseline was -14.3% (n=19).
- Related: Rhythm Pharma's Imcivree Wins UK Approval For Obesity, Hunger Control.
- The mean percent reduction in body weight from pivotal trial baseline among patients 18 years of age or older was -14.9% (n=6);
- The mean reduction in BMI Z score from pivotal trial baseline among patients younger than 18 was -0.72 (n=12).
- Setmelanotide was generally well-tolerated in the long-term extension study, and no new safety signals were observed.
- A supplemental marketing application for setmelanotide for BBS or Alström syndrome is currently under FDA review with the PDUFA goal date of March 16.
- A Type II variation application has also been submitted to the European Medicines Agency (EMA) for obesity and control of hyperphagia in adult and pediatric patients six years of age and older with BBS.
- Rhythm will also provide an update on ongoing preparations in support of a potential U.S. commercial launch of Imcivree (setmelanotide) for obesity and hyperphagia in patients with BBS during a virtual event today at 4 p.m. ET.
- Price Action: RYTM shares are down 2.65% at $6.61 during the market session on the last check Wednesday.
