- Preliminary results from a Phase 2 clinical trial assessing Rhythm Pharmaceuticals' (RYTM +4.5%) setmelanotide in patients with Bardet-Biedl syndrome (BBS) showed a positive effect. The data are being presented at ObesityWeek 2017 in Washington, D.C.
- Four of five morbidly obese BBS patients received daily injections of setmelanotide for 52 weeks. Within 6 - 19 weeks of initiation, four experienced weight loss of almost 10% to over 12%. The drug was well-tolerated with a favorable safety profile.
- The study is ongoing. According to ClinicalTrials.gov, the primary completion date is September 2018.
- BBS is a rare inherited disorder characterized by obesity, vision loss, extra toes or fingers and hypogonadism.
- Setmelanotide binds to and activates receptors in the brain's hypothalamus called MC4 that are thought to play a key role in the regulation of appetite. In addition to suppressing appetite, setmelanotide increases energy expenditure in obese people without increasing heart rate or blood pressure.
- Now read: Orphan Disease IPO, Rhythm Pharmaceuticals: Initiating Coverage With Buy Rating And Price Target (40% Upside)
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