Biopharmaceutical firm Rhythm has started two Phase II clinical trials to assess the safety and effectiveness of setmelanotide (RM-493) for rare genetic disorders of obesity.
Setmelanotide is a new melanocortin 4 receptor (MC4R) agonist that is being assessed to treat Prader-Willi Syndrome (PWS) and POMC-null obesity, which are associated with defects in the MC4 signaling pathway.
The first Phase II study will assess the safety and efficacy of setmelanotide on weight and eating behaviours in patients with PWS, which is a rare genetic disorder that causes life-threatening obesity.
The randomised, double-blind and placebo-controlled trial will assess safety and efficacy of setmelanotide administered once daily by subcutaneous injection for up to 10 weeks of treatment.
Rhythm will enrol around 36 obese adolescent and adult patients with PWS in the trial.
Rhythm chief medical officer Dr Fred Fiedorek said: "The Phase II Prader-Willi clinical trial will assess the effect of setmelanotide as essentially 'replacement therapy' for the treatment of the severe obesity and hyperphagia in PWS."
The second Phase II clinical trial will assess the safety and efficacy of setmelanotide on weight and appetite in POMC-null patients, which is a very rare and life-threatening genetic disorder for which there are no effective treatments.
These study will enrol around six obese adolescent and adult patients with POMC-null genetic defects and will assess the safety and efficacy of setmelanotide administered once daily by subcutaneous injection for up to 13 weeks.
Rhythm CEO Keith Gottesdiener said: "Our personalised medicine approach to restore MC4 pathway function in patients with Prader-Willi syndrome and POMC-null obesity is targeting what we believe is the direct cause of the extreme hyperphagia and obesity in these genetic diseases."