Rhythm sees mixed result for setmelanotide in BBS and Alström syndrome

US biotech Rhythm Pharmaceuticals (Nasdaq: RYTM) today reported top-line results from its pivotal Phase III clinical trial evaluating setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, for the treatment of insatiable hunger and severe obesity in individuals with Bardet-Biedl syndrome (BBS) or Alström syndrome, two ultra-rare genetic diseases that often manifest with these symptoms.

The study met its primary and key secondary endpoints, including statistically-significant and clinically-meaningful reduction in weight and hunger scores in participants with BBS following treatment.

Approximately 34.5% of patients in the open-label study experienced at least 10% reduction in body weight from baseline to 52 weeks (p=0.0024). In addition, a total of 11 out of 28 patients with BBS achieved a 10% reduction in body weight.