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  • Rhythm Pharmaceuticals announces positive topline ...

Rhythm Pharmaceuticals announces positive topline results from pivotal phase III trial evaluating setmelanotide in Bardet-Biedl and Alström syndromes.

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Published:26th Dec 2020
Rhythm Pharmaceuticals, Inc. announced positive topline results from a 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. The study met its primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful reductions in weight and hunger scores. All primary endpoint responders were patients with BBS. There were three evaluable patients with Alström syndrome and none of them met the primary endpoint. Rhythm enrolled 32 individuals with BBS and six individuals with Alström syndrome in the pivotal cohort for this Phase III trial. The primary analysis was conducted on 31 evaluable participants (28 with BBS and three with Alström syndrome) 12 years old and older. Five study participants (three with BBS and two with Alström syndrome) were younger than 12 years old at enrollment. The analysis of the primary endpoint shows: 11 of 31 or 34.5 per cent of participants achieved the primary endpoint of at least 10 percent reduction in body weight from baseline at approximately 52 weeks of therapy (p=0.0024); ? 11 of 28 patients with BBS achieved 10 percent reduction in body weight; ? 0 of 3 patients with Alström syndrome achieved 10 percent reduction in body weight. The analysis of the key secondary endpoints shows : Mean reduction from baseline in body weight was -6.2 percent (p<0.0001); mean reduction from baseline in most hunger rating was -30.8 percent (p><0.0001); 60.2 percent of participants achieved at least 25 percent reduction in most hunger scores from baseline at approximately 52 weeks of therapy (p><0.0001). consistent with prior clinical experience, setmelanotide was generally well tolerated:treatment-emergent adverse events (teaes) included mild injection site reactions and nausea with infrequent vomiting; there were no serious adverse events (saes) related to treatment with setmelanotide;eight patients discontinued from study drug treatment during the trial, five due to aes (one on placebo at the time), and three for other reasons (one on placebo at the time). rhythm plans to complete regulatory submissions to both the fda and the european medicines association (ema) for bbs in the second half of 2021. the company expects to finalize a path forward for alström syndrome upon completing a full analysis of the final data from this trial.>
Condition: Bardet-Biedl Syndrome
Type: drug

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