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Therapy for Prader-Willi syndrome enters extension study
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Essentialis Inc., announced the start of an extension trial to evaluate long-term effects of a once-daily diazoxide choline controlled-release tablet for treatment of symptoms related to Prader-Willi syndrome, according to a company press release
The 6-month open-label trial includes participants who completed the first two phases of clinical study PC025. Participants are aged 10 to 22 years old (mean age, 16 years) and have genetically confirmed Prader-Willi syndrome (PWS) with obesity (52% body fat) and moderate hyperphagia. They were initially treated with diazoxide choline controlled-release for 10 weeks and then randomly assigned to continue treatment or placebo for 4 more weeks.
“Given that we observed highly significant and clinically relevant reductions in hyperphagia, reductions in body fat, increases in lean body mass, reductions in aggressive, threatening and destructive behaviors and reductions in cardiovascular risk factors, with a safety profile that was well matched against these benefits, it is not surprising that nearly every patient who completed the first two phases of the study wanted to continue on drug,” said Neil M. Cowen, president and chief scientific officer of Essentialis.
Diazoxide choline was granted orphan status for the treatment of PWS by the FDA in May of last year. Results of the continuation study are expected in early 2016, according to the release.