NASH drug fails to meet primary endpoint in pediatric patients

Raptor Pharmaceuticals announced that RP103, its potential drug to treat nonalcoholic steatohepatitis, failed to meet its primary endpoint in the phase 2b CyNCh study among pediatric patients, according to a press release.

The Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease in Children study, known as the CyNCh study, is a multi-center, placebo-controlled clinical trial of 169 children between the ages of 8 and 17 years with biopsy-confirmed moderate-to-severe NAFLD. Patients were randomly assigned to receive either 600 mg, 750 mg or 900 mg per day of RP103 (cysteamine bitartrate delayed-release capsules) or placebo for 52 weeks.

The trial evaluated the safety and efficacy of RP103 in children with biopsy-confirmed NASH. It did not achieve its primary endpoint, which was defined as a two-point decrease in NAFLD Activity Score (NAS) and no worsening of fibrosis (P = .34), according to the release.

“While these trial results replicated the serological improvements seen in the earlier phase 2a study, they did not translate into a measurable effect on histology,” Julie Anne Smith, CEO of Raptor, said in the release. “We’re disappointed with this outcome given the paucity of treatment for these children with NASH.”

The study results will be presented in detail at The Liver Meeting November 13-17, 2015 in San Francisco.

“We remain wholly devoted to developing and commercializing transformational treatments for people living with rare diseases,” Smith said in the release.

Disclosures: Smith reports being employed by Raptor.