Raptor Pharma: Phase 2b CyNCh Study Fails To Meet Primary Endpoint - Quick Facts

(RTTNews) - Raptor Pharmaceutical Corp. (RPTP) announced topline results from the Phase 2b CyNCh study, which did not meet its primary endpoint of improving nonalcoholic steatohepatitis, or NASH, in children. The trial evaluated the safety and efficacy of RP103, or cysteamine bitartrate delayed-release capsules, in children with biopsy-confirmed NASH. There were no differences in adverse events observed in children on RP103 compared to placebo.

"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. We do not expect to advance this program based on topline results," said Julie Anne Smith, Raptor's President and CEO.