UPDATE: Raptor Pharma Offers Topline Results from Phase 2b CyNCh Study of RP103 in Pediatric Nonalcoholic Steatohepatitis: Did Not Meet Primary Endpoint

Raptor Pharmaceutical Corp.

today announced topline results from the Phase 2b CyNCh study, which did not meet its primary endpoint of improving nonalcoholic steatohepatitis (NASH) in children. The trial evaluated the safety and efficacy of RP103, or cysteamine bitartrate delayed-release capsules, in children with biopsy-confirmed NASH. The study did not achieve its primary endpoint defined as a two-point decrease in NAFLD Activity Score (NAS) and no worsening of fibrosis (p-value = 0.34). There were no differences in adverse events observed in children on RP103 compared to placebo. The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health, sponsored and conducted the CyNCh study through a Cooperative Research and Development Agreement (CRADA) with Raptor. Study investigators intend to submit the data for publication and present topline results at the upcoming American Association for the Study of Liver Diseases (AASLD) meeting being held November 13-17, 2015 in San Francisco. "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're disappointed with this outcome given the paucity of treatment for these children with NASH. While we'll work closely with the NIDDK to understand the full data set, we do not expect to advance this program based on topline results," said Julie Anne Smith, Raptor's President and CEO. "This clarifies our near-term priorities, which are to maximize the reach of PROCYSBI in nephropathic cystinosis, further the development of RP103 in Huntington's and mitochondrial diseases, prepare for QUINSAIR'S launch and initiate at least one trial in nontuberculous mycobacteria or bronchiectasis. We remain wholly devoted to developing and commercializing transformational treatments for people living with rare diseases."