Raptor Pharmaceutical said that its RP103 (cysteamine bitartrate) drug failed to meet the main goal in a second mid-stage study.
The drug was being tested in children with nonalcoholic steatohepatitis (NASH), which is a liver inflammation caused by a buildup of fat in the liver.
The study involved 169 participants between 8 and 17 years old. The participants randomly received either 600 mg per day, 750 mg per day, 900 mg per day of RP103 or placebo for a period of 52 weeks.
Results revealed that the patients who took the drug did not experience significant improvements in their liver disease.
The drug met its primary goal in the first mid-stage study carried out in 2010 but depending on the result of the latest clinical trial, Raptor said it will no longer pursue the development of RP103.
Raptor Pharmaceutical president and CEO Julie Anne Smith said: “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.”
The company said that it will continue developing RP103 for Huntington’s and mitochondrial diseases.
The results of the trial will be submitted for publication and presented at the American Association for the Study of Liver Diseases meeting in San Francisco, California, US, next month.
PBR Staff Writer
Published 15 September 2015