Raptor Pharmaceutical has announced positive findings from a Phase II trial of cysteamine bitartrate to treat non-alcoholic steatohepatitis (NASH), a rare adolescent liver disease for which there is no approved treatment.
After six months the open-label Phase IIa clinical trial using cysteamine bitartrate (DR Cysteamine) achieved a more than 50% reduction in blood levels of alanine aminotransferase (ALT), a common biomarker of NASH.
Additionally, over half of the study participants achieved normalised ALT levels by the end of the treatment phase.
NASH is a progressive form of liver disease believed to affect 2–5% of the US population. The disease is most common in insulin-resistant obese adults with diabetes but its prevalence is increasing with the rise of juvenile obesity rates in the US.
Principal investigator for the NASH study, paediatric gastroenterologist Joel Lavine said that the degree of ALT and AST reductions are indicative of likely improvements in severity of fatty liver damage.
"Even though study participants did not show a significant change in body mass index, DR Cysteamine appears to be a promising candidate for NASH and we look forward to further analysing these patients during the post-treatment phase," Lavine said.
The trial, which is being conducted under a collaboration agreement between Raptor and the University of California, San Diego entails six months of treatment followed by a six-month post-treatment monitoring period enrolled to receive twice-daily, escalating oral doses of up to 1,000mg of DR Cysteamine.
Full results are expected to be presented in 2010.