Hot on the heels of its merger with the struggling TorreyPines Therapeutics, Raptor Pharmaceuticals (RPTPD) reported that its experimental drug to treat liver disease hit its primary endpoint in a Phase IIa trial, and its stock immediately rocketed up 46 percent on the news.
The therapy, dubbed DR Cysteamine, was tested in adolescent patients with non-alcoholic steatohepatitis ("NASH"). After the six-month treatment phase, the study achieved the primary endpoint: mean blood levels of alanine aminotransferase (ALT), were reduced by over 50 percent. Additionally, over half of the study participants had achieved normalized ALT levels by the end of the treatment phase.
"We were encouraged by the results of this study," said Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UC San Diego. "The degree of ALT and AST reductions are indicative of likely improvements in severity of fatty liver damage. The trial results are consistent with ALT and AST reductions normally seen in patients that achieve at least 10 percent weight loss, 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 analyzing these patients during the post-treatment phase."
- check out Raptor's release
- here's the Reuters piece