(RTTNews) - Raptor Pharmaceutical Corp. (RPTPD) on Monday announced positive findings from the completed treatment phase of its open-label Phase 2a clinical trial of delayed-release cysteamine bitartrate or DR Cysteamine in adolescent patients with non-alcoholic steatohepatitis or NASH, a progressive form of liver disease.

At the end of the initial six-month treatment phase, the study achieved its primary endpoint of reduction in mean blood levels of alanine aminotransferase or ALT, a common biomarker for NASH, which lessened by over 50%. Also, over half of the study participants achieved normalized ALT levels by the end of the treatment phase.

NASH is believed to affect 2% to 5% of the U.S. Population and there are no currently approved drug therapies for it. Patients are asked to bring about lifestyle changes such as diet, exercise and weight reduction to manage the disease. NASH is most common in insulin-resistant obese adults with diabetes and abnormal serum lipid profiles, but its prevalence is increasing among juveniles as obesity rates rise.
Most patients are asymptomatic and feel healthy, but NASH causes decreased liver function and can lead to cirrhosis, liver failure and end-stage liver disease.

The open-label Phase 2a trial of a prototype formulation of DR Cysteamine is being conducted under a collaboration agreement between Raptor and the University of California, San Diego. It entails six months of treatment followed by a six-month post-treatment monitoring period.

Eligible patients with baseline ALT and aspartate aminotransferase or AST measurements at least twice that of normal levels were enrolled to receive twice-daily, escalating oral doses of up to 1,000 mg of DR Cysteamine. The trial currently has enrolled eleven NASH patients in the age group of 11-18 years.
No major adverse events were reported during the six-month treatment phase. The six-month post-treatment period is currently underway. Full results are being submitted for peer review by Raptor and UC San Diego, and are expected to be presented in 2010.

Under a license with UC San Diego, Raptor is developing DR Cysteamine for cystinosis, NASH and other potential therapeutic indications.

Joel Lavine, pediatric gastroenterologist at UC San Diego and principal investigator for the NASH study, stated, "We were encouraged by the results of this study. 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% 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."

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