Joel Lavine, M.D., Ph.D., 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."

Raptor's chief medical officer, Patrice Rioux, M.D., Ph.D., said, "These interim results have established proof-of-concept and support further clinical development of DR Cysteamine in NASH. This is an area of significant unmet need, especially with growing numbers of obese children diagnosed with the disorder. While the clinical hurdle is usually high for studies in children and adolescents, we are satisfied with the long-term safety demonstrated in this age group by the currently-marketed immediate-release cysteamine bitartrate formulation. This safety track record, coupled with our interim Phase 2a efficacy data, gives us a great sense of encouragement as we advance DR Cysteamine through the clinic."

Under a license with UC San Diego, Raptor is developing DR Cysteamine for cystinosis, NASH and other potential therapeutic indications. Cysteamine is known to be a scavenger of reactive oxygen species and potent antioxidant, most likely through its ability to increase intracellular glutathione levels. Cysteamine has also demonstrated potential efficacy in preclinical and clinical studies in Huntington's Disease, Batten Disease and other indications.

SOURCE Raptor Pharmaceutical Corp.