Raptor Pharmaceuticals Corp. (RPTP) announced the initiation of patient dosing in a Phase 2a clinical trial, in collaboration with the University of California, San Diego ("UCSD"), to evaluate cysteamine bitartrate in adolescents diagnosed with Non-alcoholic Steatohepatitis ("NASH"). NASH, a progressive form of liver disease that accounts for approximately 10% of newly diagnosed cases of chronic liver disease, is one of the leading causes of cirrhosis in the U.S.
Cysteamine is currently cleared for market by the U.S. Food and Drug Administration ("FDA") and European Medicines Agency ("EMEA") to treat nephropathic cystinosis, a rare and genetic lysosomal storage disease. Raptor is developing cysteamine and a delayed release form of cysteamine ("DR Cysteamine") for a number of new potential therapeutic indications, including NASH, under licenses acquired from UCSD. Cysteamine has demonstrated potential efficacy in preclinical and clinical studies in NASH, Huntington's Disease, Batten Disease and other indications.
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