Raptor Pharmaceuticals Announces Completion of Patient Enrollment in Phase 2a Clinical Trial in Non-Alcoholic Steatohepati...(reating NASH. Positive Phase 2a data cou...),Health

Cysteamine, currently cleared for market by the U.S. Food and Drug Administration ("FDA") and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), has demonstrated potential efficacy in preclinical and clinical studies in NASH, Huntington's Disease, Batten Disease and other indications. Under a license with UC San Diego, Raptor is developing cysteamine and a delayed-release form of cysteamine ("DR Cysteamine") for NASH and a number of new potential therapeutic indications. Preliminary data suggests that cysteamine is readily taken up by the liver, and is a precursor of the potent liver anti-oxidant glutathione ("GSH"). Raptor's UC San Diego collaborators believe that increasing GSH has the potential to reverse NASH-related liver damage.

Ted Daley, President of Raptor's clinical division stated, "Raptor's strategy is to leverage cysteamine's known safety profile for the treatment of cystinosis and potential efficacy in several other indications. In this case, cysteamine represents an exciting potential treatment for NASH, a disease that may have a severe impact on the long-term health of a growing worldwide patient population among both adults and adolescents. Positive data from the Phase 2a NASH trial may enable us to advance to later stage clinical trials in NASH and may provide further insight on how to maximize the potential value of cysteamine for NASH and other potential indications."

Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UC San Diego and principal investigator for the NASH study, commented, "We are encouraged that the Phase 2a trial has reached full enrollment and look forward to completing patient dosing and evaluating cysteamine's clinical results in adolescents with NASH. There is currently no FDA-approved, effective treatment option for their dis
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