Under a collaboration agreement between the University of California, San Diego (UCSD) and Raptor, UCSD is conducting the Phase IIa clinical trial at UCSD’s General Clinical Research Center.
In the six-month, open-label clinical trial, UCSD enrolled a total of 12 adolescent non-alcoholic steatohepatitis (NASH) patients receiving a twice daily oral dose of cysteamine and will measure possible reduction of blood alanine aminotransferase and aspartate aminotransferase levels as a biomarker of cysteamine’s efficacy for potentially treating NASH. Positive Phase IIa data could lead to later stage clinical trials of cysteamine in NASH patients.
Cysteamine, currently cleared for market by the FDA and European Medicines Agency to treat nephropathic cystinosis, has demonstrated potential efficacy in preclinical and clinical studies in NASH, Huntington’s disease, Batten disease and other indications, said Raptor.
Under a license with UCSD, Raptor is developing cysteamine and a delayed-release form of 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 UCSD collaborators believe that increasing GSH has the potential to reverse NASH-related liver damage.
Ted Daley, president of Raptor’s clinical division, said: Raptor’s strategy is to leverage cysteamine’s known safety profile for the treatment of cystinosis and potential efficacy in several other indications. Positive data from the Phase IIa 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.