Under a previously announced collaboration agreement between Raptor and University of California, San Diego (UCSD) to evaluate the safety and potential efficacy of cysteamine in non-alcoholic steatohepatitis (NASH) patients, UCSD is performing the Phase IIa open-label clinical study at UCSD’s general clinical research center while Raptor is providing funding and clinical supplies of cysteamine.
The trial is expected to enroll 12 adolescent NASH patients who have been managing the disease through diet and exercise. Positive data could provide the rationale for later stage clinical studies of cysteamine in NASH patients.
Raptor is developing cysteamine and a delayed release form of cysteamine for a number of new potential therapeutic indications, including NASH, under licenses acquired from UCSD.
Ted Daley, president of clinical division at Raptor, said: “Cysteamine represents an exciting potential treatment for NASH patients currently limited to diet and exercise for disease management. The NASH program is part of our strategy to leverage cysteamine’s known safety profile and potential efficacy in several indications where it has already shown promise in clinical and preclinical studies.”