Raptor Pharmaceuticals Corp 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 (cysteamine) in adolescents diagnosed with Non-Alcoholic Steatohepatitis (NASH). NASH, a progressive form of liver disease that accounts for approximately 10 per cent of newly diagnosed cases of chronic liver disease, is one of the leading causes of cirrhosis in the US.

Under a previously announced collaboration agreement between Raptor and UCSD to evaluate the safety and potential efficacy of cysteamine in NASH patients, UCSD is performing the phase-2a 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.

Cysteamine is currently cleared for market by the US Food and Drug Administration (FDA) and European Medicines Agency (EMEA) to treat nephropathic cystinosis (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.

Ted Daley, president of Raptor's clinical division stated, "We are pleased that dosing is now underway in the phase-2a clinical trial in NASH, a disease that may have a severe impact on the long-term health of a large number of people. Cysteamine represents an exciting potential treatment for NASH patients currently limited to diet and exercise for disease management. The NASH programme 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."

Joel Lavine, paediatric gastroenterologist at UCSD and principal investigator for the NASH study, commented, "The initiation of patient dosing in the NASH trial offers the opportunity to evaluate a potential new treatment for this disorder. We are pleased to collaborate with Raptor for this study as we work to find a treatment for NASH."

NASH is believed to affect 2 to 5 per cent of the US population. While commonly diagnosed in insulin-resistant obese adults with diabetes and abnormal serum lipid profiles, NASH can also occur in children, elderly and non-diabetic persons of average weight. Though most people with NASH feel healthy and show no outward signs of liver disease, NASH causes decreased liver function and potentially leads to cirrhosis, liver failure and end-stage liver disease. In more severe cases, the progressive nature of NASH may require liver transplantation in affected patients.

Cysteamine is cleared for market by the FDA and EMEA to treat cystinosis, a rare and genetic lysosomal storage disease. The drug's potential to accumulate in the liver may create a targeted treatment effect for patients with NASH. DR Cysteamine is designed as an enterically coated, oral formulation of cysteamine.

Raptor obtained an exclusive, worldwide license to DR Cysteamine, as well as orphan drug designation from the FDA for DR Cysteamine for the treatment of nephropathic cystinosis, through its December 2007 acquisition of Encode Pharmaceuticals. In March 2008, Raptor acquired an exclusive worldwide license to intellectual property from UCSD for the use of cysteamine and DR Cysteamine for the treatment of NASH.

Raptor Pharma is a publicly traded biotechnology company dedicated to speeding the delivery of new treatment options to patients by enhancing existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise.