Raptor's chief medical officer, Patrice Rioux, M.D., Ph.D., said, "We are very excited about the results of this delayed-release cysteamine bitartrate pilot study in NASH patients. NASH is already an area of significant unmet medical need in adults and with the growing numbers of obese children diagnosed with the disorder, its importance is anticipated to grow rapidly in the coming years. While we are very pleased with the substantial impact seen on liver transaminases, we are particularly encouraged by the rapid drug effect we saw during the early phases of the study as well as the sustained liver enzyme reductions following drug withdrawal.  Supported by the long-term safety profile of this compound in cystinosis, this suggests the potential for a longer term, sustainable benefit to overall liver function in NASH patients with DR Cysteamine treatment."   

NASH is a more aggressive form of NAFLD, and is the most common cause of chronic liver disease in North America. Although most patients are asymptomatic and feel healthy in early phases of the disease, NASH causes decreased liver function and can lead to cirrhosis, liver failure and end-stage liver disease. While NASH is most common in insulin-resistant obese adults with diabetes and abnormal serum lipid profiles, its prevalence is increasing among juveniles as obesity rates rise within this patient population. There are no currently approved drug therapies for NASH; patients are limited to lifestyle changes such as diet, exercise and weight reduction to manage the disease.

Under a license with UC San Diego, Raptor is developing DR Cysteamine for cystinosis, NASH, Huntington's Disease and other potential therapeutic indications. Cysteamine is known to be a scavenger of reactive oxygen species and potent antioxidant, most likely through its ability to increase intracellular glutathione levels.

SOURCE Raptor Pharmaceutical Corp.