Raptor Pharmaceutical has entered into a cooperative research and development agreement with the National Institute of Diabetes and Digestive and Kidney Diseases to conduct a Phase IIb clinical trial of drug candidate RP104.
Raptor's RP104 is a proprietary delayed-release tablet formulation of cysteamine bitartrate, used to treat non-alcoholic steatohepatitis, an advanced form of non-alcoholic fatty liver disease in children.
The randomised, multicentre, double-blind and placebo-controlled trial will enrol 160 paediatric participants at ten US centres in the non-alcoholic steatohepatitis clinical research network to assess the safety and potential efficacy of RP104.
The primary aim of the trial is to investigate whether 52 weeks of treatment with the drug reverses the damage caused by non-alcoholic steatohepatitis as measured by changes in non-alcoholic fatty liver disease activity score, while secondary endpoints will include blood markers for liver health including alanine transaminase and aspartate transaminase, as well as safety and tolerability.
According to the cooperative research and development agreement, Raptor will retain exclusive development and commercial rights to the clinical data resulting from the trial.
The study is anticipated to commence in the first calendar quarter of 2012, with National Institute of Diabetes and Digestive and Kidney Diseases and Raptor sharing its cost.
Of the total $14-16m the trial is expected to cost, Raptor will provide $6m, as well as clinical trial materials and drug manufacturing/quality support estimated at approximately $1m, while the remainder will be funded by the National Institute of Diabetes and Digestive and Kidney Diseases.
Raptor president Ted Daley said the collaboration will advance the RP104 programme, and will allow the company to initiate and complete the Phase IIb clinical trial far sooner than if they were to conduct the trial on their own.
The CyNCh trial follows positive results of an open-label Phase IIa clinical trial, which involved children with biopsy-confirmed diagnosis of moderate to severe non-alcoholic fatty liver disease.
It showed a considerable decline in ALT and AST levels during the treatment period with RP104.