RP104 is a proprietary delayed-release tablet formulation of cysteamine bitartrate, used as a treatment option for non-alcoholic steatohepatitis (NASH), an advanced form of non-alcoholic fatty liver disease (NAFLD) in children.
The Phase 2b clinical trial is a randomized multicenter double-blind placebo-controlled study that will enroll a total of 160 pediatric participants at ten US centers in the NIDDK-sponsored NASH Clinical Research Network.
The trial will investigate the safety and potential efficacy of RP104, and is anticipated to begin in the first calendar quarter of 2012 with NIDDK and Raptor sharing the expenses for the trial.
In the study, the primary endpoint of the trial is to assess whether 52 weeks of treatment with RP104 in children reverses damage caused by NASH, and the secondary endpoints will include blood markers for liver health including alanine transaminase (ALT) and aspartate transaminase (AST), as well as safety and tolerability.
Under the CRADA, Raptor will retain exclusive development and commercial rights to the clinical data resulting from the CyNCh trial.
Raptor president Ted Daley said the partnership will advance its RP104 program for NASH, and enables them to work with an already established network of many of the leading NASH clinics in the US.