Shares of Raptor Pharmaceuticals (RPTP) closed down 37.49% today after the company announced that it failed a phase 2b liver study in children. More specifically the targeted disease in these children is nonalcoholic steatohepatitis -- NASH. As the name suggests this is fatty liver disease not associated with excessive alcohol drinking. 

The phase 2b trial enrolled up to 169 children between the ages of eight and 17, randomized into different dosing groups. Patients were randomized to take either 600, 750, or 900 mg per day of the company's drug RP103 or a placebo compound. RP103 is a delayed release capsule, taken orally, with the intended use of targeting the underlying causes of metabolic diseases 

This phase 2b trial was known as the CyNCh trial, which stands for "Cysteamine Bitartrate Delayed-Release for the treatment of nonalcholic Fatty Liver Disease -- NAFLD". The primary endpoint of the trial was to be able to see a two-point decrease in the NAFLD Activity Score -- NAS and no worsening of the fibrosis of the liver in these patients. The trial failed to meet on improving either one of these objectives therefore the primary endpoint failed to show any statistical significance against the placebo compound. 

Raptor Pharmaceuticals now intends to discontinue any further development of this trial for children with NASH. The company will still publish some additional detail about these results to distribute at the upcoming American Association for the Study of Liver Diseases  (AASLD) meeting being held November 13th through the 17th.  RP103 is still being studied for application to other diseases.