Liver Drug RP103 Fails To Match Its Main Goals in Phase 2b CyNCh Study

Raptor Pharmaceutical Corp said it will no more develop its liver drug RP103 after the drug failed to meet the main goal in a second mid-stage study. The disappointing results of the drug wiped out more than third of the company’s market value.

The biopharmaceutical company on Monday said in a statement that the drug called RP103 (cysteamine bitartrate) did not meet the main goals in the Phase 2b CyNCh study.

Researchers of the study involving around 169 participants between 8 and 17 years old evaluated the effectiveness and safety of RP103 in children diagnosed with nonalcoholic steatohepatitis (NASH).

NASH is a liver inflammation and the damage is attributed to the build-up of fat in the liver. The condition could cause scarring of the liver, which could even lead to cirrhosis.

For the trial, the participants were randomly given either 600 mg per day, 750 mg per day, or 900 mg per day of the experimental drug or placebo for a period of 52 weeks.

Results of the study showed that the patients who were given RP103 did not show any significant improvements in their liver disease.

The company said its drug met its main goal in the first mid-stage study conducted in 2010 but based on the result of the latest clinical trial it has decided not to pursue the development of the drug for NASH.

It will continue the development of the drug for Huntington's disease and mitochondrial diseases, said Raptor. RP103 is also being developed as treatment for the degenerative disease Huntington's and Leigh Syndrome, a neurological disorder.

Raptor President Julie Anne Smith expressing her disappointment over the outcome of the trial said the company remains devoted to the development of treatments for people who were diagnosed with rare diseases.