Receptos: good results for ulcerative colitis drug

Members of the Hugh Rosen laboratory at Scripps, where the drug candidate RPC-1063 was discovered.

San Diego biotech Receptos said Monday that a Phase 2 trial of its drug for ulcerative colitis drug has succeeded. Receptos said it will begin a Phase 3 trial of the experimental drug, RPC1063, and begin a Phase 2 trial for another inflammatory bowel ailment, Crohn's disease.

RPC1063 met its primary efficacy endpoint and all secondary endpoints, Receptos said in a statement. Discovered at The Scripps Research Institute, RCP1063 was licensed to Receptos in 2009.

The drug is one of Receptos' most promising assets, potentially useful for a variety of autoimmune diseases. In December, the company started a Phase 3 trial of it for relapsing multiple sclerosis.

In the trial, a randomized, double-blind, placebo-controlled test of 199 patients, the drug was found to achieve its main objective of clinical remission by 8 weeks in 16.4 percent of patients given a 1 mg dose. In a placebo group, 6.2 percent of patients had entered remission by 8 weeks. This represented a statistically significant improvement. A lower dose of 0.5 mg did not produce a significant improvement.

Achievement of secondary endpoints, which pointed to an improving condition, occurred in 58.2 percent of patients on the 1 mg dose of RPC1063, as compared to 36.9 percent of patients on placebo. More detailed information will be given at an upcoming scientific meeting.

The results of this trial demonstrated a significant treatment effect of orally administered RPC1063 at the 1 mg dose, with what appears to be a clear dose response and consistency across both the primary and secondary efficacy endpoints, said Dr. William Sandborn, a professor of medicine and director of the UC San Diego Inflammatory Bowel Disease Center, in a Receptos press release. The results also showed a favorable overall safety profile. A Phase 3 program is clearly warranted in order to confirm and extend these results.

Trial results were welcomed by scientist Hugh Rosen of The Scripps Research Institute, whose lab performed the original research that identified the compound.

"We believe in fundamental science as an intelligent intervention in disease," said Rosen, a Receptos co-founder. "It's exciting for scientists at the bench to see their work going on to transform the lives of patients."

The roots of RCP1063 go back to a list of compounds collected by the National Institutes of Health, Rosen said. His lab screened the compounds to look for desired properties, especially the ability to regulate cellular receptors that govern immune response. One of these receptors, called S1P1, regulates the activity of white blood cells called lymphocytes. Multiple sclerosis is caused when these lymphocytes attack the myelin sheath that protects nerves.

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Receptos: good results for ulcerative colitis drug