Receptos Soars As Bowel Disorder Drug Meets Phase 2 Goals
October 28, 2014
By Riley McDermid, BioSpace.com Breaking News Staff
San Diego biotech Receptos, Inc. said late Monday that it saw encouraging results for its ulcerative colitis drug during a recent Phase II trial, raising hopes that the therapy will soon be used to treat both colitis and some forms of multiple sclerosis.
The drug, RCP1063, was discovered at The Scripps Research Institute and licensed to Receptos in 2009. The company said in a statement it recently met its primary endpoint and all secondary endpoints with statistical significance in patients on the 1 mg dose of RPC1063 in an eight week induction period, during a trial dubbed Touchstone. News of the successful trial results sent Receptos's share price soaring more than 26 percent in early morning trading Tuesday.
During the randomized, double-blind, placebo-controlled trial, the company tested two orally administered doses (0.5 mg and 1 mg) of RPC1063 in patients with ulcerative colitis in 199 patients across 57 sites in 13 countries. Receptos said the study had 10 percent more participants than planned, “due to a strong interest among investigators and patients to participate in the study.”
It said its primary goal, to see the proportion of patients in clinical remission in eight weeks, as defined by the industry standard Mayo scoring criteria, was achieved by 16.4 percent of patients on the 1 mg dose of RPC1063, as compared to 6.2 percent of patients on placebo, which was statistically significant (p<0.05). In the low dose group of 0.5 mg, 13.8 percent of patients achieved clinical remission, which was not statistically significantly different from the placebo group.
The drug remains the company’s most alluring autoimmune therapy, with the recent results of this study and an almost year-long Phase III trial in MS patients showing enormous promise for investors and clinicians.
"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 William Sandborn, a professor of medicine and directors of the Division of Gastroenterology at the University of California San Diego Inflammatory Bowel Disease Center, in a statement. "The results also showed a favorable overall safety profile. A Phase III program is clearly warranted in order to confirm and extend these results."
The company said RPC1063 was generally well tolerated, with any adverse events mild or moderate in nature, and thus far no concerning signals of adverse cardiac or liver events. Based on these results, Receptos said it plans to begin a Phase III trial program in 2015 to compare the safety and efficacy of the 1 mg dose of RPC1063 to placebo in patients with UC.
“In addition, these results suggest the potential for RPC1063 to be used in the treatment of Crohn's disease, a related inflammatory bowel disease,” it said in a statement. The company said it now plans to initiate a Phase III study of RPC1063 for the treatment of Crohn's disease in 2015.