Receptos' Lead Drug Succeeds In Phase 2 Study For Ulcerative Colitis
By C. Rajan, contributing writer
San Diego biotech firm Receptos has announced the success of its lead drug, RPC1063, in a Phase 2 trial for ulcerative colitis (UC). The experimental drug showed good safety and tolerability, while meeting both its primary and secondary endpoints. The company now plans to move the drug forward into a Phase 3 trial for UC next year, as well as begin a Phase 2 trial for another related condition, Crohn's disease.
Both ulcerative colitis and Crohn's disease are painful and debilitating autoimmune conditions, and are two common forms of inflammatory bowel disease (IBD). According to CDC estimates, about 1.3 million people in the U.S. suffer from IBD.
Receptos’ global Phase 2 trial for UC (called TOUCHSTONE) was a randomized, double-blind study evaluating the safety and efficacy of two doses of oral RPC1063 versus placebo in 199 patients with moderate to severe ulcerative colitis. The results demonstrated that the drug reached its main goal of clinical remission by 8 weeks in 16.4 percent of patients after they’d been given a 1 mg dose, compared to 6.2 percent of patients in the placebo group. RPC1063 also met the secondary endpoints of improved conditions in patients. The ongoing maintenance portion of the trial will be completed in 2015.
Receptos’ lead drug RPC1063, is a sphingosine 1-phosphate 1 receptor (S1P1R) small molecule modulator, which controls immune responses by regulating the cellular receptor S1P1, which in turn regulates the activity of the immune system’s lymphocytes. RPC1063 was discovered at The Scripps Research Institute (TSRI) and licensed to Receptos in 2009.
The company is developing RPC1063 for several autoimmune conditions, including relapsing multiple sclerosis (RMS) and inflammatory bowel disease. The drug showed positive results in a Phase 2 trial for MS in June, and has already moved into a Phase 3 trial for relapsing MS.
"The positive results of the TOUCHSTONE study exceeded our expectations with respect to the treatment effect of RPC1063," said Faheem Hasnain, President and CEO of Receptos. "The consistency of the efficacy data across the various endpoints for the high dose, combined with the favorable safety profile, gives us confidence to move forward expeditiously with Phase 3 trials in ulcerative colitis and a Phase 2 trial in Crohn's disease. There is a tremendous unmet need for a novel orally administered therapy in these patient populations. In addition, we believe that RPC1063 may have promise in other therapeutic areas, and we intend to continue to explore other autoimmune indications where RPC1063 may provide utility."