Receptos Reports Positive Results for the Maintenance Period of the Phase 2 TOUCHSTONE Trial of Ozanimod in Ulcerative Colitis

Receptos, Inc. RCPT today announced that the maintenance period of TOUCHSTONE, the Phase 2 trial of ozanimod (formerly RPC1063) in ulcerative colitis (UC), met all efficacy endpoints with statistical significance in patients on the 1 mg dose of ozanimod after 32 weeks of treatment. The overall safety and tolerability profile of ozanimod was consistent with the results of TOUCHSTONE's induction period and those observed in the RADIANCE Phase 2 trial in relapsing multiple sclerosis (RMS), and continues to support the potential for orally administered ozanimod to significantly improve the treatment paradigm for UC patients. This randomized, double-blind, placebo-controlled trial assessed the efficacy, safety and tolerability of two orally administered doses (0.5 mg and 1 mg) of ozanimod versus placebo in 199 patients with moderately-to-severely active UC across 57 sites in 13 countries. For the induction period, the trial met its primary efficacy and all secondary endpoints with statistical significance for patients on the 1 mg dose after 8 weeks of treatment. In the maintenance portion of the trial, 103 patients who had achieved clinical response at week 8 continued with treatment for an additional 24 weeks. The proportion of patients in clinical remission at week 32 as defined by the industry standard Mayo scoring criteria was statistically significant in favor of both the 1 mg group and the 0.5 mg group (p < 0.05) compared to placebo. All secondary endpoints at week 32, including clinical response, change in the Mayo score and mucosal improvement on endoscopy were also positive and statistically significant for the 1 mg dose. The detailed results of the maintenance period portion of the TOUCHSTONE trial are expected to be presented at a major scientific meeting in the coming months. Ozanimod was generally well tolerated, and the incidence of adverse events across the active treatment groups and placebo appeared to be similar. Most adverse events were either mild or moderate in nature, and there appeared to be no concerning signals in the adverse events of special interest, including the cardiac and hepatic safety profiles. Rates of liver transaminase elevations observed in patients receiving ozanimod were low and consistent with the earlier Phase 2 trial in RMS. "The robust results observed in the maintenance portion of this trial support the longer term efficacy of ozanimod," said Dr. William Sandborn, M.D., Professor of Medicine and Chief, Division of Gastroenterology and Director, University of California San Diego Inflammatory Bowel Disease Center. "Ozanimod appears to show effectiveness in both inducing and maintaining clinical remission and response in patients with ulcerative colitis, with a favorable overall safety profile." Receptos plans to initiate a Phase 3 program in 2015 in patients with moderately-to-severely active UC to confirm these findings. In addition, these results continue to suggest the potential for ozanimod to be used in the treatment of Crohn's disease, a related inflammatory bowel disease. Receptos plans to initiate a Phase 2 study of ozanimod for the treatment of Crohn's disease in 2015. "The positive results of the maintenance period of the TOUCHSTONE study exceeded our expectations with respect to the potential for long term clinical benefit of ozanimod in ulcerative colitis," said Faheem Hasnain, President and Chief Executive Officer of Receptos. "The consistency of the efficacy data across the various endpoints for the high dose, combined with the favorable safety profile, gives us continued confidence to advance the development of ozanimod in inflammatory bowel disease. Having completed our end-of-phase 2 meeting with the FDA and Scientific Advice meetings with the Competent Authorities in Europe, we believe that we now have regulatory alignment and are prepared to accelerate the initiation of the Phase 3 program in ulcerative colitis."