Phase II trial of RPC 1063 (ozanimod) demonstrates efficacy and safety in ulcerative colitis- Celgene Corporation

Celgene Corporation has announced detailed results from the maintenance phase of the TOUCHSTONE phase II clinical trial of RPC 1063 (ozanimod) in patients with moderate to severe ulcerative colitis. In the trial, a significantly greater proportion of patients on ozanimod achieved or maintained clinical remission at 32 weeks compared with those on placebo. The trial evaluated the efficacy and safety of 0.5 mg and 1 mg doses of ozanimod compared with placebo after eight weeks of treatment (induction phase) in 197 patients with moderate to severe ulcerative colitis. The primary endpoint was the proportion of patients in remission at week 8. Secondary endpoints were the proportion of patients achieving a clinical response, the proportion of patients with mucosal improvement and the change from baseline in Mayo score.

Previously reported results showed TOUCHSTONE met its primary and secondary endpoints with statistical significance for patients on the 1 mg dose of ozanimod versus placebo in the 8-week induction phase. For the maintenance phase, 103 patients who achieved a clinical response at week 8 (reduction in Mayo score of at least 3 and at least 30% with a decrease in rectal bleeding score of at least 1 or a rectal bleeding score of 1 or less) continued with their original treatment for an additional 24 weeks; of these, 91 patients completed 32 weeks of treatment. Twenty one percent of patients on ozanimod 1 mg achieved or maintained clinical remission at week 32, compared with 6% on placebo, and clinical response at week 32 was achieved or maintained by 51% and 20% of patients, respectively. Mucosal improvement was also significantly more likely with ozanimod 1 mg than with placebo at week 32 (32.8% vs. 12.3%).

Adverse events occurred in 26.2% of patients in the ozanimod 1 mg arm, 11.1% in the ozanimod 0.5 mg arm and 32.0% in the placebo arm. Data were presented at the American College of Gastroenterology Annual Scientific Meeting.

Comment: Ozanimod is positioned to potentially become the first S1P receptor modulator to be approved for inflammatory bowel disease.