Tofacitinib for induction therapy in Japanese patients with active ulcerative colitis: Subgroup analyses in a multinational, Phase 3 clinical trial

Tofacitinib is an oral, small molecule JAK inhibitor that is being investigated for ulcerative colitis (UC). Subgroup analyses were performed in 62 Japanese patients (placebo, n=13) enrolled in a Phase 3, randomised, multinational, multicentre, double-bind, placebo-controlled study (OCTAVE 1, NCT01465763) investigating the efficacy/safety of tofacitinib 10 mg twice daily (BID) as induction therapy in patients with moderate to severe UC. Remission rates (total Mayo score<2, no subscore>1 and rectal bleeding subscore of 0) and rates of mucosal healing (endoscopic subscore by central reading of 1 or 0) at Week 8 were 22.4% and 30.6% with tofacitinib versus 7.7% and 15.4% with placebo. Efficacy was greater vs placebo in both TNF inhibitor (TNFi)-treated and TNFi-naïve patients. Increase from baseline of total inflammatory bowel disease questionnaire (IBDQ) score was greater with tofacitinib vs placebo. Almost all adverse events (AEs) were mild to moderate. Three serious AEs (C. difficile infection; UC/condition aggravation) were reported in 2 patients with tofacitinib. No deaths or malignancies were reported. Limitations include the small number of Japanese patients. However, for induction therapy, the results for efficacy and safety appear consistent with the Global population.