Phase III results with Xeljanz (tofacitinib) in ulcerative colitis published in New England Journal of Medicine- Pfizer

Pfizer announced that detailed results from the Phase III Oral Clinical Trials for Xeljanz (tofAcitinib) in ulceratiVE colitis (OCTAVE) clinical program were published in The New England Journal of Medicine (NEJM). Data from all three pivotal Phase III studies – OCTAVE Induction 1, OCTAVE Induction 2 and OCTAVE Sustain – met their respective primary endpoints, demonstrating that tofacitinib citrate was more effective than placebo in inducing and maintaining remission in patients with moderate to severe ulcerative colitis (UC). Remission was defined as a Mayo score* of 2 points or lower, with no individual subscore exceeding 1 point, and a rectal bleeding subscore of 0.1c.

In OCTAVE Induction 1, a statistically significant and greater proportion of patients receiving tofacitinib 10 mg BID (18.5%) were in remission at Week 8, compared to 8.2% receiving placebo. Similar results were observed in OCTAVE Induction 2, with 16.6% of patients receiving tofacitinib 10 mg BID achieving remission at Week 8, compared to 3.6% receiving placebo.

In OCTAVE Sustain, 34.3% and 40.6% of patients achieved remission at Week 52 with tofacitinib 5 mg BID and tofacitinib 10 mg BID, respectively, compared to 11.1% taking placebo. In addition, both doses of tofacitinib met the key secondary endpoints of the study, mucosal healing and sustained steroid-free remission among baseline remitters. Across tofacitinib 5 mg BID, tofacitinib 10 mg BID and placebo arms, mucosal healing was achieved by 37.4%, 45.7% and 13.1% of patients, respectively, and sustained steroid-free remission was achieved by 35.4%, 47.3% and 5.1%, respectively.

Across all studies, five tofacitinib-treated patients had adjudicated non-melanoma skin cancer, five had adjudicated cardiovascular events and there were increases in lipids with tofacitinib. There were two cases of malignancy in the control groups limited to one case of non-melanoma skin cancer and one case of invasive ductal breast carcinoma during OCTAVE Sustain.

See: "Tofacitinib as Induction and Maintenance Therapy for Ulcerative Colitis" William J. Sandborn et al. N Engl J Med 2017; 376:1723-1736 May 4, 2017 DOI: 10.1056/NEJMoa1606910