Clin Gastroenterol Hepatol. 2022 Jan;20(1):116-125.e5.doi: 10.1016/j.cgh.2020.10.004. Epub 2020 Oct 9.

Jean-Frederic Colombel ¹, Mark T Osterman ², Andrew J Thorpe ³, Leonardo Salese ³, Chudy I Nduaka ³, Haiying Zhang ³, Nervin Lawendy ⁴, Gary S Friedman ³, Daniel Quirk ³, Chinyu Su ³, Walter Reinisch ⁵

Author information

¹Dr. Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York, New York.

²University of Pennsylvania, Perelman School of Medicine, Philadelphia, Pennsylvania.

³Pfizer Inc, Collegeville, Pennsylvania.

⁴Pfizer Inc, Collegeville, Pennsylvania. Electronic address: nervin.lawendy@pfizer.com.

⁵Medical University of Vienna, Vienna, Austria.

Abstract

Background & aims: Tofacitinib is an oral, small molecule Janus kinase inhibitor for the treatment of ulcerative colitis (UC). The efficacy and safety of tofacitinib in patients with moderate to severe UC, up to 1 year, have been reported. We investigated maintenance of efficacy in patients in remission after 52 weeks of maintenance treatment in the pivotal phase 3 study (OCTAVE Sustain); these patients received open-label, long-term treatment with tofacitinib 5 mg twice daily.

Methods: Patients with moderate to severe UC who completed a 52-week, phase 3 maintenance study (OCTAVE Sustain) were eligible to enroll into the ongoing, phase 3, multicenter, open-label, long-term extension (OCTAVE Open). We analyzed data from 142 patients who were in remission following tofacitinib treatment in OCTAVE Sustain who received tofacitinib 5 mg twice daily during OCTAVE Open. We assessed efficacy (including remission [based on total Mayo score], endoscopic improvement, clinical response, and partial Mayo score up to month 36 of OCTAVE Open) and safety data.

Results: After 12 months of tofacitinib 5 mg twice daily in OCTAVE Open, 68.3% of patients were in remission, 73.9% had endoscopic improvement, and 77.5% had a clinical response. At month 36, 50.4%, of the patients were in remission, 55.3% had endoscopic improvement, and 56.0% had a clinical response. The safety profile of tofacitinib 5 mg twice daily revealed no new safety risks associated with long-term exposure up to 36 months.

Conclusions: Efficacy endpoints were maintained for up to 36 months, regardless of prior tofacitinib dose, including patients who reduced from tofacitinib 10 mg to 5 mg twice daily upon OCTAVE Open entry. No new safety risks were identified. ClinicalTrials.gov: OCTAVE Sustain (NCT01458574); OCTAVE Open (NCT01470612).