J Crohns Colitis. 2021 Jul 5;15(7):1120-1129. doi: 10.1093/ecco-jcc/jjab012.

William J Sandborn ¹, Brian G Feagan ², Stephen Hanauer ³, Severine Vermeire ⁴, Subrata Ghosh ⁵, Wenzhong J Liu ⁶, AnnKatrin Petersen ⁶, Lorna Charles ⁶, Vivian Huang ⁶, Keith Usiskin ⁶, Douglas C Wolf ⁷, Geert D'Haens ⁸

Author information

¹Division of Gastroenterology, University of California San Diego, La Jolla, CA, USA.

²Western University, London, Ontario, Canada.

³Feinberg School of Medicine, Chicago, IL, USA.

⁴University of Leuven, Leuven, Belgium.

⁵University of Calgary, Alberta, Canada.

⁶Bristol Myers Squibb, Princeton, NJ, USA.

⁷Center for Crohn's Disease & Ulcerative Colitis, Atlanta Gastroenterology Associates, Atlanta, GA, USA.

⁸Inflammatory Bowel Disease Center, Amsterdam University Medical Center, Amsterdam, the Netherlands.

Abstract

Background and aims: This analysis examined the long-term safety and efficacy of ozanimod in patients with moderately to severely active ulcerative colitis [UC] with ≥ 4 years of follow-up in the phase 2 TOUCHSTONE open-label extension [OLE].

Methods: Patients receiving placebo or ozanimod HCl 0.5 mg or 1 mg during the double-blind period could enter the OLE [ozanimod HCl 1 mg daily]. Partial Mayo score [pMS] clinical response and remission were assessed through OLE week 200 and summarized descriptively using observed cases [OC] and non-responder imputation [NRI]. Endoscopy was required at OLE week 56 and the end of treatment. Parameters associated with endoscopy were summarized at weeks 56 and 104 [OC], and week 56 [NRI]. C-reactive protein and faecal calprotectin were assessed. Adverse events were monitored throughout the study.

Results: Of 197 patients receiving double-blind treatment, 170 entered the OLE. Discontinuation rates were 28% at year 1 and 15-18% annually through year 4. Partial Mayo measures indicated clinical response and remission rates at OLE week 200 of 93.3% and 82.7%, respectively, using OC and 41% and 37% with the more conservative NRI analysis. At weeks 56 and 104, respectively, histological remission rates were 46.3% and 38.5%, and endoscopic improvement rates were 46.4% and 46.5% [OC]. No new safety signals were identified during ≥ 4 years of follow-up.

Conclusions: There was a high rate of continued study participation and long-term benefit with ozanimod HCl 1 mg daily based on clinical, histological and biomarker measures in patients with moderately to severely active UC in the TOUCHSTONE OLE.