Long-Term Efficacy and Safety of Ozanimod in Moderately to Severely Active Ulcerative Colitis: Results From the Open-Label Extension of the Randomized, Phase 2 TOUCHSTONE Study

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


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. [NCT02531126].

Keywords: Ozanimod; clinical trial; ulcerative colitis.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial

MeSH terms

  • Adult
  • C-Reactive Protein / metabolism
  • Colitis, Ulcerative / drug therapy*
  • Colonoscopy
  • Double-Blind Method
  • Feces / chemistry
  • Female
  • Humans
  • Indans / therapeutic use*
  • Leukocyte L1 Antigen Complex / metabolism
  • Male
  • Oxadiazoles / therapeutic use*
  • Remission Induction
  • Sphingosine 1 Phosphate Receptor Modulators / therapeutic use*


  • Indans
  • Leukocyte L1 Antigen Complex
  • Oxadiazoles
  • Sphingosine 1 Phosphate Receptor Modulators
  • C-Reactive Protein
  • ozanimod

Associated data

  • ClinicalTrials.gov/NCT02531126