OP006 Apremilast for active ulcerative colitis: a phase 2, randomised, double-blind, placebo-controlled induction study
S. Danese1*, M. Neurath2, A. Kopon3, S. Zakko4, T. Simmons5, R. Fogel6, J. Maccarone7, X. Zhan7, K. Usiskin7, D. Chitkara7
1Instituto Clinico Humanitas, Milan, Italy, 2University Erlangen, Nurnberg, Germany, 3Torunskie Centrum Gastrologiczne Gastromed, Torun, Poland, 4Connecticut Clinical Research Foundation of Bristol Hospital, Bristol, USA, 5West Gastroenterology Medical Group, Los Angeles, USA, 6Clinical Research Institute of Michigan, Chesterfield, USA, 7Celgene Corporation, Summit, USA
Apremilast is an oral small-molecule inhibitor of phosphodiesterase 4 that works intracellularly to modulate a wide array of pro- and anti-inflammatory mediators in ulcerative colitis (UC).
Patients with active UC (defined as a total Mayo score [TMS] of ≥6 to ≤11, with a Mayo endoscopic score [MES] of ≥2) who failed at least one conventional therapy for UC and were naïve to biologic therapy were randomised in a 1:1:1 ratio to receive apremilast 30 mg BID (APR30), apremilast 40 mg BID (APR40), or placebo (PBO) for up to 12 weeks. The primary endpoint of the study was TMS clinical remission at Week 12. Endoscopy was read centrally by independent experts blinded to treatment allocation and time point.
A total of 170 patients were randomised to PBO (
In this 12-week, phase 2 study, patients with active UC treated with APR30 had clinically meaningful improvements in symptoms, endoscopy, biomarkers, and mucosal healing compared with PBO. The observed adverse events were consistent with those expected in UC patients and were also consistent with the known safety profile of apremilast.