Abstract
Abstract Background Obefazimod is an investigational, oral, once-daily (QD), small molecule which enhances expression of microRNA-124. Obefazimod demonstrated efficacy and safety in patients with moderate to severe active UC at week-8 in two double-blind, placebo-controlled induction trials and in subsequent open-label maintenance (OLM) studies [1-3], and a randomized controlled phase 3 study, ABTECT, is ongoing. Methods Patients with UC on obefazimod 50mg QD with endoscopic improvement (Mayo Endoscopic subscore = 0 or 1), could enrol in an open label study in which they were administered obefazimod 25mg QD for up to an additional 54 months. This interim analysis (cut-off date: September 11, 2024) reports the findings of patients who completed Weeks 48 (W48) and 96 (W96) visit (as observed analysis). Results Patients with UC were treated with obefazimod 50mg QD for approximately four years in the Phase 2a OLM study and for approximately two years in the Phase 2b OLM study. Out of 174 patients who completed these studies (10 in Phase 2a OLM, 164 in Phase 2b OLM), 138 were eligible. As of the cut-off date, 130 patients rolled over in the 25 mg OLM and were dosed. Among this cohort, 16 patients (12%) discontinued prior to W48 and 6 (5%) discontinued between W48 and W96. Overall, 113 patients (87%) completed the W48 visit, 74 (57%) completed the W96 visit. After dose de-escalation, rates of clinical remission at W48 and W96 were 84% (95/113) and 87% (64/74), respectively. At W48 and W96, 91% (103/113) and 91% (68/75) of patients were in symptomatic remission, respectively. Similar trends were observed with other efficacy analyses (Table 1). At W48, the levels of fecal calprotectin (FCP) were <250 and <150 µg/g for 90% (85/94) and 81% (76/94) of patients with available FCP values, respectively. At W96, 75% (49/65) of patients had FCP levels <250 µg/g, and 69% (45/65) had levels <150 µg/g. No new safety findings were identified over these 96 weeks. Conclusion Following 2-4 years of open-label treatment with obefazimod at a dose of 50 mg QD, large percentages of patients maintained efficacy measures on a lower maintenance dose of 25 mg od for two additional years. No new safety signals were observed over up to six years of obefazimod treatment, supporting a favorable long-term risk-benefit profile.
Published Version
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call