ABX464: HIV drug tested in ulcerative colitis

HIV studies have already shown the potent anti-inflammatory properties of ABX464, impacting the expression of miR124. In a first-in-disease phase 2a study, ABX464 was tested in patients with moderate-to-severe ulcerative colitis who were intolerant and/or refractory to existing treatments [1]. ABX464 50 mg once daily orally for 8 weeks was found to be safe and well tolerated. Clinical, endoscopy, histopathology, and biomarker analyses all showed consistent changes in favour of ABX464.

The study was performed in 32 patients from 15 European centres. A total of 29 (90.6%) patients (20 randomised to ABX464 and 9 to placebo) completed the induction study. The overall safety of ABX464 was very good, with no serious adverse events. The safety profile was similar to that seen in the clinical development in the HIV indication. The main efficacy results are presented in the Table.

Table: ABX464-101 study endpoints results after 56 days [1]



After the blinded induction phase, patients had the option to enrol in a 52-week, open-label, 50 mg once daily ABX464 study. The interim data from this maintenance study (n=22) shows further improvement in partial Mayo score and reduction in faecal calprotectin. This data supports a phase 2b multicentre, placebo-controlled, dose-ranging study in ulcerative colitis and a phase 2a study in Crohn’s disease.

1. Vermeire S, et al. ECCO 2019, OP21.

Posted on 9 May, 20199 April, 2024