Acurx says Phase 2 ibezapolstat trial met primary objective

Acurx Pharmaceuticals announced efficacy results from the Phase 2 clinical trial of ibezapolstat for the treatment of C. difficile Infection. The company previously announced that it decided to terminate the Phase 2b vancomycin-controlled trial segment early based on aggregate blinded data showing a high observed clinical cure rate with no emerging safety concerns. The company made this decision in consultation with its medical and scientific advisors based on the compelling clinical observation that the clinical cure rate for ibezapolstat was projected to be at least 90% pooled across the open-label Phase 2a and the blinded Phase 2b segments with no safety concerns noted. Other factors also influenced the decision, including the cost of maintaining clinical trial sites and the challenging enrollment environment in the U.S. due to the COVID-19 pandemic and its aftermath. The Phase 2b trial was originally designed to be a non-inferiority trial and later amended to include an interim efficacy analysis with review by an Independent Data Monitoring Committee. The decision to end the trial early based on blinded clinical observations obviated the need for an interim analysis, IDMC review, and NI assessment. The Company determined, in consultation with its clinical and statistical experts, that presenting clinical cure rates for the primary efficacy endpoint is the most appropriate representation for the clinical activity of ibezapolstat in treating CDI. The overall observed Clinical Cure rate in the combined Phase 2 trials in patients with CDI was 96%, based on 10 out of 10 patients in Phase 2a in the Modified Intent to Treat Population, plus 15 out of 16 patients in Phase 2b in the Per Protocol Population, who experienced Clinical Cure during treatment with ibezapolstat. Ibezapolstat was well-tolerated, with three patients each experiencing one mild adverse event assessed by the blinded investigator to be drug-related. All three events were gastrointestinal in nature and resolved without treatment. There were no drug-related treatment withdrawals or no drug-related serious adverse events, or other safety findings of concern. In the Phase 2b vancomycin control arm, 14 out of 14 patients experienced clinical cure. The Company is confident that based on the pooled Phase 2 ibezapolstat clinical cure rate of 96% and the historical vancomycin cure rate of approximately 81%, we will demonstrate non-inferiority of ibezapolstat to vancomycin in Phase 3 trials in accordance with the applicable FDA Guidance for Industry.