Clinical Development Program

Our lead DNA pol IIIC inhibitor, ibezapolstat, is ready to advance to international, Phase 3, pivotal registration trials in the US and EU -ready for oral treatment of acute C. difficile Infection. We’re also starting up a ground-breaking clinical trial with ibezapolstat in patients with multiply-recurrent CDI (rCDI) that has the potential to shift the treatment paradigm for acute CDI and rCDI.

The rCDI exploratory Phase 2 clinical trial is designed as an open-label study of up to 20 patients with at least 3 episodes of CDI over the past 12 months. Enrollment is anticipated to begin in the second half of 2026 and the trial is anticipated to take 12 months to fully enroll. If successful, this trial could potentially change the paradigm of treatment for CDI and rCDI from the current need for two therapies down to 1 therapy given that ibezapolstat would have the potential to treat acute CDI and prevent rCDI.

Acurx has completed its Phase 2b clinical trial of ibezapolstat compared to vancomycin, a standard of care to treat C. difficile infection or CDI.  The Phase 2b clinical trial was a randomized (1-to-1), non-inferiority, double-blind trial (n=32) of oral ibezapolstat compared to oral vancomycin.  Data was analyzed and topline efficacy results were reported in Q4 2023.

The Company successfully completed Phase 1 and Phase 2a clinical trials of ibezapolstat. The Phase 2a trial demonstrated 100% clinical cure at end of treatment and 100% sustained clinical cure in patients with C. difficile Infection (CDI), along with beneficial microbiome changes during treatment including overgrowth of Actinobacteria and Firmicutes phylum species while on therapy and new findings which demonstrate potentially beneficial effects on bile acid metabolism.

The Phase 1 clinical trial included a head-to-head comparison of oral ibezapolstat to oral vancomycin for impact on the microbiome and the results were exceptional, leading to the creation of a new exploratory endpoint in the Phase 2b clinical trial to continue to compare the impact on the microbiome between ibezapolstat and standard of care oral vancomycin.

We met with the FDA and EMA after analyzing the Phase 2b clinical trial data to finalize the size and scope of the Phase 3 clinical trial program and both regulatory bodies are aligned with our Phase 3 trial design and protocol. Regulatory precedent indicates that two Phase 3 trials of approximately 475 patients each may need to be conducted.

In June 2018, ibezapolstat was designated by the U.S. Food and Drug Administration (FDA) as a Qualified Infectious Disease Product (QIDP) for the treatment of patients with CDI and will be eligible to benefit from the incentives for the development of new antibiotics established under the Generating New Antibiotic Incentives Now (GAIN) Act. In January 2019, FDA granted "Fast Track" designation to ibezapolstat for the treatment of patients with CDI. The CDC has designated C. difficile as an urgent threat highlighting the need for new antibiotics to treat CDI.