AN2 Therapeutics Initiates Pivotal Phase 2/3 Trial Evaluating Epetraborole for Treatment-Refractory MAC Lung Disease

AN2 Therapeutics, Inc. recently announced it has screened its first patient in its pivotal Phase 2/3 clinical trial evaluating once-daily, oral epetraborole for treatment-refractory Mycobacterium avium complex (MAC) lung disease, the most common form of nontuberculous mycobacterial (NTM) lung disease.

“Initiating this pivotal Phase 2/3 trial marks an important step toward delivering a new treatment option to patients with treatment-refractory MAC lung disease, an area which has seen little innovation,” said Eric Easom, President and Chief Executive Officer of AN2 Therapeutics. “As a once-daily oral therapy, epetraborole has the potential to become an important component of a multi-drug treatment regimen for patients with treatment-refractory MAC lung disease. We look forward to sharing clinical data from the Phase 2 portion of this trial in mid-2023, when we expect to get our first clinical readout on the impact of epetraborole added to a standard of care regimen.”

AN2 Therapeutics believes that compelling data from this pivotal Phase 2/3 clinical trial could allow the company to seek approval for epetraborole with the US FDA using the Limited Population Pathway for Antibacterial and Antifungal drugs (LPAD) and in other geographies. Under the LPAD pathway, the FDA may approve and review new antibacterial drugs to treat serious bacterial diseases in patients with an unmet medical need and for which effective bacterial drugs are limited or lacking, potentially resulting in a New Drug Application submission based on streamlined development. Epetraborole has been granted Qualified Infectious Disease Product and Fast Track status by the FDA for treatment-refractory MAC lung disease, as well as orphan drug designation for the treatment of infections caused by NTM.

This double-blind, placebo-controlled superiority trial uses an adaptive design to evaluate the safety and efficacy of epetraborole in patients with treatment-refractory MAC lung disease. In total, this multi-center trial is expected to enroll approximately 314 patients across North America, Europe, and potentially other geographies. The Phase 2 part of the trial (n=80) will assess the clinical response using various patient-reported outcome tools as well as to determine the safety, efficacy, and pharmacokinetics of epetraborole plus an optimized background regimen (OBR), when compared to a placebo plus an OBR. The Phase 2 findings will be utilized to inform the clinical response measures evaluated in the Phase 3 part of the trial and confirm the final sample size. The primary objective in the Phase 3 part of the trial (n≈ 234) will be to determine if epetraborole plus an OBR, consisting of two or more standard-of-care drugs, is superior to placebo plus an OBR. The company expects to report topline data from the Phase 2 part of the trial in mid-2023 and from the Phase 3 part in mid-2024, pending any sample size adjustments. For more information, visit www.clinicaltrials.gov (NCT05327803).

AN2 Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing treatments for rare, chronic, and serious infectious diseases with high unmet needs. AN2 is developing epetraborole, a once-daily, oral treatment with a novel mechanism of action for patients with NTM lung disease, a rare, chronic, and progressive infectious disease caused by bacteria, known as mycobacteria, that leads to irreversible lung damage and can be fatal.