AN2 Therapeutics commences Phase 3 Part of Phase 2/3 Clinical Trial Evaluating Epetraborole for Treatment-Refractory Mycobacterium avium complex (MAC) Lung Disease

AN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical-stage biopharmaceutical company focused on developing treatments for rare, chronic, and serious infectious diseases with high unmet needs, today announced that it has commenced the Phase 3 part of its Phase 2/3 clinical trial evaluating once-daily, oral epetraborole for treatment-refractory MAC lung disease after completing Phase 2 enrollment. The Phase 2/3 clinical trial is expected to support regulatory filings for approval in the U.S. and Japan. AN2 anticipates reporting Phase 2 topline data in summer 2024.

“Completing Phase 2 enrollment and initiating Phase 3 in this important clinical trial are significant milestones for AN2 and the patients we intend to serve. There is currently no approved oral therapy for MAC lung disease. Given epetraborole’s novel mechanism of action, encouraging clinical and non-clinical data to date, and convenient once daily oral dosing, we believe that if approved, epetraborole has the potential to become the backbone of therapy for patients suffering with often debilitating treatment-refractory MAC lung disease,” said Eric Easom, Co-Founder, President and Chief Executive Officer of AN2 Therapeutics. “We look forward to sharing topline results from the Phase 2 portion of the trial in summer 2024, at which time we expect to be well on our way to completing Phase 3 enrollment. We will provide more information on the projected Phase 3 enrollment timeline as we get closer to the Phase 2 topline readout.”

The FDA has granted epetraborole Fast Track designation for treatment-refractory MAC lung disease, Qualified Infectious Disease Product designation for treatment-refractory MAC lung disease, and orphan drug designation for the treatment of infections caused by nontuberculous mycobacteria (NTM).

About the Epetraborole Pivotal Phase 2/3 Trial (EBO-301)

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. The Phase 2 part of the trial (n=80) assesses clinical response using various patient-reported outcome tools as well as 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 Phase 3 (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. For more information, please visit: www.clinicaltrials.gov (NCT05327803).