AN2 Therapeutics announced that the U.S. Food and Drug Administration has cleared an investigational new drug application to conduct a 90‑patient, investigator‑initiated trial of its oral antibiotic epetraborole in patients with Mycobacterium abscessus lung disease.
The study, led by Dr. Kevin Winthrop at Oregon Health & Science University, will enroll treatment‑naïve patients across 10–15 U.S. sites and will evaluate safety, efficacy and pharmacokinetics. The trial is a key step toward establishing human proof of concept for epetraborole, which could become the first FDA‑approved oral therapy for this difficult‑to‑treat infection.
The U.S. population with nontuberculous mycobacterial (NTM) lung disease is estimated at 120,000–150,000, with 10–15% caused by M. abscessus. A positive safety and efficacy signal would open a sizable market for an oral treatment and would validate AN2’s boron‑based platform for future development.
AN2 previously conducted a Phase 2/3 study (EBO‑301) of epetraborole in treatment‑refractory MAC lung disease. Topline results from the Phase 2 portion were disappointing, leading to the termination of both the Phase 2 and Phase 3 parts, although the drug was generally well‑tolerated. This prior outcome adds caution to the current development and underscores the need for robust clinical data in the M. abscessus indication.
CEO Eric Easom said the IND clearance is a “significant milestone” and that epetraborole has the potential to be the first FDA‑approved therapy for M. abscessus. Dr. Winthrop emphasized the current reliance on burdensome, off‑label intravenous regimens and the pressing need for an effective oral treatment, noting that epetraborole could address this unmet need.
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