AN2 Therapeutics, a clinical‑stage biopharmaceutical company focused on its boron‑based chemistry platform, has announced a Phase 2 proof‑of‑concept study of its oral drug epetraborole in adults with phlebotomy‑dependent polycythemia vera (PV). The study is slated to begin in the third quarter of 2026, with first data readouts expected in the fourth quarter of 2026 and additional results throughout 2027.
Epetraborole is a small‑molecule boron compound that inhibits bacterial leucyl‑tRNA synthetase. In healthy volunteers and non‑PV patients, the drug has produced dose‑dependent reductions in hematocrit, suggesting a mechanism that selectively targets red‑cell production. The company believes its oral delivery and red‑cell selectivity could provide a differentiated therapeutic profile for PV patients who currently rely on phlebotomy, low‑dose aspirin, hydroxyurea, interferon‑α, or JAK inhibitors.
The PV program expands AN2’s pipeline beyond its existing infectious‑disease and oncology programs. The company’s cash position was $88.6 million as of December 31, 2024, and it projects that this runway will fund operations through 2027 or 2028. AN2 has previously discontinued an epetraborole program for nontuberculous mycobacterial lung disease after a Phase 3 trial, making the PV indication a new avenue for the drug. The company now has three Phase 2 studies slated to start in 2026 and two preclinical oncology compounds expected to move into development this year.
"We believe epetraborole may offer a differentiated hematological profile that combines hematocrit control via red‑cell selectivity, early onset, titratability and oral delivery, attributes that could address key treatment objectives in polycythemia vera and offer patients a new therapeutic option where current approaches fall short," said Eric Easom, Co‑Founder, Chairman, President and CEO of AN2 Therapeutics. "This program creates additional, near‑term value inflection points within our current runway and broadens our pipeline, which now includes three Phase 2 studies initiating in 2026, and two preclinical oncology compounds that are expected to move into development this year."
Polycythemia vera is a chronic myeloproliferative neoplasm that elevates red‑blood‑cell mass and increases thrombotic risk. Current oral options are limited, and many patients require frequent phlebotomy. An oral agent that can reduce hematocrit through a novel mechanism would address a clear unmet need and could position AN2 as a contender in a market dominated by injectable or procedural therapies.
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