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AN2 Therapeutics Announces Plans to Advance Oral Epetraborole into Phase 2 Study for Polycythemia Vera (PV)
Substantial clinical evidence provides rationale to initiate Phase 2 study in PV Phase 2 study expected to begin 3Q26 with potential for data readouts as
About this update from An2 Therapeutics, Inc.
[{"type":"text","content":"\nSubstantial clinical evidence provides rationale to initiate Phase 2 study in PV\n\n\nPhase 2 study expected to begin 3Q26 with potential for data readouts as early as 4Q26 and throughout 2027\n\n\nAN2 to host webcast on Wednesday, March 4, 2026 at 9:30am ET/ 6:30am PT\n\n\n MENLO PARK, Calif.--(BUSINESS WIRE)--\nAN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical-stage biopharmaceutical company developing novel small molecule therapeutics derived from its boron chemistry platform, today announced its plans to expand the development of oral epetraborole into a Phase 2 proof-of-concept clinical study in adults with phlebotomy-dependent polycythemia vera (PV). PV is a blood cancer characterized by overproduction of red blood cells in the bone marrow. This overproduction increases hematocrit which can lead to serious medical complications, including arterial and venous thromboembolic events.\n\n\nThe Company’s decision to pursue PV is supported by data from multiple clinical trials of oral epetraborole in healthy volunteers and non-PV patients in which the drug consistently demonstrated early, controlled, sustained, and dose-dependent reductions in hematocrit at potentially clinically meaningful levels for PV.\n\n\nEpetraborole’s effects were characterized by the following pharmacodynamic and clinical observations:\n\n\n\nConsistent hematocrit reductions across multiple clinical populations, including healthy volunteers and nontuberculous mycobacterial (NTM) lung disease patients, with effects sustained over a six-month treatment period\n\n\n\n\nEarly onset of hematocrit reduction after dose initiation with durable, stable control and reversibility at treatment cessation\n\n\n\n\nNo clinically relevant change in white blood cell counts and minimal change in platelet counts\n\n\n\n\nDemonstrated durable hematocrit reduction in 9-month chronic non-human primate studies\n\n\n\n\nA potentially differentiated mechanism of action, likely acting on globin synthesis rather than directly on heme synthesis\n\n\n\nEpetraborole has been generally well tolerated in clinical trials to date at doses anticipated for the treatment of PV.\n\n\n“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 ...