AN2 Therapeutics Advances Three Phase 2 Programs as Boron Chemistry Pipeline Expands
核心洞察
AN2 Therapeutics expects to have three Phase 2 programs underway by the end of 2026, spanning polycythemia vera (搜索), M. abscessus lung disease (搜索), and chronic Chagas disease (搜索).
The Phase 2 EBO-PV-201 study of oral epetraborole in phlebotomy-dependent polycythemia vera (搜索) will expand to U.S. and Australian sites, with enrollment anticipated to begin in Q4 2026.
Positive non-human primate efficacy and favorable Phase 1 data support planned Phase 2 initiation of AN2-502998 in chronic Chagas disease (搜索) by year-end.
AN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical-stage biopharmaceutical company focused on novel small-molecule therapeutics derived from its boron chemistry platform, reported financial results for the second quarter ended June 30, 2026, alongside updates on three Phase 2 programs expected to be active by year-end.
"AN2 will have three Phase 2 programs underway by the end of this year, all of which have the potential to address major unmet needs. Our near-term focus is advancing start-up activities for the Phase 2 EBO-PV-201 study in polycythemia vera (搜索). We recently held a pre-IND meeting with the FDA and are expanding the Phase 2 study to include sites in the U.S. and Australia," said Eric Easom, Co-Founder, Chairman, President and CEO of AN2 Therapeutics.
Polycythemia Vera Program Expands to Global Trial
In March 2026, the company outlined plans to expand development of oral epetraborole into a Phase 2 proof-of-concept study in adults with phlebotomy-dependent polycythemia vera (搜索) (PV). PV is a slowly progressing blood cancer characterized by overproduction of red blood cells in the bone marrow, which increases hematocrit and can lead to serious complications including arterial and venous thromboembolic events. If untreated, PV can be life-threatening. Despite available therapies such as burdensome periodic therapeutic phlebotomies, many patients experience uncontrolled hematocrit levels and persistent symptoms. PV is estimated to affect approximately 155,000 people in the U.S.
Following a pre-IND meeting with the FDA, the company now plans to expand the Phase 2 study (EBO-PV-201) to add sites in the U.S. and Australia, with an IND filing expected in the third quarter of 2026. As a result of this expansion, Phase 2 enrollment is anticipated to commence in the fourth quarter of 2026, beginning with an open-label sentinel cohort at a sub-therapeutic dose aimed at assessing pharmacokinetics and safety in PV patients. Following successful conclusion of the sentinel group, a safety monitoring committee will advise on dose selections for Part 1, an open-label, single-arm, 28-week evaluation of epetraborole's ability to maintain hematocrit control and reduce the frequency of phlebotomy. The company anticipates releasing Part 1 data periodically throughout 2027.
M. abscessus Lung Disease Trial Enrolling
Enrollment is ongoing in a Phase 2 investigator-initiated trial of epetraborole in patients with M. abscessus lung disease (搜索), one of the most difficult-to-treat non-tuberculous mycobacterial (NTM) infections for which no FDA-approved therapy exists. Building on microbiological and safety data from AN2's prior NTM study in treatment-refractory MAC, the company believes epetraborole has the potential to address a critical unmet need in this indication.
M. abscessus lung disease (搜索) is a serious NTM infection requiring prolonged therapy, initially often with IV-only antibiotics. It is estimated that approximately 120,000–150,000 people in the U.S. are living with NTM lung disease, of whom 10–15% have infection caused by M. abscessus. The 84-patient multicenter, randomized, double-blind, placebo-controlled, prospective clinical study is led by Dr. Kevin Winthrop, Professor of Public Health and Infectious Diseases at the Oregon Health and Sciences University, in conjunction with other investigators across an estimated 10–15 sites in the U.S. The company anticipates reporting topline results in late 2027, subject to enrollment progress.
Chronic Chagas Disease Program Advances Toward Phase 2
The company is studying AN2-502998, an oral, boron-based small molecule CPSF3 (搜索) inhibitor for the treatment of chronic Chagas disease (搜索), also known as American trypanosomiasis. Chagas disease is caused by the parasite Trypanosoma cruzi (T. cruzi). Over 300,000 people are estimated to be infected in the U.S., 200,000 across Europe and Japan, and about 10 million worldwide. Left untreated, chronic T. cruzi infection is lifelong and can be life-threatening, with approximately 20–30% of people developing serious cardiac damage resulting in heart failure, stroke, or sudden death. There are no FDA-approved treatments for adults with Chagas disease.
In June 2026, the company announced positive results from two studies supporting planned initiation of a Phase 2 trial. In a non-human primate (NHP) efficacy study, 28 days of treatment with AN2-502998 resulted in 100% parasitic elimination at target exposures attainable in humans, in NHPs with naturally acquired, chronic T. cruzi infection. In the Phase 1 first-in-human study, AN2-502998 was generally well tolerated at exposure levels consistent with NHP efficacy thresholds. AN2-502998 is the only compound of which the company is aware to have demonstrated curative activity in preclinical studies across multiple species, including NHPs with long-term, naturally acquired chronic infections caused by diverse T. cruzi genetic types. The company expects to initiate a Phase 2 proof-of-concept study in adults with chronic Chagas disease (搜索) in late 2026.
Boron Chemistry Pipeline Expansion
The company is prioritizing targets in oncology and bone disorders where boron chemistry may offer a competitive advantage, including initially ENPP1 (搜索) and PI3Kα (搜索). The unique binding modes of boron-containing compounds enable discovery of inhibitors with high ligand efficiency against targets considered undruggable or difficult to access with traditional chemistry approaches. Boron chemistry has produced first-in-class molecules against targets including CPSF3 (搜索) (AN2-502998 and acoziborole) and LeuRS (搜索) (epetraborole, ganfeborole and tavaborole). Earlier this year, the company declared a development candidate (ENPP1) for the treatment of solid tumors and expects to advance a second development candidate by the end of 2026.
Second Quarter Financial Results
Research and development expenses for the second quarter of 2026 were $6.0 million, compared to $3.2 million for the same period in 2025, driven by increased chemistry manufacturing and controls (CMC) expenses, consulting and outside services, preclinical and research studies, and clinical trial expenses, partially offset by a decrease in personnel-related expenses. General and administrative expenses were $2.9 million, compared to $4.0 million in the prior-year period. Net loss for the second quarter of 2026 was $8.2 million, compared to $6.5 million for the same period in 2025.
The company had cash, cash equivalents and investments of $79.9 million at June 30, 2026, and projects that existing resources will sustain operations into 2029 under the current operating plan.