Oral epetraborole program in polycythemia vera advancing toward Phase 2
Phase 2 investigator-initiated trial with epetraborole for M. abscessus lung disease underway
Data expected in 2Q/2026 from two studies of oral AN2-502998, under development for chronic Chagas disease: a Phase 1 first-in-human clinical trial and a non-human primate efficacy study
Declared ENPP1 candidate for solid tumors advancing from early research into development
MENLO PARK, Calif.--(BUSINESS WIRE)--May 11, 2026--
AN2 Therapeutics, Inc. (Nasdaq: ANTX), a clinical stage biopharmaceutical company focused on the discovery and development of novel small molecule therapeutics derived from its boron chemistry platform, today reported financial results for the first quarter ended March 31, 2026.
"This was an important quarter for AN2 as we announced an exciting new program in polycythemia vera that is planned to enter Phase 2 development in the third quarter of 2026. This program is supported by what we believe is a robust package of epetraborole preclinical and clinical enabling data. We commenced an investigator-initiated Phase 2 study in M. abscessus lung disease, completed dosing in a Phase 1 study for our Chagas disease program, and declared our ENPP1 candidate for the treatment of solid tumors, as well as strengthened our balance sheet," said Eric Easom, Co-Founder, Chairman, President and CEO of AN2 Therapeutics. "Looking ahead, we expect to advance three programs into Phase 2 development this year and report multiple data readouts within our cash runway into 2029. I couldn't be more excited about our progress and the potential to substantially improve the lives of patients across our diverse portfolio that is enabled by our boron chemistry pipeline."
First Quarter & Recent Business Updates:
Polycythemia vera
-- Advancing oral epetraborole into a Phase 2 trial for polycythemia vera
In March 2026, the Company outlined 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. 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, requiring long-term management to maintain adequate disease
control. PV is estimated to affect approximately 155,000 people in the
U.S. The Company is proceeding through the regulatory process and
plans to initiate the Phase 2 clinical trial in the third quarter of
2026. Subject to regulatory clearance and enrollment progress, the
Company expects to provide periodic data readouts beginning as early as
the fourth quarter of 2026 and throughout 2027.
M. abscessus complex lung disease
-- Commenced Phase 2 investigator-initiated clinical trial of epetraborole
in patients with M. abscessus lung disease Building on the
microbiological and safety data from AN2's prior non-tuberculous
mycobacterial (NTM) study, the Company believes that epetraborole has the
potential to address a critical unmet need in M. abscessus lung disease,
one of the most difficult-to-treat NTM infections and one for which no
FDA-approved therapy exists. M. abscessus lung disease is a serious NTM
infection requiring prolonged therapy, initially often with IV-only
antibiotics. People affected by this illness face limited, burdensome
treatment options and high rates of morbidity and 5-year mortality. NTM
lung disease represents a growing global health concern. It is estimated
that approximately 120,000--150,000 people in the U.S. are living with
NTM lung disease, of which 10-15% are caused by M. abscessus. The
Company is supporting an investigator-initiated trial and anticipates
that data from this study, if positive, could provide clinical
proof-of-concept in M. abscessus lung disease and thereby inform the
design of a subsequent pivotal trial. Patient screening commenced in
March 2026. The multicenter, randomized, double-blind, placebo-controlled,
prospective clinical study is being 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 regulatory clearance and
enrollment progress.
Chagas disease
-- Oral AN2-502998: Phase 1 first-in-human clinical trial and non-human
primate data expected 2Q/2026; Phase 2 proof-of-concept study planned to
initiate in 2026 pending results of Phase 1 study In March 2026, the
Company completed dosing all cohorts in the Phase 1 first-in-human trial
of oral AN2-502998, an investigational, boron-based small molecule in
development for the treatment of chronic Chagas disease, or American
trypanosomiasis. In the second quarter of 2026, the Company anticipates
reporting Phase 1 data from this study, as well as results from a
non-human primate $(NHP)$ study that tested the curative potential of
AN2-502998 with a 28-day dosing duration. AN2-502998 is the only compound
of which the Company is aware to have demonstrated curative activity in
preclinical studies across multiple species, including in NHPs with
long-term, naturally acquired, chronic infections caused by diverse T.
cruzi genetic types. Because NHP infections are naturally acquired in the
environment, these efficacy data may be more predictive of efficacy in
human clinical trials than other animal models. The Company expects to
initiate a Phase 2 proof-of-concept study in adults with chronic Chagas
disease later in 2026, pending results of the Phase 1 study.
Boron chemistry pipeline
-- Declared ENPP1 candidate for the potential treatment of solid tumors
The Company is prioritizing targets in oncology and bone disorders for
which boron chemistry may offer a competitive advantage in terms of
binding-site differentiation, pharmacodynamics, drug-like properties, and
intellectual property, including initially ENPP1 and PI3K<ALPHA>. The
unique binding modes of boron-containing compounds enable the 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 a number of
targets including CPSF3 (AN2-502998 and acoziborole) and LeuRS
(epetraborole, ganfeborole and tavaborole). The Company has discovered
preclinical compounds with profiles that are sub-nanomolar, highly
selective and characterized by excellent oral pharmacokinetics. The
Company recently declared its ENPP1 candidate for the treatment of solid
tumors, marking an important step in transitioning the program from early
research into development.
Selected First Quarter Financial Results
-- Research and Development (R&D) Expenses: R&D expenses for the first
quarter of 2026 were $6.7 million, compared to $7.7 million for the same
period during 2025 due to decreased chemistry manufacturing and controls
$(CMC)$ expenses, primarily due to decreased expenses related to completion
of CMC activities in certain programs, and license fees. These decreases
were partially offset by increases in personnel-related expenses,
clinical trial expenses, consulting and outside services, and facilities
and other expenses.
-- General and Administrative (G&A) Expenses: G&A expenses for the first
quarter of 2026 and the same period during 2025 were $3.8 million.
-- Interest Income: Interest income for the first quarter of 2026 was $0.5
million, compared to $0.9 million for the same period during 2025 due to
lower average cash, cash equivalents and investment balances and lower
interest rates in 2026 as compared to 2025.
-- Net Loss: Net loss for the first quarter of 2026 was $10.0 million,
compared to $10.6 million for the same period during 2025.
-- Cash Position: The Company had cash, cash equivalents and investments
of $85.3 million at March 31, 2026. In March 2026, AN2 raised gross
proceeds of $40.0 million through a private placement, before deducting
placement agent fees and other expenses. The Company projects that
existing cash, cash equivalents, and investments will sustain operations
into 2029 under the current operating plan.
About AN2 Therapeutics, Inc.
AN2 Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on the discovery and development of novel small-molecule therapeutics derived from our boron chemistry platform. Our development pipeline spans hematologic diseases, infectious diseases, and oncology with three Phase 2 studies expected to be active in 2026, two preclinical candidates, as well as advanced research programs focused on targets in oncology, bone disorders, and infectious diseases. We are committed to delivering high-impact drugs to patients that address critical unmet needs and improve health outcomes.
Forward-Looking Statements
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