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AN2 Therapeutics Reports Positive Enabling Data Supporting Advancement of Oral AN2-502998 to Phase 2 Study for Chronic Chagas Disease Parasite elimination achieved with 28-day treatment in 100% of nonhuman primates (NHPs

Key Takeaway: AN2 Therapeutics announced positive results from studies on their oral therapeutic, AN2-502998, which targets chronic Chagas disease. The treatment achieved complete parasite elimination in 100% of nonhuman primates over a 28-day period, with a favorable safety profile demonstrated in Phase 1 human trials. The company is moving forward with plans to initiate a Phase 2 study, aiming to address the unmet medical need for an effective, well-tolerated treatment for this disease that affects millions globally. The promising data supports their goal of developing the first FDA-approved therapy for chronic Chagas disease in adults.

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POSITIVE FACTORS

  • 100% parasite elimination achieved in treated nonhuman primates.
  • Favorable safety profile observed in Phase 1 First-In-Human study.
  • Potential for AN2-502998 to be the first FDA-approved therapy for chronic Chagas disease.
  • Significant commercial opportunity with potential multi-billion-dollar market.

Full Press Release Details

AN2 Therapeutics Reports Positive Enabling Data Supporting Advancement of
Oral AN2-502998 to Phase 2 Study for Chronic Chagas Disease
Parasite elimination achieved with 28-day treatment in 100% of nonhuman primates (NHPs)
naturally infected with Trypanosoma cruzi (T. cruzi) at target exposures planned in humans
Phase 1 First-In-Human (FIH) study showed favorable safety
and tolerability profile; no dose-limiting toxicities
Significant U.S. commercial opportunity, including Priority Review Voucher
eligibility upon approval
Menlo Park, CA - June 4, 2026 - AN2 Therapeutics, Inc. (Nasdaq: ANTX) a clinical-stage biopharmaceutical
company developing novel small molecule therapeutics derived from its boron chemistry platform, today announced positive results from two studies evaluating the Company's oral CPSF3 inhibitor,
AN2-502998, for the treatment of chronic Chagas disease (American trypanosomiasis) caused by infection with the parasite T. cruzi.
Key findings from the two studies:
"Results from these studies converge
on the picture we were hoping to see: efficacy in NHPs at an exposure level achievable in humans, with an excellent safety profile as shown in the FIH study. Notably, parasites were eliminated after one month of treatment in NHPs with the same
naturally acquired, chronic infection that we see in humans," said Eric Easom, Co-founder, Chairman, President and CEO of AN2 Therapeutics. "Together, these data support our goal of making AN2-502998 the first FDA-approved therapy for chronic Chagas disease in adults."
Easom continued, "We believe an oral therapy capable of delivering high rates of parasitic cure after just one month of treatment could enable
large-scale test-and-treat campaigns against this often lethal disease, which affects over 300,000 people in the U.S. and approximately 10 million globally. We look
forward to initiating a Phase 2 proof-of-concept study late this year."
"Chagas disease is hiding in plain sight. We have simple, inexpensive serologic tests, and we know
exactly which populations are at risk, no different from where we were with hepatitis C or HIV before systematic screening became standard of care," said Dr. David Hamer, Professor of Global Health and Medicine, Boston University Schools
of Public Health and Medicine. "The patients are there; it's a matter of clinical awareness and systematic screening. What has been missing is an effective, well-tolerated treatment that physicians and patients can rely on for adults
with chronic Chagas disease, who represent the vast majority of patients. It is genuinely encouraging to see serious drug development in this space - chronic Chagas disease represents one of the highest unmet needs in infectious disease, and these
data suggest we may finally be moving toward a meaningful therapeutic option for patients who have had too few for too long."
Chagas disease (also known as American trypanosomiasis) is caused by the parasite 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. The parasite T. cruzi silently damages the heart and
digestive system, with ~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.
Chagas Disease Opportunity
The Company believes that Chagas disease represents a potential multi-billion-dollar global market opportunity globally, given its large and readily treatable
patient population. Well-established risk profiles including country of birth, travel history, and exposure to triatomine vectors enable targeted, cost-effective screening, and low-cost diagnostics are widely
available. The commercial profile of Chagas disease therapeutics shares key structural characteristics with that of other large infectious disease markets, most notably hepatitis C, for which accessible diagnostics, defined risk populations, high
urgency to treat, and broad insurance coverage transformed a historically underdiagnosed condition into a substantial market. In parallel, through its work with the Drugs for Neglected Diseases initiative (DNDi), the Company is committed to ensuring
global access to AN2-502998, should it eventually be approved, in Chagas-endemic developing regions. AN2-502998, if approved, would also be eligible for an FDA Tropical
Disease Priority Review Voucher.
Key Findings: 28-Day Nonhuman Primate (NHP) Study
The NHP efficacy study evaluated AN2-502998 administered for 28 days in macaques with naturally-acquired, chronic T.
cruzi infection, contracted via triatomine (kissing bug) vectors in their natural habitats, the same vector that transmits the parasite to humans. The Chagas disease vector is increasingly prevalent across the southern half of the U.S. The
Company believes that efficacy in naturally infected NHPs is the most clinically relevant predictor of efficacy for human chronic Chagas disease.
Key Findings: Phase 1 First-in-Human Study
The Phase 1 FIH study (NCT07024589) evaluated single ascending oral doses of AN2-502998 and then multiple ascending doses administered over 10 days in healthy adult volunteers:
AN2-502998 is a boron-based small molecule therapeutic candidate from the benzoxaborole class, which has a broad
therapeutic profile and includes two FDA-approved drugs (crisaborole and tavaborole). AN2-502998 is an orally active inhibitor of CPSF3 in T. cruzi. CPSF3 is an
essential enzyme involved in messenger RNA processing, is clinically validated, and the same target as acoziborole that was recently approved by the EMA for the treatment of human African trypanosomiasis (HAT), caused by a related trypanosome
AN2 Therapeutics is advancing AN2-502998 into Phase 2 clinical study in collaboration with DNDi, a non-profit research and development organization whose medical expertise, established presence in Chagas-endemic regions, and commitment to delivering accessible treatments for neglected diseases provides a critical
foundation for both the timely execution of clinical development and ensuring broad global access upon approval. The program also benefits from ongoing collaboration with the University of Georgia Research Foundation and the laboratory of Prof. Rick
Tarleton, whose pioneering work in T. cruzi biology has been instrumental in advancing the scientific understanding of Chagas disease.
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 oncology 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
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases, you can
identify forward-looking statements by terms such as "may," "will," "should," "would," "expect," "plan," "anticipate," "could," "intend,"
"target," "project," "believe," "estimate," "predict," "potential," or "continue," or the negative of these terms or other similar expressions. Forward-looking
statements expressed or implied in this press release include, but are not limited to, statements regarding: the potential of the Company's boron chemistry platform and advancement of the Company's development programs; NHP study data as
a predictor of efficacy outcomes in human trials; market size and sales potential; the commercial profile of Chagas disease therapeutics; advancement of AN2-502998 into Phase 2 in collaboration with DNDi and
other ongoing collaborations; expectations regarding the Company's clinical trials; the predictivity of data; and other statements that are not historical fact. These statements are based on AN2's current estimates, expectations, plans,
objectives, and intentions, are not guarantees of future performance, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking
statements as a result of these risks and uncertainties, which include, but are not limited to, risks and uncertainties related to: AN2's ability to implement its plans for its internal boron chemistry platform and pipeline programs; timely
enrollment of patients in AN2's clinical trials and investigator-initiated clinical trials; AN2's ability to procure sufficient supply of its product candidates for its clinical trials; the potential for results from clinical trials to
differ from preclinical, early clinical, preliminary, or expected results; the ability of particular preclinical models in non-human primates to predict safety and efficacy in humans; significant adverse
events, toxicities, or other undesirable side effects associated with AN2's product candidates; the significant uncertainty associated with AN2's product candidates ever receiving any regulatory approvals; AN2's ability to obtain,
maintain, or protect intellectual property rights related to its current and future product candidates; implementation of AN2's strategic plans for its business and product candidates; the sufficiency of AN2's capital resources and need
for additional capital to achieve its goals; global macroeconomic conditions and global conflicts and other risks, including those described under the heading "Risk Factors" in AN2's Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and other reports filed with the U.S. Securities and Exchange Commission (SEC). These filings, when made, are available on the investor
relations section of AN2's website at www.an2therapeutics.com and on the SEC's website at www.sec.gov. Forward-looking statements contained in this press release are made as of this date, and AN2 undertakes no duty to update such
information except as required under applicable law.
Chief Financial Officer
INVESTOR AND MEDIA CONTACT:

Frequently Asked Questions

What is AN2-502998 used to treat?

AN2-502998 is developed for treating chronic Chagas disease caused by T. cruzi.

What were the results of the Phase 1 study?

The Phase 1 study showed favorable safety and tolerability, with no dose-limiting toxicities.

How effective was AN2-502998 in animal studies?

In nonhuman primates, 100% parasite elimination was achieved after 28 days of treatment.

What is the market opportunity for Chagas disease treatments?

Chagas disease presents a multi-billion-dollar global market opportunity due to its prevalence.

When will the Phase 2 study for AN2-502998 begin?

The Phase 2 proof-of-concept study is expected to start later this year.

Last updated: Jun 4, 2026