\nAN2 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\u2019s oral CPSF3 inhibitor, AN2-502998, for the treatment of chronic Chagas disease (American trypanosomiasis) caused by infection with the parasite T. cruzi.<\/i><\/p>\n
\nKey findings from the two studies:<\/p>\n
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- \nIn NHPs with naturally acquired, chronic T. cruzi<\/i> parasite infection, 28 days of AN2-502998 treatment resulted in 100% parasitic elimination at target exposures attainable in humans, through four months following the end of treatment.<\/p>\n<\/li>\n
- \nIn the Phase 1 FIH study, AN2-502998 was generally well tolerated at exposure levels consistent with NHP efficacy thresholds, with no dose-limiting toxicities.<\/p>\n<\/li>\n<\/ul>\n
\n\u201cResults 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,\u201d said Eric Easom, Co-founder, Chairman, President and CEO of AN2 Therapeutics. \u201cTogether, these data support our goal of making AN2-502998 the first FDA-approved therapy for chronic Chagas disease in adults.\u201d<\/p>\n
\nEasom continued, \u201cWe 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.\u201d<\/p>\n
\n“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,\u201d said Dr. David Hamer, Professor of Global Health and Medicine, Boston University Schools of Public Health and Medicine. \u201cThe 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.”<\/p>\n
Overview of Chagas Disease<\/b><\/p>\n
\nChagas disease (also known as American trypanosomiasis) is caused by the parasite T. cruzi<\/i>. 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 <\/i>infection is lifelong and can be life threatening. The parasite T. cruzi <\/i>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.<\/p>\n
Chagas Disease Opportunity<\/b><\/p>\n
\nThe 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.<\/p>\n
Key Findings: 28-Day Nonhuman Primate (NHP) Study<\/b><\/p>\n
\nThe NHP efficacy study evaluated AN2-502998 administered for 28 days in macaques with naturally-acquired, chronic T. cruzi<\/i> 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.<\/p>\n
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- \n100% of treated animals achieved parasite elimination at target exposures attainable in humans.<\/p>\n<\/li>\n
- \nElimination of parasitemia in treated animals was durable and maintained through four months following the end of treatment.<\/p>\n<\/li>\n
- \nParasitemia was evaluated using an enhanced PCR method with improved sensitivity and robustness.<\/p>\n<\/li>\n
- \nAN2-502998 was well tolerated with no drug-related adverse events.<\/p>\n<\/li>\n
- \nAN2-502998 is the only compound to have demonstrated curative activity in NHPs with long-term, naturally acquired T. cruzi<\/i> infection.<\/p>\n<\/li>\n<\/ul>\n
Key Findings: Phase 1 First-in-Human Study<\/b><\/p>\n
\nThe 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:<\/p>\n
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- \nAN2-502998 was generally well tolerated with no dose-limiting toxicities.<\/p>\n<\/li>\n
- \nHuman PK was well characterized; plasma exposures at or above NHP efficacy thresholds were achieved.<\/p>\n<\/li>\n<\/ul>\n
About AN2-502998<\/b><\/p>\n
\nAN2-502998 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<\/i>. 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 parasite.<\/p>\n
\nAN2 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<\/i> biology has been instrumental in advancing the scientific understanding of Chagas disease.<\/p>\n
About AN2 Therapeutics<\/b><\/p>\n
\nAN2 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.<\/p>\n
Forward-Looking Statements<\/b><\/p>\n
\nThis 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 \u201cmay,\u201d \u201cwill,\u201d \u201cshould,\u201d \u201cwould,\u201d \u201cexpect,\u201d \u201cplan,\u201d \u201canticipate,\u201d \u201ccould,\u201d \u201cintend,\u201d \u201ctarget,\u201d \u201cproject,\u201d \u201cbelieve,\u201d \u201cestimate,\u201d \u201cpredict,\u201d \u201cpotential,\u201d or \u201ccontinue,\u201d 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\u2019s boron chemistry platform and advancement of the Company\u2019s 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\u2019s clinical trials; the predictivity of data; and other statements that are not historical fact. These statements are based on AN2\u2019s 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\u2019s ability to implement its plans for its internal boron chemistry platform and pipeline programs; timely enrollment of patients in AN2\u2019s clinical trials and investigator-initiated clinical trials; AN2\u2019s 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\u2019s product candidates; the significant uncertainty associated with AN2\u2019s product candidates ever receiving any regulatory approvals; AN2\u2019s ability to obtain, maintain, or protect intellectual property rights related to its current and future product candidates; implementation of AN2\u2019s strategic plans for its business and product candidates; the sufficiency of AN2\u2019s 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 \u201cRisk Factors\u201d in AN2\u2019s 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\u2019s website at www.an2therapeutics.com and on the SEC\u2019s 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.<\/p>\n
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View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20260604006839\/en\/<\/a><\/span><\/p>\n
COMPANY CONTACT:
\n<\/b>
Lucy O. Day
\n
Chief Financial Officer
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l.day@an2therapeutics.com<\/a><\/p>\nINVESTOR AND MEDIA CONTACT:
\n<\/b>
Anne Bowdidge
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ir@an2therapeutics.com<\/a><\/p>\nKEYWORDS:<\/b> United States North America California<\/p>\n
INDUSTRY KEYWORDS:<\/b> Biotechnology FDA Health General Health Public Relations\/Investor Relations Pharmaceutical Communications Research Infectious Diseases Science Clinical Trials<\/p>\n
- \nIn the Phase 1 FIH study, AN2-502998 was generally well tolerated at exposure levels consistent with NHP efficacy thresholds, with no dose-limiting toxicities.<\/p>\n<\/li>\n<\/ul>\n
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