Pipeline

AN2 has a pipeline of novel boron-based compounds that have the potential to be best in class therapeutics. Our development pipeline spans hematologic diseases, infectious diseases, and oncology, alongside advanced research programs focused on targets in oncology, bone disorders and infectious diseases. The company intends to advance its global health initiatives using primarily non-dilutive funding, which the company obtains from sources such as public and private agencies and foundations. The Company is committed to delivering high-impact drugs to patients that address critical unmet needs and improve health outcomes.

development pipeline graphic

Epetraborole

Polycythemia Vera

We are expanding the development of oral epetraborole through 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, many patients experience uncontrolled hematocrit levels and persistent symptom burden, requiring long-term management to maintain adequate disease control. PV is estimated to affect approximately 155,000 people in the U.S.

Mycobacterium abscessus complex lung disease

The U.S. Food and Drug Administration has cleared an Investigational New Drug Application to proceed with a Phase 2 investigator-initiated study in collaboration with Oregon Health and Sciences University (OSHU) evaluating epetraborole for the treatment of Mycobacterium abscessus (M. abscessus) complex lung disease. This multicenter, randomized, double-blind, placebo-controlled, prospective clinical study will be led by Dr. Kevin Winthrop, Professor of Public Health and Infectious Diseases at OHSU, in conjunction with other investigators across an estimated 10-15 sites in the U.S. M. abscessus lung disease is a serious and difficult-to-treat non-tuberculous mycobacterial infection requiring prolonged therapy including IV-only antibiotics and characterized by limited treatment options and high rates of morbidity, and 5-year mortality. No FDA-approved drugs currently exist for its treatment.

AN2-502998

Chagas Disease

We are advancing oral AN2-502998 in a Phase 1 first-in-human trial as part of our Chagas disease development program. Chagas disease (American trypanosomiasis), is an infectious disease caused by Trypanosoma cruzi, which affects an estimated 6-10 million people worldwide, including approximately 300,000 people in the U.S. and over 100,000 in Europe. Chagas disease can remain asymptomatic for years before progressing to chronic disease. In approximately 20–30% of infected individuals, chronic infection leads to serious cardiac and gastrointestinal complications, including cardiomyopathy, heart failure, arrhythmias, stroke, and megacolon or megaesophagus, which can result in significant morbidity and premature death. AN2-502998 is the only compound of which we are aware to have demonstrated curative activity in preclinical studies across multiple species, including in nonhuman primates (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. There are no FDA-approved treatments for adults with Chagas disease. A Phase 1 clinical study is underway evaluating the safety, tolerability, and pharmacokinetics of oral AN2-502998 in healthy volunteers.

Our Research and Development Initiatives

We are pursuing a number of oncology targets where we believe our boron chemistry may offer a competitive advantage in terms of binding-site differentiation, pharmacodynamics, drug-like properties and intellectual property. Our lead oncology programs target PI3Kα and ENPP1. We plan to advance two oncology candidates into development targeting PI3Kα and ENPP1.

Beyond oncology, we are pursuing advanced research programs focused on targets in bone disorders and infectious diseases.

Global Health Programs

Our global health strategy focuses on leveraging our proprietary boron chemistry platform to develop treatments for infectious diseases that disproportionately affect underserved populations. These programs are funded primarily through non-dilutive grant funding, including sources such as public and private agencies and foundations. Our global health programs include melioidosis, a severe bacterial infection associated with high death rates in endemic regions (funded by the U.S. government), and tuberculosis, a leading cause of death from infectious disease worldwide that affects millions of people each year in partnership with the Gates Foundation and GSK.

Clinical Trials

AN2 Therapeutics is committed to delivering high-impact drugs to patients that address critical unmet needs and improve health outcomes. Successful clinical trials are required to gain regulatory approval for new medications to advance patient care and may be required to support any approved products.

For information on AN2 Therapeutics clinical trials that may be recruiting, search AN2 Therapeutics at www.clinicaltrials.gov