Pipeline

AN2 Therapeutics is focused on developing epetraborole as a once-daily oral treatment for patients with chronic non-tuberculous mycobacterial (NTM) lung disease. The company is developing epetraborole to treat Mycobacterium avium complex, or MAC, lung disease, which accounts for approximately 80% of NTM lung disease. In addition to AN2’s development and commercial endeavors in NTM lung disease, the company intends to develop epetraborole for global health initiatives, including melioidosis, using non-dilutive funding, which the company plans to obtain from sources such as public and private agencies and foundations.

development pipeline graphic

development pipeline graphic

About Epetraborole

We are developing oral epetraborole for the treatment of non-tuberculous mycobacterial, or NTM, lung disease. Epetraborole is a boron-containing, orally-available, small molecule inhibitor of bacterial leucyl-tRNA synthetase, or LeuRS, an enzyme that catalyzes the attachment of leucine to transfer RNA, or tRNA, molecules, an essential step in protein synthesis. Epetraborole forms a complex with a tRNALeu molecule, trapping the terminal ribonucleotide of tRNALeu in the editing site of the enzyme, which prevents the synthetic site from attaching leucine to tRNALeu thus shutting down tRNA leucylation and leading to a block in protein synthesis.

About Non-Tuberculous Mycobacterial (NTM) Lung Disease

NTM lung disease is a rare, chronic, and progressive infectious disease caused by bacteria known as mycobacteria that leads to irreversible lung damage and can be fatal.

Unlike most bacteria, which replicate quickly and spread outside of cells, mycobacteria replicate slowly and mostly infect alveolar (lung) macrophages and survive within them. Due to the slow growth and survival within macrophages of mycobacteria, the current standard of care for NTM lung infections requires prolonged treatments, often for 18 months or longer, with a combination of three or more antibiotics. Initially, we are focused on developing epetraborole to treat the most common type of NTM, MAC, which accounts for approximately 80% of NTM lung disease in the United States.

There are an estimated 200,000 patients with NTM lung disease in the United States. We believe that many remain underdiagnosed due to lack of clinical suspicion, nonspecific respiratory symptoms, and underlying lung diseases that are frequent in patients with this infection. The prevalence of NTM lung disease is increasing in the United States by an estimated 8% per year. Among the approximately 55,000 patients diagnosed with NTM lung disease in the United States, approximately 44,000 patients have MAC lung disease, and approximately 35% of these patients have treatment-refractory MAC lung disease.

In addition, Japan has some of the highest rates of NTM lung disease in the world. It is estimated that there are 220,000 patients with NTM lung disease and 21,000 patients with treatment-refractory MAC lung disease in Japan.

Clinical Trials

AN2 Therapeutics is committed to addressing the challenges of infectious diseases and improving global human health. 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

Presentations and Publications

Epetraborole In Vitro Activity Against Mycobacterium Avium Complex Recent Clinical Isolates from Japan (#2135)
ID Week 2023

A Phase 1, Multicenter, Open-Label, Parallel-Group Study to Assess the Safety and Pharmacokinetics of Oral Epetraborole Tablets in Adult Subjects with Varying Degrees of Renal Function (#2144)
ID Week 2023

A Phase 1, Open-Label, Single Dose Study to Evaluate the Pharmacokinetics (PK), Safety, and Tolerability of Epetraborole Tablets and the Impact of Alcohol Dehydrogenase Genotype on the PK of Epetraborole and Metabolite M3 in Healthy Japanese Adult Subjects (#2556)
ID Week 2023

Epetraborole: A Novel Antibiotic for NTM Lung Disease & Melioidosis
ID Week 2023

Epetraborole: A Novel, Oral Antibiotic for NTM Lung Disease
ID Week 2022

Pharmacokinetic-Pharmacodynamic Target Attainment Analyses to Support Epetraborole Dose Selection for the Treatment of Patients with Mycobacterium avium Complex Lung Disease
ID Week 2022

Population Pharmacokinetic Model Development for Epetraborole and MAC Lung Disease Patients Using Data from Phase 1 and 2 Studies
ID Week 2022

Dose-response Studies of the Novel Bacterial Leucyl-tRNA Synthetase Inhibitor, Epetraborole, in the Intracellular Hollow Fiber System Model of Mycobacterium avium complex Lung Disease
ID Week 2022

Pharmacokinetics/pharmacodynamics of Epetraborole, a Novel Bacterial Leucyl-tRNA Synthetase Inhibitor, and High Intracellular Penetration in the Intracellular Hollow Fiber System Model of Mycobacterium avium Complex Lung Disease
ID Week 2022

Epetraborole, a Novel Bacterial Leucyl-tRNA Synthetase Inhibitor, Demonstrates Potent Efficacy and Improves Efficacy of Standard of Care Regimen Against Mycobacterium avium complex in a Chronic Mouse Lung Infection Model
ID Week 2022

In Vitro Activity of Epetraborole, a Novel Bacterial Leucyl-tRNA Synthetase Inhibitor, in Drug Combinations Against Nontuberculous Mycobacteria Including Resistance Frequency and MIC Characterization of Mycobacterium avium ATCC 700898 Epetraborole-resistant Mutants
ID Week 2022

In Vitro Activities of Epetraborole, a Novel Bacterial Leucyl-tRNA Synthetase Inhibitor, Against Mycobacterium avium Complex Isolates
ID Week 2022

In Vitro Drug-Drug Interaction Evaluation of Epetraborole, a Novel Bacterial Leucyl-tRNA Synthetase Inhibitor
ID Week 2022

Tolerability and Pharmacokinetics of Oral Epetraborole at the Predicted Therapeutic Dosage for Mycobacterium avium Complex (MAC) Lung Disease: A Phase 1b Dose-ranging and Food Effect Study
ID Week 2022