AN2 Pauses Phase 3 Enrollment in MAC Lung Disease Trial

AN2 Therapeutics, a biopharmaceutical firm specializing in treatments for rare and serious infectious diseases, has decided to voluntarily halt Phase 3 enrollment in its ongoing clinical trial, EBO-301. This trial is assessing the efficacy of epetraborole in treating Mycobacterium avium complex (MAC) lung disease, a condition with limited therapeutic options and low response rates to existing treatments. The pause comes after an aggregate analysis indicated potential efficacy lower than anticipated, but it is not due to safety concerns.

The trial, which is double-blind and placebo-controlled, is divided into two phases. The Phase 2 segment, completed in September 2023, enrolled 80 patients and is set to inform the Phase 3 segment. AN2 Therapeutics expects to announce topline data from Phase 2 in the summer of 2024. The Phase 3 segment has already enrolled nearly 100 patients, a faster pace than anticipated.

The pause in enrollment will allow for a more thorough review of the study's data and for the independent Data Safety Monitoring Board (DSMB) to conduct an assessment and recommend subsequent actions. This may include modifications to the Phase 3 protocol. Existing participants in the Phase 2/3 trial will be permitted to continue.

Eric Easom, the Co-Founder, President, and CEO of AN2 Therapeutics, emphasized the complexity of developing new therapies for treatment-refractory MAC lung disease and stated that the decision to pause enrollment will facilitate a deeper evaluation of the data. He also mentioned that further details on the epetraborole trial will be provided in the second quarter of 2024.

The pivotal Phase 2/3 trial of epetraborole (EBO-301) is designed to evaluate the safety and efficacy of the drug in patients with MAC lung disease using an adaptive design. The Phase 2 findings will guide the clinical response measures and confirm the final sample size for Phase 3. The primary objective of Phase 3 is to determine if epetraborole combined with standard-of-care drugs is superior to a placebo.

AN2 Therapeutics is focused on developing treatments for diseases with significant unmet medical needs. Its lead candidate, epetraborole, is being studied as a daily oral treatment for nontuberculous mycobacteria (NTM) lung disease, a rare and progressive condition that can lead to irreversible lung damage and be fatal.