AN2 Therapeutics, Inc. (Nasdaq: ANTX), a biopharmaceutical company specializing in novel small molecule therapies from its boron chemistry platform, today announced topline results from the Phase 2 portion of the EBO-301 Phase 2/3 study. This study evaluates the efficacy of epetraborole combined with an optimized background regimen (OBR) for treating refractory MAC lung disease.
The Phase 2 segment of the EBO-301 study successfully demonstrated the potential validation of a novel patient-reported outcome (PRO) tool, showing a higher PRO-based clinical response rate with epetraborole + optimized background regimen (OBR) (39.5%) compared to placebo + OBR (25.0%; treatment difference 13.9%, p=0.19). However, sputum culture conversion at Month 6, a key secondary endpoint, was similar between the treatment groups (13.2% for epetraborole + OBR vs. 10.0% for placebo + OBR; treatment difference 3.4%, p=0.64).
In February, AN2 Therapeutics paused new patient enrollment in the Phase 3 segment due to observed lower-than-expected efficacy in blinded aggregate data. Based on today’s topline results, the company will terminate both the Phase 2 (80 patients) and Phase 3 (97 patients enrolled prior to pause) parts of the EBO-301 trial. The oral epetraborole 500 mg daily was generally well-tolerated, and the study was not terminated due to safety concerns.
Eric Easom, Co-founder, Chairman, President, and CEO, expressed disappointment, stating, “These results are deeply disappointing as there is a high unmet need for new therapies in treatment-refractory MAC lung disease. We sincerely thank everyone involved in this journey.”
Easom added, “In the coming months, we will evaluate the EBO-301 study results and consider future development of epetraborole for other NTM lung disease populations. We plan to accelerate our R&D efforts on our boron chemistry platform, focusing on promising programs in infectious diseases and oncology. We will also undertake a strategic restructuring to extend our cash runway through 2027, concentrating on advancing our pipeline.”
AN2’s core technology leverages boron chemistry to bind biological targets through reversible covalent bonds, aiming to address targets difficult to inhibit with traditional carbon-based molecules.
Pipeline Programs/Milestones:
- Chagas Disease: The company plans to start Phase 1 clinical development of a candidate aimed at curing chronic Chagas disease, which affects 6-7 million people worldwide, including approximately 300,000 in the U.S.
- Melioidosis: A Phase 2 study will be initiated with epetraborole to address melioidosis, a severe bacterial infection and global bioterrorism threat, aiming to reduce the 3-month mortality rate of ~50%.
- Early-Stage Pipeline: Ongoing research targets high unmet needs in infectious diseases and oncology, with multiple compounds expected within the company’s cash runway.
- Global Health: AN2 will continue to tackle global health issues like tuberculosis and malaria through non-dilutive funding, including support from the Gates Foundation.
About AN2 Therapeutics, Inc.
AN2 Therapeutics, Inc. is a biopharmaceutical company focused on discovering and developing novel small molecule therapeutics from its boron chemistry platform. The company’s pipeline includes boron-based compounds for Chagas disease, NTM, and melioidosis, along with early-stage programs in infectious diseases and oncology.
