AN2 Therapeutics has completed a comprehensive 200-patient observational study in acute melioidosis that revealed alarming mortality rates and highlighted critical gaps in current treatment approaches. The study, conducted under real-world conditions in acute hospital settings, found a mortality rate of nearly 40% by Day 90 among confirmed melioidosis cases receiving standard of care treatment with IV meropenem or ceftazidime.
The National Institutes of Health-funded study was completed in just 11 months across three sites in acute melioidosis-endemic regions, demonstrating efficient enrollment and strong site engagement. Principal Investigators observed that approximately 25% of screened patients died within a short period of 3-4 days before a definitive diagnosis of infection with Burkholderia pseudomallei could be confirmed, and these deaths were not included in the topline mortality rate.
Study Design and Outcomes
The observational study generated an important follow-up dataset for patients treated for acute melioidosis with current standard of care antibiotics, tracking patients for over 90 days. The results underscore the devastating impact of melioidosis and the limitations of current therapies, even under best-of-care conditions.
"The study represents a critical step in our mission to transform outcomes for patients with melioidosis," said Eric Easom, Co-founder, Chairman, President and CEO of AN2 Therapeutics. "It has given us a much deeper understanding of the patient population, clarified enrollment criteria, and provided the insights needed to design a Phase 2 proof-of-concept trial with the greatest chance of success."
Implications for Drug Development
The mortality findings highlight the serious impact of melioidosis, the critical importance of early detection and the urgent need for more effective treatment options. AN2 believes epetraborole has the potential to significantly reduce mortality when added to standard treatment, and these findings will directly support the company's planned IND submission.
"We look forward to initiating our Phase 2 trial later this year to address this urgent and unmet medical and biodefense need," Easom stated. The company plans to design a Phase 2 proof-of-concept trial with the greatest chance of success based on the insights gained from this observational study.
Disease Burden and Biothreat Classification
Melioidosis is estimated to cause over 200,000 cases globally each year, with the disease-causing pathogen mostly found in tropical climates, especially in Southeast Asia and northern Australia. In the United States, B. pseudomallei occurs in Puerto Rico, the U.S. Virgin Islands, and the Gulf Coast area of Mississippi.
Due to its high mortality and ease of environmental acquisition, B. pseudomallei is classified as a high priority biothreat agent. If approved for the treatment of acute melioidosis, AN2 would seek a priority review voucher and could generate revenue from U.S. and other government stockpiling, as well as from use as a treatment in disease endemic countries.
Research Collaboration
Easom acknowledged the invaluable contribution of all organizations involved in the study, especially the University of Oxford, MORU (Mahidol Oxford Tropical Medicine Research Unit) and LOMWRU (Lao-Oxford-Mahosot Hospital-Wellcome Trust Research Unit), together with the hospital sites and patient participants and their families.
The project was funded entirely with Federal funds from the National Institute of Allergy and Infectious Diseases, National Institutes of Health, Department of Health and Human Services, under Contract No. 75N93022C00059.
