aibioAN2 Therapeutics reports EBO-301 miss, projects $78.5 million in cash lasts into 2028
Tickers ANTX
AN2 Therapeutics says the Phase 3 portion of its EBO-301 study missed its primary endpoint, and it projects $78.5 million in cash lasting into 2028 while it shifts to Chagas, melioidosis and oncology programs that have no human efficacy data yet.
Key facts
- Company
- AN2 Therapeutics, Inc. (NASDAQ: ANTX)
- News type
- Distress
- Primary source
- AN2 Therapeutics, Inc. 8-K (2025-05-01)
AN2 Therapeutics, Inc. (Nasdaq: ANTX) published topline results on May 1, 2025 from the Phase 3 portion of its EBO-301 study, a trial the company had terminated early. The study tested epetraborole added to an optimized background regimen in patients with treatment-refractory MAC lung disease, an infection caused by Mycobacterium avium complex that is hard to treat. The shortened Phase 3 portion enrolled 97 patients. It did not meet its primary endpoint, which was improvement in the respiratory domain of the Quality of Life-Bronchiectasis instrument, measured from baseline to month 6.
The company says the results were unable to confirm the clinical efficacy seen in an earlier Phase 2 study. Its chief executive, Eric Easom, who is also co-founder, chairman and president, said the company was disappointed.
Why the company says the trial failed
The release points to the population. It describes patients with severe, advanced MAC lung disease, a long duration of disease, high rates of cavitary or fibrocavitary disease, and multidrug resistance to standard antibiotics at baseline. That is the company's explanation, and the release offers no test of it against a comparison group.
The company says the study's results do not close the door on epetraborole. It says it is pursuing nondilutive means to advance potential clinical development in other nontuberculous mycobacterial populations, such as M. abscessus lung disease. The annual report filed March 25, 2025 lists the outcome of potential interactions with the FDA about future development in NTM, if any, among its forward-looking subjects. The release does not say that any such meeting has been held.
The pipeline the company is shifting to
The company says its focus stays on boron chemistry. Its Chagas program, AN2-502998, has started start-up activities for a Phase 1 study in healthy volunteers, with completion expected in the second half of 2025. The company estimates a peak annual sales potential of $1 billion for that drug and says it would be eligible for a priority review voucher if approved. The release reports 100% cure of naturally infected chronic Chagas disease in a nonhuman primate model, and it cites a related drug, acoziborole, for up to a 95% cure rate in a Phase 3 trial against sleeping sickness run by DNDi and Sanofi. Neither result is human efficacy data for AN2-502998.
The melioidosis program has topline data expected for an observational trial in the second quarter of 2025, followed by a Phase 2 study planned for the second half of 2025. The release gives the mortality rate in that disease as approaching 40%. The oncology portfolio is still preclinical in the release. The company says its first oncology compounds should advance into development in the second half of 2025, targeting ENPP1, PI3K alpha and other undisclosed targets.
Cash and the 2028 projection
The release says cash, cash equivalents and investments totaled $78.5 million at March 31, 2025, an unaudited figure. The company now projects that this cash will support operating plans into 2028, which extends its earlier guidance through 2027. The release credits measures over the past year that include a strategic reduction in workforce, reprioritization of the pipeline and non-dilutive funding for several key programs.
The projection is only as good as the spending assumed behind it, and the release gives no quarterly burn rate, so the 2028 date cannot be checked against spending here. The annual report lists the sufficiency of existing cash and the need for additional financing among its forward-looking subjects. The company also names continued NIAID funding of its melioidosis program as a risk, and says tuberculosis and malaria work is being funded by the Gates Foundation.
What comes next
The next dated milestones are the melioidosis observational data in the second quarter of 2025, the Chagas Phase 1 completion and the melioidosis Phase 2 start in the second half of 2025, and the first oncology compounds entering development in the same half year. The company has not named a date for any decision on epetraborole in other NTM populations, and any financing beyond the cash it holds would appear in later filings.