Appili Therapeutics Inc. is developing a best-in-class live attenuated vaccine ATI-1701 (F. tularensis SCHU S4 ΔclpB), a genetically modified strain of F. tularensis Type A SCHU S4 that is attenuated by deletion of the clpB gene designed to work against aerosolized bacteria Francisella tularensis, which the U.S. National Institutes of Health (NIH) defines as a Category A pathogen (an organism that poses the highest risk to national security and public health). Estimated to be 1,000-fold more infectious than anthrax, experts consider the aerosolized form to have a high potential for use in a bioterrorist attack. Successfully weaponized and likely deployed in the 20th Century, several US adversaries already have operational weapons programs leveraging this pathogen.
In preclinical animal studies, Appili announced positive one-year results from its preclinical study evaluating the efficacy of biodefense vaccine candidate ATI-1701 and demonstrated 100% protection from aerosolized weapons-grade F. tularensis, as compared to a 30% protection from LVS, the only vaccine currently used (but not FDA approved) for prevention of the disease.
Appili is the license holder for ATI-1701 and will strategically develop ATI-1701 under FDA’s Animal Rule, which replaces human efficacy studies with animal efficacy studies, shortening the time to reach market approval. Appili is delighted to be chosen for this tremendous opportunity to partner with government agencies under a public/partnership model to advance the ATI-1701 to the next stage of development. In November 2022, Appili announced that the US DOD, in partnership with DTRA (Defense Threat Reduction Agency) and via United States Air Force Academy (USAFA), will provide at least US$14 million to fund the pre-clinical development of ATI-1701. Under this public/private partnership with DTRA and USAFA, Appili will oversee a comprehensive development program for ATI-1701 to support an IND submission to the FDA.
If approved by the FDA, ATI-1701 may be eligible for a valuable Priority Review Voucher. Priority Review Vouchers typically sell for approximately US $100 million, and have sold for as much as US $350 million.