This application for a Biodefense Product Development Challenge Grant requests support for the early clinical trials of a new tuberculosis drug. The new drug-SQ109-is effective against multi-drug resistant TB, which is a Category C NIAID high priority pathogen. SQ109 was developed by a small biopharmaceutical company, in collaboration with the NIH. All discovery phase research is completed, and the compound is at the final stage of pre-clinical development. The scope of work proposed would advance SQ109 through the final regulatory requirements for filing an IND, through all of the Phase I safety trials, and through two of the anticipated three Phase II clinical trials. The Phase II clinical trials to be completed will be a seven-day study of SQ109 with early bactericidal activity (EBA) as a key indicator of anti-TB efficacy, and a two-month combination-treatment study with sputum conversion as the primary outcome measure. These studies will position SQ109 for the last stages of clinical development-a final, longer-term Phase II study that will serve as a pilot study for the Phase III clinical trial, and the Phase III clinical trial itself. Development of a new TB drug that is effective against multi-drug resistant TB will have enormous public health benefits and global health impact, as well as providing defense against the possible use of TB as a bio-weapon.
