Advancing a new TB drug through early clinical trials

描述（由申请人提供）：本生物防御产品开发挑战拨款申请要求支持一种新的结核病药物的早期临床试验。新药sq109对耐多药结核病有效，耐多药结核病是NIAID C类高优先级病原体。SQ109是由一家小型生物制药公司与美国国立卫生研究院合作开发的。所有发现阶段的研究已经完成，该化合物处于临床前开发的最后阶段。拟议的工作范围将推动SQ109通过提交IND的最终监管要求，通过所有I期安全性试验，以及通过预期的三个II期临床试验中的两个。即将完成的II期临床试验将是一项为期7天的SQ109研究，其早期杀菌活性（EBA）作为抗结核疗效的关键指标，以及一项为期2个月的联合治疗研究，以痰转化为主要结局指标。这些研究将使SQ109进入临床开发的最后阶段——一项最终的、长期的II期研究，将作为III期临床试验的试点研究，以及III期临床试验本身。开发一种对耐多药结核病有效的新型结核病药物将带来巨大的公共卫生效益和全球卫生影响，并为防止结核病可能被用作生物武器提供防御。

DESCRIPTION (provided by applicant): 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.