A NOVEL THERAPY FOR RESPIRATORY SYNCYTIAL VIRUS

描述（由申请人提供）：临床上需要一种具有成本效益的无毒口服疗法来治疗幼儿和免疫功能低下成人的呼吸道合胞病毒（RSV）感染。 Microbiotix正在开发NMSO 3，一种硫酸化唾液酸脂质的易溶钠盐。 该化合物是一种有效的RSV抑制剂，在动物模型感染中具有口服和胃肠外体内功效。 NMSO3对RSV的抑制具有特异性。 申请人的目标是尽快制备该化合物的扩展生物学特征，以便其作为研究性新药（IND）申请候选物进行进一步开发。 建议进行研究以确定明显的多层作用机制。 这对于NMSO 3作为有效治疗药物的发展至关重要。 Microbiotix还将完善治疗棉鼠RSV肺部感染的最佳给药方案，并确定这些给药条件下的治疗指数。 在II期SBIR研究中，Microbiotix计划制备符合良好生产规范（GMP）质量的NMSO 3，在棉鼠模型中建立NMSO 3的基本药代动力学和口服生物利用度以及急性毒性。目标是在I期SBIR研究启动后三年内提交IND。

DESCRIPTION (provided by applicant): There is a clinical need for a cost-effective, non-toxic oral therapy to treat Respiratory Syncytial Virus (RSV) infections in young children and immuno ompromised adults. Microbiotix is developing NMSO3, a readily soluble sodium salt of a sulfated sialic acid-lipid. This compound is a potent RSV inhibitor with both oral and parenteral in vivo efficacy in animal model infections. NMSO3 has shown specificity for inhibition of RSV. It is the applicant's aim to prepare an expanded biological profile of this compound as soon as possible so that it might undergo further development as an Investigational New Drug (IND) application candidate. Studies are proposed to define the apparent multi tiered mechanism of action. This will be essential to the development of NMSO3 as an effective therapeutic drug. Microbiotix will also refine the optimum-dosing regimen for treating RSV lung infections in cotton rats, and define the therapeutic index under those dosing conditions. In the Phase II SBIR studies, Microbiotix plans to prepare good manufacturing practice (GMP) quality NMSO3, establish the essential pharmacokinetics and oral bioavailability and the acute toxicity for NMSO3 in cotton rat model. The goal is to file an IND within three years of initiation of the Phase I SBIR studies.