PRECLINICAL DEVELOPMENT OF A NEW DRUG FOR PCP

描述(改编自《调查员摘要》)：总体 这一阶段STTR应用的目标是完成旨在 在推动一种治疗艾滋病的有前途的新药候选方面- 与卡氏肺孢子虫肺炎(PCP)相关的人体临床试验。 这将通过一项工作计划来实现，以实现以下目标 具体目标： (1)开发实用、经济、收敛的NPC1161合成方法 这与千克放大、未来的放射标记(用于阶段 作用机制和药代动力学研究)，以及展望 商业发展； (2)通过化学和/或方法制备NPC1161的假定代谢物 生物转化/生物催化方法； (3)建立灵敏、定量的分析方法(S) 生物体液中的推定代谢物(和母体药物)； (4)测定体外对人红细胞的摄取和毒性 NPC1161衍生物和推定的代谢物。 建议的商业应用： 潜在的商业应用程序不可用。

DESCRIPTION (Adapted from the Investigator's Abstract): The overall objective of this Phase 1 STTR application is to accomplish studies aimed at advancing a promising new drug candidate for the treatment of AIDS- related Pneumocystis carinii pneumonia (PCP) to human clinical trials. This will be accomplished through a plan of work to achieve the following specific aims: (1) develop a practical, economical and convergent synthesis of NPC1161 that is compatible with kilogram scale-up, future radiolabeling (for Phase II mechanism of action and pharmocokinetic studies), and prospective commercial development; (2) prepare putative metabolites of NPC1161 by chemical and/or bioconversion/biocatalysis methods; (3) develop a sensitive and quantitative analytical method(s) to detect putative metabolites (and parent drugs) in biological fluids; (4) determine in vitro uptake and toxicity to human red blood cells by NPC1161 derivatives and putative metabolites. PROPOSED COMMERCIAL APPLICATION: Potential commercial application not available.