发现3R4R15:一种先进的B因子抑制剂进入补充介导疾病的第三阶段
Changyou Ma1, Jincai Su1, Jiang Liu1
1Department of the Research Institute, Nanjing Chia-Tai Tianqing Pharmaceutical Co., Ltd., Nanjing 210046, P. R. China.
Journal of medicinal chemistry
|January 9, 2026
概括
一种新的候选药物 (3R,4R) -15显示出对补体系统的替代途径的强烈抑制. 它的每日一次剂量和对性夜间血红蛋白尿症患者的有希望的疗效可能会改善治疗遵守性.
科学领域:
- 补充系统生物学 补充系统生物学
- 药物发现和开发 药物发现和开发
背景情况:
- 补体系统的替代途径 (AP) 与PNH,AMD和aHUS等疾病有关.
- 目前的Iptacopan等治疗方法需要每天服用两次,这会影响患者的服药.
研究的目的:
- 确定一种针对AP的新型治疗剂,具有改善的药理动力学特性和剂量方便性.
- 在临床前和早期临床研究中评估新型化合物 (3R,4R) -15的疗效和安全性.
主要方法:
- 使用跳跃架策略来识别新型抑制剂.
- 试验室试验用于确定对B因子 (FB) 和AP的抑制活性.
- 进行了药理动力学研究,包括对小鼠进行口服生物可用性评估.
- 在PNH患者中进行了初步临床研究.
主要成果:
- 鉴定出的临床候选物 (3R,4R) - 15显示出强烈的FB (IC50 = 10.2 nM) 和AP (IC50 = 59.3 nM) 的抑制.
- (3R,4R) -15表现出良好的口服生物利用率 (在小鼠中为69.2%).
- 在PNH患者的初步临床数据表明有希望的疗效和每天服用一次剂量的潜力.
结论:
- (3R,4R) - 15是针对AP的有前途的临床候选人.
- 该化合物的有利的药理动力学和每天一次剂量的潜力可以显著提高患者的服药性.
- (3R,4R) -15正在进入PNH治疗的第三阶段试验.
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