Xu Yiming, Cai Tian-Quan, Castriota Gino, Zhou Yuchen, Hoos Lizbeth, Jochnowitz Nina, Loewrigkeit Christopher, Cook John A, Wickham Alexandra, Metzger Joseph M, Ogletree Martin L, Seiffert Dietmar A, Chen Zhu
Zhu Chen, PhD, Cardiometabolic Diseases, Merck Research Laboratories, K15-3A-309, Galloping Hill Road, Kenilworth, NJ 07033, USA, Tel.: +1 908 740 0642, E-mail:
Thromb Haemost. 2014 Apr 1;111(4):694-704. doi: 10.1160/TH13-08-0668. Epub 2013 Dec 12.
Coagulation factor XII (FXII) plays a central role in initiating the intrinsic cascade of blood coagulation. Purified recombinant Human Albumin-tagged Infestin-4 (rHA-Infestin-4) is a recently described FXIIa inhibitor that displayed strong anticoagulant activity without compromising haemostasis in several animal models. We pursued detailed in vitro characterisation of rHA-Infestin-4 and demonstrated that it is a competitive inhibitor of FXIIa with slow on and off rate constants for binding (kon=5x10⁵ M⁻¹s⁻¹, koff=6x10⁻⁴ s⁻¹), it can block FXIIa activation of its physiological substrates (plasma prekallikrein and FXI), and it can inhibit ellagic acid-triggered thrombin generation in plasma. Potency and selectivity profiling in enzyme assays suggest that rHA-Infestin-4 is indeed highly potent on FXIIa (IC50=0.3 ± 0.06, 1.5 ± 0.06, 1.2 ± 0.09 nM, for human, rat, and rabbit FXIIa, respectively) with at least >100-fold selectivity against factors IIa, Xa, IXa, XIa, VIIa, and plasma kallikrein in all three species. rHA-Infestin-4 dose-dependently and markedly reduced clot weight in the arteriovenous shunt thrombosis model in rats and rabbits, accompanied with minimal increase in cuticle bleeding times in either species. rHA-Infestin-4 treatment at 5 mg/kg in rabbit resulted in a 13% reduction in ex vivo FXa activity, demonstrating a modest off-target effect. In summary, our findings confirmed and extended previous reports that inhibition of FXIIa by rHA-Infestin-4 can produce strong antithrombotic efficacy while preserving haemostasis. Our comprehensive selectivity profiling, mode of action, and kinetic studies of rHA-Infestin-4 reveal limitations of this molecule and offer new perspectives on any potential effort of discovering novel FXIIa inhibitors.
凝血因子 XII(FXII)在启动内源性凝血级联反应中起核心作用。纯化的重组人白蛋白标记的Infestin-4(rHA-Infestin-4)是一种最近描述的FXIIa抑制剂,在几种动物模型中显示出强大的抗凝活性,同时不影响止血功能。我们对rHA-Infestin-4进行了详细的体外特性研究,证明它是FXIIa的竞争性抑制剂,结合的开、关速率常数较慢(kon=5x10⁵ M⁻¹s⁻¹,koff=6x10⁻⁴ s⁻¹),它可以阻断FXIIa对其生理底物(血浆前激肽释放酶和FXI)的激活,并且可以抑制血浆中鞣花酸触发的凝血酶生成。酶分析中的效价和选择性分析表明,rHA-Infestin-4对FXIIa确实具有高度效力(人、大鼠和兔FXIIa的IC50分别为0.3±0.06、1.5±0.06、1.2±0.09 nM),在所有三个物种中对因子IIa、Xa、IXa、XIa、VIIa和血浆激肽释放酶的选择性至少>100倍。rHA-Infestin-4在大鼠和兔的动静脉分流血栓形成模型中剂量依赖性地显著降低了血栓重量,同时两种物种的表皮出血时间增加最小。兔以5 mg/kg的剂量用rHA-Infestin-4治疗导致离体FXa活性降低13%,表明有适度的脱靶效应。总之,我们的研究结果证实并扩展了先前的报道,即rHA-Infestin-4抑制FXIIa可以产生强大的抗血栓形成功效,同时保持止血功能。我们对rHA-Infestin-4进行的全面选择性分析、作用模式和动力学研究揭示了该分子的局限性,并为发现新型FXIIa抑制剂的任何潜在努力提供了新的视角。