BCATc Inhibitor 2 exhibited an IC50 of 0.8 microM in the hBCATc assays; it is an active and selective inhibitor. BCATc Inhibitor 2 also blocked calcium influx into neuronal cells following inhibition of glutamate uptake, and demonstrated neuroprotective efficacy in vivo[1]. Branched-chain amino acid transferases (BCATs) have been implicated in catalyzing reversible transamination of isoleucine, leucine, and valine branched-chain amino acids to their corresponding α-keto acids, generating L-glutamate. It has been identified that there are two forms of BCAT in mammals: mitochondrial BCAT (BCATm) and cytosolic BCAT (BCATc). BCATc is expressed in particular brain region and involved in regulating glutamate synthesis for release during neuronal excitation. Thus, BCATc inhibition may be useful for the treatment of neurodegenerative and behavioral disorders involving disturbances of the glutamatergic system [2].

BCATm (rat):3.0 μM (IC50), BCATc (rat):0.2 μM (IC50), BCATc (human):0.8 μM (IC50)

BCATc抑制剂可能有助于治疗涉及谷氨酸系统紊乱的神经退行性及其他神经障碍。在hBCATc实验中，BCATc Inhibitor 2的IC50值为0.8 ± 0.05 μM。在重组大鼠BCATc实验和粗制大鼠BCATm实验中，其IC50分别为0.2 μM ± 0.02和3.0 μM ± 0.5（n=5）。BCATc Inhibitor 2在神经细胞培养中减少了钙离子流入，IC50为4.8 ± 1.2 μM（n=4）[1]。

BCATc Inhibitor 2在抑制谷氨酸摄取后阻断了钙离子流入神经细胞，并在体内显示出神经保护效果。在Lewis大鼠中，经30 mg/kg BCATc Inhibitor 2（皮下注射）处理后，血浆峰值浓度(Cmax)在0.5小时(tmax)达到8.28 μg/ml。平均血浆暴露量（AUC）值为19.9 μg h/ml，平均末端半衰期介于12至15小时，表明BCATc Inhibitor 2具有有利的药代动力学（PK）参数。每日给予线粒体神经毒素3-硝基丙酸（3-NP）产生纹状体损害并导致运动缺陷。连续9天给药的BCATc Inhibitor 2几乎完全逆转了3-NP的效应[1]。