Kornecook Thomas J, Yin Ruoyuan, Altmann Stephen, Be Xuhai, Berry Virginia, Ilch Christopher P, Jarosh Michael, Johnson Danielle, Lee Josie H, Lehto Sonya G, Ligutti Joseph, Liu Dong, Luther Jason, Matson David, Ortuno Danny, Roberts John, Taborn Kristin, Wang Jinti, Weiss Matthew M, Yu Violeta, Zhu Dawn X D, Fremeau Robert T, Moyer Bryan D
Department of Neuroscience (T.J.K., R.Y., S.A, C.P.I., M.J., D.J., J.H.L., S.G.L., J.Li., D.L., J.Lu., D.M., D.O., K.T., J.W., V.Y., D.X.D.Z., R.T.F., B.D.M.), Department of Medicinal Chemistry (M.M.W.), and Department of Pharmacokinetics and Drug Metabolism (X.B., V.B., J.R.), Amgen Inc., Cambridge, Massachusetts and Thousand Oaks, California.
Department of Neuroscience (T.J.K., R.Y., S.A, C.P.I., M.J., D.J., J.H.L., S.G.L., J.Li., D.L., J.Lu., D.M., D.O., K.T., J.W., V.Y., D.X.D.Z., R.T.F., B.D.M.), Department of Medicinal Chemistry (M.M.W.), and Department of Pharmacokinetics and Drug Metabolism (X.B., V.B., J.R.), Amgen Inc., Cambridge, Massachusetts and Thousand Oaks, California
J Pharmacol Exp Ther. 2017 Jul;362(1):146-160. doi: 10.1124/jpet.116.239590. Epub 2017 May 4.
Potent and selective antagonists of the voltage-gated sodium channel Na1.7 represent a promising avenue for the development of new chronic pain therapies. We generated a small molecule atropisomer quinolone sulfonamide antagonist AMG8379 and a less active enantiomer AMG8380. Here we show that AMG8379 potently blocks human Na1.7 channels with an IC of 8.5 nM and endogenous tetrodotoxin (TTX)-sensitive sodium channels in dorsal root ganglion (DRG) neurons with an IC of 3.1 nM in whole-cell patch clamp electrophysiology assays using a voltage protocol that interrogates channels in a partially inactivated state. AMG8379 was 100- to 1000-fold selective over other Na family members, including Na1.4 expressed in muscle and Na1.5 expressed in the heart, as well as TTX-resistant Na channels in DRG neurons. Using an ex vivo mouse skin-nerve preparation, AMG8379 blocked mechanically induced action potential firing in C-fibers in both a time-dependent and dose-dependent manner. AMG8379 similarly reduced the frequency of thermally induced C-fiber spiking, whereas AMG8380 affected neither mechanical nor thermal responses. In vivo target engagement of AMG8379 in mice was evaluated in multiple Na1.7-dependent behavioral endpoints. AMG8379 dose-dependently inhibited intradermal histamine-induced scratching and intraplantar capsaicin-induced licking, and reversed UVB radiation skin burn-induced thermal hyperalgesia; notably, behavioral effects were not observed with AMG8380 at similar plasma exposure levels. AMG8379 is a potent and selective Na1.7 inhibitor that blocks sodium current in heterologous cells as well as DRG neurons, inhibits action potential firing in peripheral nerve fibers, and exhibits pharmacodynamic effects in translatable models of both itch and pain.
电压门控钠通道Na1.7的强效选择性拮抗剂是开发新型慢性疼痛疗法的一个有前景的途径。我们合成了一种小分子阻转异构喹诺酮磺酰胺拮抗剂AMG8379和一种活性较低的对映体AMG8380。在此我们表明,在全细胞膜片钳电生理实验中,使用可检测处于部分失活状态通道的电压方案,AMG8379能有效阻断人Na1.7通道,其半数抑制浓度(IC)为8.5 nM,对背根神经节(DRG)神经元中的内源性河豚毒素(TTX)敏感钠通道的IC为3.1 nM。与其他钠家族成员相比,AMG8379具有100至1000倍的选择性,这些成员包括肌肉中表达的Na1.4、心脏中表达的Na1.5以及DRG神经元中的TTX抗性钠通道。使用离体小鼠皮肤 - 神经制备模型,AMG8379以时间和剂量依赖性方式阻断C纤维中机械诱导的动作电位发放。AMG8379同样降低了热诱导的C纤维放电频率,而AMG8380对机械和热反应均无影响。在多个依赖Na1.7的行为终点评估了AMG8379在小鼠体内的靶点结合情况。AMG8379剂量依赖性地抑制皮内组胺诱导的搔抓和足底注射辣椒素诱导的舔舐,并逆转紫外线辐射皮肤烧伤诱导的热痛觉过敏;值得注意的是,在相似的血浆暴露水平下,AMG8380未观察到行为效应。AMG8379是一种强效且选择性的Na1.7抑制剂,可阻断异源细胞以及DRG神经元中的钠电流,抑制外周神经纤维中的动作电位发放,并在瘙痒和疼痛的可转化模型中表现出药效学作用。