Walker Ann L, Ancellin Nicolas, Beaufils Benjamin, Bergeal Marylise, Binnie Margaret, Bouillot Anne, Clapham David, Denis Alexis, Haslam Carl P, Holmes Duncan S, Hutchinson Jonathan P, Liddle John, McBride Andrew, Mirguet Olivier, Mowat Christopher G, Rowland Paul, Tiberghien Nathalie, Trottet Lionel, Uings Iain, Webster Scott P, Zheng Xiaozhong, Mole Damian J
Discovery Partnerships with Academia, GlaxoSmithKline , Gunnels Wood Road, Stevenage SG1 2NY, UK.
Flexible Discovery Unit, GlaxoSmithKline , Paris, France.
J Med Chem. 2017 Apr 27;60(8):3383-3404. doi: 10.1021/acs.jmedchem.7b00055. Epub 2017 Apr 11.
Recently, we reported a novel role for KMO in the pathogenesis of acute pancreatitis (AP). A number of inhibitors of kynurenine 3-monooxygenase (KMO) have previously been described as potential treatments for neurodegenerative conditions and particularly for Huntington's disease. However, the inhibitors reported to date have insufficient aqueous solubility relative to their cellular potency to be compatible with the intravenous (iv) dosing route required in AP. We have identified and optimized a novel series of high affinity KMO inhibitors with favorable physicochemical properties. The leading example is exquisitely selective, has low clearance in two species, prevents lung and kidney damage in a rat model of acute pancreatitis, and is progressing into preclinical development.
最近,我们报道了犬尿氨酸 3-单加氧酶(KMO)在急性胰腺炎(AP)发病机制中的新作用。此前,已有多种犬尿氨酸 3-单加氧酶(KMO)抑制剂被描述为神经退行性疾病尤其是亨廷顿舞蹈病的潜在治疗药物。然而,迄今为止报道的抑制剂相对于其细胞活性而言,水溶性不足,无法与 AP 所需的静脉注射给药途径兼容。我们已鉴定并优化了一系列具有良好理化性质的新型高亲和力 KMO 抑制剂。其中的主要示例具有极高的选择性,在两种动物模型中清除率较低,可预防急性胰腺炎大鼠模型中的肺和肾损伤,目前正进入临床前开发阶段。