Drug Research Division, Sumitomo Dainippon Pharma. Co., Ltd., 3-1-98, Kasugade-naka, Konohana-ku, Osaka 554-0022, Japan.
Drug Research Division, Sumitomo Dainippon Pharma. Co., Ltd., 3-1-98, Kasugade-naka, Konohana-ku, Osaka 554-0022, Japan.
Bioorg Med Chem Lett. 2021 Feb 1;33:127753. doi: 10.1016/j.bmcl.2020.127753. Epub 2020 Dec 28.
Huntington's disease (HD) is one of the serious neurodegenerative diseases and no disease modifiers are available to date. The correction of unbalanced kynurenine pathway metabolites may be useful to treat disease progression and kynurenine monooxygenase (KMO) is considered an ideal drug target. A couple of KMO inhibitors have been reported, but their brain permeability was very poor. We found pyridazinylsulfonamide as a novel lead compound, and it was optimized to the brain-permeable and highly potent KMO inhibitor 12, which was equipotent with CHDI-340246 and superior to CHDI-340246 in terms of brain penetration. Compound 12 was effective in R6/2 mice (HD model mice), i.e. neuroprotective kynurenic acid was increased, whereas neurotoxic 3-hydroxykynurenine was suppressed. In addition, impaired cognitive function was improved. Therefore, the brain-permeable KMO inhibitor was considered to be a disease modifier for HD treatment.
亨廷顿病(HD)是一种严重的神经退行性疾病,目前尚无疾病修饰剂。纠正不平衡的犬尿氨酸途径代谢物可能有助于治疗疾病进展,而犬尿氨酸单加氧酶(KMO)被认为是一个理想的药物靶点。已经报道了几种 KMO 抑制剂,但它们的脑通透性很差。我们发现哒嗪基磺酰胺是一种新型的先导化合物,并对其进行了优化,得到了具有脑渗透性和高活性的 KMO 抑制剂 12,它与 CHDI-340246 等效力,并且在脑穿透方面优于 CHDI-340246。化合物 12 在 R6/2 小鼠(HD 模型小鼠)中有效,即增加了具有神经保护作用的犬尿氨酸,同时抑制了具有神经毒性的 3-羟基犬尿氨酸。此外,受损的认知功能也得到了改善。因此,具有脑渗透性的 KMO 抑制剂被认为是治疗 HD 的疾病修饰剂。