Assessment of a selective kinase inhibitor in pre-clinical models of NASH

摘要 这项应用的最终目标是开发一种Eigen的专有的、选择性的和类药物的激酶 有效治疗与非酒精性等慢性疾病相关的肝纤维化的抑制剂 脂肪性肝炎(NASH)，一种严重的非酒精性脂肪性肝病(NAFLD)。非酒精性脂肪肝是最常见的 肝脏疾病，与肥胖和2型糖尿病有关。目前还没有有效的治疗方法 纳什。我们的方法将涉及一种先进的先导化合物的临床前评估，根据 以疾病特异性进展性分界标准，在两个NASH动物模型中建立概念验证 在这一迹象中。这项工作的成功结果将使人们能够开始努力建立一个更详细的 了解最有希望的啮齿动物NASH模型和候选动物的药理学 开始毒理学和化学、制造和控制(CMC)工作，以支持提交 研究新药(IND)的应用，并最终在人体上启动临床试验。

Summary The ultimate goal of this application is to develop one of Epigen’s proprietary, selective and drug-like kinase inhibitors for the effective treatment of liver fibrosis associated with chronic diseases such as non-alcoholic steatohepatitis (NASH), a severe type of non-alcoholic fatty liver disease (NAFLD). NAFLD is the most common liver disease and is associated with obesity and type-2 diabetes. There are no effective treatments available for NASH. Our approach will involve the pre-clinical evaluation of one advanced lead compound, identified according to a disease-specific progressive cutoff criteria, in two animal models of NASH to establish the proof-of-concept in this indication. The successful outcome of this work will enable initiation of efforts to establish a more detailed understanding of the pharmacology of the most promising candidate in rodent NASH models and commencement of toxicology and chemistry, manufacturing and controls (CMC) work to support filing of an investigational new drug (IND) application and eventually initiation of clinical trials in humans.