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

总结 这项申请的最终目标是开发一种Epigen专有的、选择性的和药物样的激酶 用于有效治疗与慢性疾病如非酒精性疾病相关的肝纤维化的抑制剂 脂肪性肝炎（NASH），一种严重类型的非酒精性脂肪性肝病（NAFLD）。NAFLD是最常见的 肝脏疾病，并与肥胖和2型糖尿病有关。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价，根据 以疾病特异性进行性截断标准，在两种NASH动物模型中建立概念验证 在这个指示。这项工作的成功结果将使我们能够开始努力， 了解啮齿动物NASH模型中最有希望的候选药物的药理学， 开始毒理学和化学，制造和控制（CMC）工作，以支持提交 研究性新药（IND）申请，并最终启动人体临床试验。

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.