PASADENA, Calif.---- Arrowhead Pharmaceuticals Inc. today announced that it has received clearance from the U.S. Food and Drug Administration to proceed with an adaptive Phase 2/3 trial with the potential to serve as a pivotal registrational study of ARO-AAT, the company’s second generation subcutaneously administered RNA interference therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency .
Arrowhead intends to initiate the adaptive design, Phase 2/3 study of ARO-AAT in patients with AATD associated liver disease at various sites in the U.S. in the second quarter of 2019, followed by various international sites in Europe, pending regulatory submission and review. The proposed primary objectives are to evaluate safety and pharmacodynamic dose response, and to evaluate efficacy, defined as an improvement in a histologic grading scale of AATD associated liver disease, and no worsening of liver fibrosis based on Ishak score on end of study biopsy. The company plans to provide additional study details following its initiation.
加州帕萨迪纳。--- Arrowhead医药今天宣布，获得FDA批准，开始进行适应性2/3期试验，有望成为 ARO-AAT 的关键注册研究。该公司的第二代皮下注射 RNA 干扰疗法开发用于治疗罕见的遗传性肝病与α-1抗胰蛋白酶缺乏。
Arrowhead 计划于2019年第二季度在美国各地启动针对 AATD 相关肝病患者的 ARO-AAT 自适应设计、第2/3期研究，随后在欧洲开展多个国际站点，以等待监管机构的提交和审查。建议的主要目标是评估安全性和药效剂量反应，并评估疗效，定义为改善 AATD 相关性肝病的组织学分级标准，以及在研究结束活检时基于 Ishak 评分的肝纤维化没有恶化。该公司计划在启动后提供更多的研究细节。