[Translation] A Phase II, randomized, double-blind, placebo-controlled, dose-finding study evaluating the efficacy and safety of ALXN2050 in adult subjects with proliferative lupus nephritis (LN) or immunoglobulin A nephropathy (IgAN)
ALXN2050 是一种强效、可逆口服小分子 D 因子 (FD) 抑制剂,是补体旁路途径 (AP) 的重要组成部分。补体系统参与增生性 LN 和 免疫球蛋白A肾病 (IgAN) 的病理生理学过程。本研究的目的是评估口服 ALXN2050 与安慰剂相比的疗效和安全性,证明对 FD 抑制有效性的概念验证,并确定解决接受背景标准疗法治疗的 LN 或 IgAN 患者的未满足医疗需求的有效剂量。
[Translation] ALXN2050 is a potent, reversible, oral, small molecule inhibitor of factor D (FD), an important component of the complement alternative pathway (AP). The complement system is involved in the pathophysiology of proliferative LN and immunoglobulin A nephropathy (IgAN). The objectives of this study are to evaluate the efficacy and safety of oral ALXN2050 compared to placebo, demonstrate proof of concept for the effectiveness of FD inhibition, and determine an effective dose to address unmet medical needs in patients with LN or IgAN treated with background standard of care.