云顶新耀宣布治疗原发性膜性肾病的新型BTK抑制剂EVER001在1b/2a期临床试验最新阶段性数据，持续展现优异的有效性与安全性

Core Viewpoint - The latest phase data from the 1b/2a clinical trial of EVER001 shows promising efficacy and safety in treating primary membranous nephropathy (pMN), supporting its potential as a treatment for autoimmune glomerular diseases characterized by proteinuria [2][3][4]. Group 1: Clinical Trial Results - As of March 21, 2025, the ongoing 1b/2a clinical trial has collected long-term data, with 11 patients in the low-dose group followed for 52 weeks, and 16 and 12 patients in the high-dose group completing 24 and 36 weeks of treatment, respectively [1][3]. - The geometric mean levels of anti-PLA2R antibodies decreased by 62.2% and 87.3% in the low and high-dose groups at 12 weeks, with both groups showing over 93% reduction at 24 weeks [1][3]. - The 24-hour proteinuria geometric mean values decreased by 57.0% and 67.6% at 24 weeks for the low and high-dose groups, respectively, with further reductions to 76.7% and 80.6% at 36 weeks, maintained until the 52-week follow-up [4][5]. Group 2: Safety and Tolerability - EVER001 demonstrated good safety and tolerability, with most adverse events being mild to moderate and transient, consistent with previous observations [1][3][6]. - No clinically significant adverse events commonly associated with other BTK inhibitors, such as bleeding or severe infections, were observed [3][6]. Group 3: Market Potential and Future Development - The company aims to accelerate the global clinical development of EVER001, which has the potential to address the unmet medical needs of over 10 million patients with pMN and other autoimmune kidney diseases [5][15]. - Currently, there are no approved drugs for the treatment of pMN globally, highlighting the significant market opportunity for EVER001 [4][6]. - The company plans to initiate Phase 3 clinical trials and engage with regulatory authorities in the U.S. and China to expedite the development process [6][17]. Group 4: Mechanism and Advantages - EVER001 is a next-generation covalent reversible BTK inhibitor, offering advantages such as better selectivity and reduced off-target toxicity compared to traditional irreversible BTK inhibitors [5][13]. - The drug's mechanism allows for rapid and sustained immune and clinical responses, with significant reductions in antibody levels and proteinuria observed early in treatment [11][12][13]. Group 5: Broader Applications - The company believes that EVER001 may also be effective for other autoimmune diseases, including IgA nephropathy and lupus nephritis, due to its mechanism of action [15][18]. - There is a significant unmet need in these conditions, with millions of patients potentially benefiting from this treatment [15][18].