Intellia：nex-z 1期数据显示TTR降低90%持续至3年

1期数据更新显示，单次剂量nex-z持续产生深度、持久的TTR降低（平均≥90%）直至三年，72%的可评估患者在24个月时显示出具有临床意义的mNIS+7改善。这些结果强化了正在进行的3期MAGNITUDE-2试验的监管路径，该试验的入组完成现预计在2026年上半年，BLA提交目标为2028年。

关键事实

申报时间: 2025年9月25日 20:19
事件: 顶线结果
方向: 积极
公司: NTLAIntellia
资产: nexiguran ziclumeran
来源: SEC 8-K
可信度: 有来源
24 个月 mNIS+7 改善: 72%（13/18 名患者）
24 个月平均 TTR 降低: 92%
36 个月平均 TTR 降低: 90%
PND 改善或稳定: 89%

来源摘录

EX-99.1 2 d870108dex991.htm EX-99.1 EX-99.1 Exhibit 99.1 Intellia Therapeutics Announces Positive Longer-Term Phase 1 Data for Nexiguran Ziclumeran (nex-z) in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy • One dose of nex-z led to rapid, deep and durable TTR reductions, with mean reductions of at least 90% from baseline sustained through three years • Stabilization or improvement in disease-related clinical measures observed at 24 months • Continue to observe generally favorable safety data with no new drug-related events within the follow-up period • Data presented today at the 5 th International ATTR Amyloidosis Annual Meeting for Patients and Doctors with simultaneous publication in the New England Journal of Medicine • Expect to complete

SEC 8-K