Intellia:第1相nex-z、TTR90%減少を3年間維持

The Phase 1 data update demonstrates that a single dose of nex-z continues to produce deep, durable TTR reductions (≥90% mean) out to three years, with 72% of evaluable patients showing clinically meaningful mNIS+7 improvement at 24 months. These results reinforce the regulatory path for the ongoing Phase 3 MAGNITUDE-2 trial, whose enrollment completion is now guided for H1 2026 and BLA submission targeted for 2028.

主要事実

提出: 2025年9月25日 20:19
イベント: トップライン結果
方向: ポジティブ
企業: NTLAインテリア
アセット: nexiguran ziclumeran
出典: SEC 8-K
信頼度: 出典あり
24ヶ月mNIS+7改善: 72% (18例中13例)
24ヶ月平均ttr減少: 92%
36ヶ月平均ttr減少: 90%
PND改善または安定: 89%

出典の抜粋

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