Intellia reports nex-z Phase 1 data: 92% mean TTR reduction at 24 months, 72% of patients show mNIS+7 improvement

Intellia Therapeutics, Inc.

Mean serum TTR reduction of 92% at 24 months (n=33) and 90% at 36 months (n=12) after single dose.
72% of evaluable patients (13/18) achieved ≥4-point improvement in mNIS+7 by month 24; most who had progressed on patisiran improved.
89% of all 36 patients showed improvement or stability in PND scores through 24 months; mBMI, QoL-DN, NfL all trended toward improvement.
Nex-z generally well tolerated; infusion-related reactions mild/moderate, no discontinuations; three Grade ≥3 liver enzyme elevations resolved spontaneously.
Phase 3 MAGNITUDE-2 enrollment (approx. 50 patients) expected to complete in H1 2026; BLA submission for ATTRv-PN targeted by 2028.

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