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Intellia Therapeutics Reports Longer-Term Phase 1 Data for Nexiguran Ziclumeran
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Intellia Therapeutics Reports Longer-Term Phase 1 Data for Nexiguran Ziclumeran
Sep 25, 2025 7:59 AM

10:37 AM EDT, 09/25/2025 (MT Newswires) -- Intellia Therapeutics ( NTLA ) said Thursday its longer-term data from the ongoing phase 1 trial of its investigational therapy, nexiguran ziclumeran, or nex-z, in hereditary transthyretin amyloidosis or ATTR with polyneuropathy showed "deep and durable" reductions of serum TTR levels.

For those who received a one-time dose of nex-z, the mean serum TTR reduction at 24 months was 92%, while among the 12 patients who had reached three years of follow-up, the mean reduction was 90%, the company said.

Intellia said stabilization or improvement in clinical measures was observed at 24 months, with a favorable safety profile and no new drug-related events during follow-up.

Enrollment in the phase 3 magnitude-2 trial is expected to be completed in the H1 of 2026, with a potential US regulatory filing targeted for 2028.

Intellia's stock fell 3% in recent trading.

Price: 17.22, Change: -0.54, Percent Change: -3.03

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