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.
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