Intellia Therapeutics reports positive long-term data for Nexiguran Ziclumeran

By Hao Liu • Friday, September 26 06:02 • Permanent Link

Intellia Therapeutics, Inc. (NASDAQ:NTLA) has released positive longer-term phase 1 data for Nexiguran Ziclumeran (Nex-Z) in patients with hereditary transthyretin (ATTR) amyloidosis with polyneuropathy. The data was presented at the 5th International ATTR Amyloidosis Annual Meeting and simultaneously published in the New England Journal of Medicine.

The results show that a single dose of Nex-Z led to rapid, deep, and durable reductions in serum TTR levels, with mean reductions of at least 90% from baseline sustained through three years. Specifically, across patients who received a one-time dose of 0.3 mg/kg or higher, the mean serum TTR reduction at 24 months was 92%, and among patients who had reached 36 months of follow-up, the mean serum TTR reduction was 90%.

Furthermore, favorable trends indicating stability or improvement were observed in most patients with ATTRv-PN after a single dose of Nex-Z. Among the 18 patients who completed a 24-month assessment, 13 showed improvements of a clinically meaningful threshold of ≥4 points in the modified neuropathy impairment score +7 (MNIS+7).

In terms of safety, Nex-Z has been generally well tolerated, with the most commonly reported treatment-related adverse events being mild or moderate infusion-related reactions. Notably, three participants had grade >3 liver enzyme elevations, which were asymptomatic and resolved spontaneously without medical intervention.

Looking ahead, Intellia Therapeutics expects to complete enrollment in the multinational phase 3 Magnitude-2 trial in the first half of 2026 to support a potential Biologics License Application (BLA) filing by 2028. The trial is designed to evaluate the efficacy and safety of Nex-Z in approximately 50 patients with hereditary transthyretin ATTR amyloidosis with polyneuropathy, with the primary endpoints being a change in modified neuropathy impairment score and a change in serum TTR levels. Today the company's shares have moved -5.07% to a price of $16.86. For more information, read the company's full 8-K submission here.

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