Intellia Therapeutics Announces Positive Two-Year Data for Nexiguran Ziclumeran

By Philip Seaworth • Monday, May 19 10:04 • Permanent Link

Intellia Therapeutics, Inc. has announced positive two-year follow-up data from the ongoing phase 1 trial of Nexiguran Ziclumeran (Nex-Z) for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). The results were presented at the 2025 Peripheral Nerve Society (PNS) Annual Meeting.

Here are the key findings:

Patients who received a one-time dose of 0.3 mg/kg or higher experienced a mean serum TTR reduction of 90% by day 28, with levels remaining virtually unchanged for at least 24 months.
Favorable trends indicating stability or improvement were observed in patients with ATTRv-PN, including those previously on Patisiran, with evidence of disease progression prior to entering the study.
Among the 18 patients in whom a modified neuropathy impairment score +7 (MNIS+7) assessment was completed at 24 months, 14 out of 18 demonstrated a clinically meaningful improvement of ≥4 points as of the April 11, 2025 data cutoff, including 5 of the 6 patients who were previously progressing on Patisiran.
The most commonly reported treatment-related adverse events were mild or moderate infusion-related reactions, which did not result in any discontinuations.

The ongoing phase 3 Magnitude-2 trial is designed to measure clinical outcomes and evaluate how a single dose of Nex-Z can lead to a reduction in serum TTR, potentially supporting a BLA submission by 2028.

As a result of these announcements, the company's shares have moved 5.26% on the market, and are now trading at a price of $8.50. For the full picture, make sure to review Intellia Therapeutics's 8-K report.

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