Intellia Highlights Clinical Improvements With One-Time Dose Of Experimental Drug

Core Insights - Intellia Therapeutics Inc. has released long-term follow-up data from its Phase 1 study of nexiguran ziclumeran (nex-z) for treating hereditary ATTR amyloidosis with polyneuropathy, showcasing significant TTR reductions and clinical improvements [1][2][6] Data Summary - A one-time dose of 0.3 mg/kg or higher resulted in a mean serum TTR reduction of 92% at 24 months, with an absolute serum TTR level of 17.3 g/mL [2] - Among 12 patients with 36 months of follow-up, the mean serum TTR reduction was 90%, corresponding to a serum TTR level of 20 g/mL [2] - Favorable trends indicating stability or improvement were observed in most patients after a single dose of nex-z [3] Clinical Measures - Stability or improvement was assessed using various clinical and biomarker measures, including NIS, mNIS+7, mBMI, QoL-DN questionnaire, NfL, and PND score [4] - Of the 18 patients assessed at 24 months, 72% showed clinically meaningful improvements of ≥4 points in mNIS+7, including those who had previously progressed on Alnylam's Onpattro [5] Secondary Endpoints - Mean values for secondary endpoints mBMI, QoL-DN, and NfL trended towards disease improvement, with 89% of patients showing improvement or stability in PND scores through 24 months [6] - Nex-z was generally well tolerated, with mild to moderate infusion-related reactions being the most common treatment-related adverse events [6][7] Future Outlook - Patient screening for the Phase 3 MAGNITUDE-2 trial is progressing rapidly, with enrollment expected to complete in the first half of 2026 [8] - Intellia plans to submit a biologics license application (BLA) for ATTRv-PN by 2028 [8]