18-05-2025
Intellia Therapeutics announces two-year follow-up data from trial of nex-z
Intellia Therapeutics (NTLA) announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran ziclumeran, nex-z, for the treatment of hereditary ATTR amyloidosis with polyneuropathy. Results were shared in an oral presentation on Sunday, May 18 at the 2025 Peripheral Nerve Society, PNS, Annual Meeting in Edinburgh, United Kingdom. The Phase 3 MAGNITUDE-2 trial design of nex-z in ATTRv-PN was also exhibited in a poster presentation. Across patients who received a one-time dose of 0.3 mg/kg or higher, the mean serum TTR reduction by Day 28 was 90%, with levels remaining virtually unchanged for at least 24 months. Favorable trends indicating stability or improvement were observed in patients with ATTRv-PN, including six patients previously on patisiran for a mean of 5.5 years, who had evidence of disease progression prior to entering the study. Stability or improvement was based on evaluation of multiple clinical and biomarker measures, including Neuropathy Impairment Score, modified Neuropathy Impairment Score +7, modified BMI, Norfolk Quality of Life-Diabetic Neuropathy questionnaire and neurofilament light chain
