Intellia Therapeutics(US:NTLA) Globenewswireยท2025-09-25 13:52Core Insights - Intellia Therapeutics announced promising long-term follow-up data from the Phase 1 study of investigational gene therapy nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) [1][2] - The results indicate significant and durable reductions in serum TTR levels, suggesting potential for improved patient outcomes [2][3] Group 1: Clinical Results - A one-time dose of nex-z led to a mean serum TTR reduction of 92% at 24 months and 90% at 36 months among patients [3][4] - Among 18 patients assessed at 24 months, 72% showed clinically meaningful improvements in the modified Neuropathy Impairment Score (mNIS+7) [5] - Secondary endpoints such as modified body mass index (mBMI), Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN), and neurofilament light chain (NfL) also trended towards improvement [5] Group 2: Safety Profile - Nex-z has been generally well tolerated, with mild to moderate infusion-related reactions being the most common adverse events [6] - No new drug-related events were reported during the follow-up period, and previously reported liver enzyme elevations resolved without intervention [6][8] Group 3: Future Developments - The Phase 3 MAGNITUDE-2 trial is progressing rapidly, with patient enrollment expected to complete in the first half of 2026 [7][8] - Intellia anticipates submitting a biologics license application (BLA) for nex-z by 2028 [7][8] - The trial aims to evaluate the efficacy and safety of nex-z in approximately 50 patients with ATTRv-PN [7][9] Group 4: About Nex-z and ATTR Amyloidosis - Nex-z is based on CRISPR/Cas9 technology and aims to inactivate the TTR gene, potentially becoming the first one-time treatment for ATTR amyloidosis [11] - ATTR amyloidosis is a rare and progressive disease affecting an estimated 50,000 people with hereditary forms and between 200,000 to 500,000 with wild-type forms [12]