Intellia Therapeutics(NTLA) GlobeNewswire News Room·2025-04-03 11:30Core Viewpoint - Intellia Therapeutics has initiated the first patient dosing in the MAGNITUDE-2 Phase 3 trial for nexiguran ziclumeran (nex-z), targeting hereditary ATTR amyloidosis with polyneuropathy, which could potentially redefine treatment paradigms for this debilitating disease [1][2][3]. Group 1: Clinical Trial Details - The MAGNITUDE-2 study is a pivotal Phase 3, randomized, double-blind, placebo-controlled trial involving approximately 50 patients with ATTRv-PN [4]. - The primary endpoints include a modified neuropathy impairment score and changes in serum TTR levels, with patients randomized to receive either a single 55 mg infusion of nex-z or a placebo [4]. - Intellia aims to submit a biologics licensing application (BLA) for ATTRv-PN by 2028, following the presentation of longer-term data from Phase 1 studies later this year [3][4]. Group 2: Product Information - Nexiguran ziclumeran (nex-z) is based on CRISPR/Cas9 technology and is designed to inactivate the TTR gene responsible for producing the mutated transthyretin protein [5]. - Interim Phase 1 data indicated that a single dose of nex-z resulted in consistent, deep, and long-lasting reductions in TTR levels [5]. - Nex-z has received Regenerative Medicine Advanced Therapy designations from the U.S. FDA for both polyneuropathy and cardiomyopathy, as well as Orphan Drug Designation from both the U.S. FDA and European Commission [5]. Group 3: Disease Background - Transthyretin amyloidosis (ATTR) is a rare, progressive, and fatal disease, with hereditary ATTR (ATTRv) caused by mutations in the TTR gene leading to the production of abnormal TTR proteins [6]. - Approximately 50,000 individuals globally are affected by ATTRv amyloidosis, with no known cure and limited treatment options available to slow the progression of the disease [6].