Intellia Therapeutics Announces First Patient Dosed in the MAGNITUDE-2 Phase 3 Study of Nexiguran Ziclumeran (nex-z), a One-Time Gene Editing-Based Treatment for Transthyretin (ATTR) Amyloidosis with Polyneuropathy

Core Insights - 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 [1][2] - The trial aims to evaluate the efficacy and safety of nex-z, which has the potential to redefine treatment for this debilitating disease [2][4] - The company plans to submit a biologics licensing application for ATTRv-PN by 2028, following promising Phase 1 data [3][5] Company Overview - Intellia Therapeutics is a clinical-stage gene editing company focused on CRISPR-based therapies, aiming to address significant unmet medical needs [7][8] - The company is developing nex-z as a one-time treatment for transthyretin amyloidosis, leveraging CRISPR/Cas9 technology [5][6] - Intellia has received Regenerative Medicine Advanced Therapy designations from the U.S. FDA for both cardiomyopathy and polyneuropathy indications [5] Clinical Trial Details - The MAGNITUDE-2 study is a randomized, double-blind, placebo-controlled trial involving approximately 50 patients [4] - The primary endpoints include a modified neuropathy impairment score and changes in serum TTR levels [4] - Patients will be randomized to receive either a single 55 mg infusion of nex-z or a placebo [4] Disease Context - Transthyretin amyloidosis (ATTR) is a rare and progressive disease, with hereditary forms (ATTRv) caused by mutations in the TTR gene [6] - There are an estimated 50,000 individuals with hereditary ATTR amyloidosis and between 200,000 to 500,000 with wild-type ATTR amyloidosis [6] - Current treatments are limited to slowing the accumulation of misfolded TTR proteins, with no known cure available [6]