Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (nex-z)

Core Viewpoint - Intellia Therapeutics has temporarily paused patient dosing and screening for its Phase 3 clinical trials of nex-z due to safety concerns following a serious adverse event in a trial participant [1][2][3] Company Actions - The company is consulting with experts and regulatory authorities to develop a strategy for resuming enrollment in the MAGNITUDE and MAGNITUDE-2 trials [2][3] - As of now, over 650 patients with ATTR-CM are enrolled in the MAGNITUDE trial, and 47 patients with ATTR-PN are enrolled in the MAGNITUDE-2 trial, with more than 450 patients estimated to have been dosed with nex-z [3] Product Information - Nex-z is based on CRISPR/Cas9 gene editing technology and aims to be the first one-time treatment for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and/or polyneuropathy (ATTR-PN) [5] - The drug is designed to inactivate the TTR gene and has shown promising interim Phase 1 clinical data indicating consistent and long-lasting TTR reduction [5] Regulatory Status - Nex-z has received Orphan Drug and RMAT Designation from the FDA and Orphan Drug Designation from the European Commission [5]