Intellia Therapeutics Announces FDA Lift of Clinical Hold on MAGNITUDE-2 Phase 3 Clinical Trial in ATTRv-PN

Core Viewpoint - Intellia Therapeutics has announced the lifting of the clinical hold by the FDA on the IND for the MAGNITUDE-2 Phase 3 clinical trial of nexiguran ziclumeran (nex-z) for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) [1][2] Group 1: FDA Engagement and Clinical Trials - The FDA's clinical hold on the IND for MAGNITUDE-2 was lifted, allowing Intellia to resume patient enrollment [1][2] - The clinical hold was initially imposed on October 29, 2025, due to safety concerns related to liver transaminases and bilirubin levels in a patient [4] - Intellia is also in ongoing discussions with the FDA regarding the clinical hold on the MAGNITUDE trial for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) [3] Group 2: Trial Details and Modifications - MAGNITUDE-2 is a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of nex-z, with an increased target enrollment from approximately 50 to 60 patients [5] - The primary endpoints of MAGNITUDE-2 include changes in modified neuropathy impairment score and serum TTR levels [5] - Enhanced safety monitoring measures have been implemented as part of the protocol amendment for MAGNITUDE-2 [4] Group 3: About Nex-z and Intellia Therapeutics - Nex-z is based on CRISPR/Cas9 gene editing technology and aims to be the first one-time treatment for ATTR amyloidosis [6] - Interim Phase 1 clinical data indicated that nex-z led to consistent and long-lasting TTR reduction [6] - Intellia Therapeutics focuses on developing novel medicines using gene editing technology to address unmet medical needs [7]