Intellia Therapeutics MAGNITUDE Trial Clinical Hold Lifted

Intellia Therapeutics announced that the U.S. Food and Drug Administration has removed the clinical hold on the Investigational New Drug application for the MAGNITUDE Phase 3 clinical trial of nexiguran ziclumeran for patients with transthyretin amyloidosis with cardiomyopathy. The clinical holds on the INDs for MAGNITUDE and MAGNITUDE-2 were imposed by the FDA on October 29, 2025, following the observation of Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in MAGNITUDE that met the trial's protocol-defined pausing criteria. The company has aligned with the FDA on mitigation measures for MAGNITUDE and MAGNITUDE-2 that include enhanced monitoring of liver laboratory tests, guidance for short-term steroid treatment if elevated liver transaminases are observed in the initial period following dosing and the exclusion of patients with certain liver abnormalities. For MAGNITUDE, additional exclusion criteria are being incorporated for patients with a recent history of cardiovascular instability and those with ejection fraction less than25% at the time of screening. Intellia is engaged with clinical trial investigators, ethics committees, international regulatory authorities and other stakeholders to resume enrollment activities in MAGNITUDE and MAGNITUDE-2. MAGNITUDE is a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of nex-z in approximately 1,200 patients with ATTR-CM. The primary endpoint of the trial is based on a composite measure of cardiovascular-related events, including mortality. Adult patients with ATTR-CM are randomized 2:1 to receive a single 55 mg infusion of nex-z or placebo