FDA Lifts Clinical Hold on Intellia's ATTR Cardiomyopathy Trial
The FDA removed the clinical hold on Intellia Therapeutics' Phase 3 trial of nexiguran ziclumeran for transthyretin amyloidosis with cardiomyopathy. The hold was imposed in October following liver safety concerns in one patient.
The Food and Drug Administration lifted the clinical hold on the investigational new drug application for Intellia Therapeutics' late-stage trial of a treatment for patients with transthyretin amyloidosis with cardiomyopathy. In premarket trading, Intellia's shares were up 8.9% to $15.01. The stock had already rallied 70% over the last three months.
The FDA removed the clinical hold on the application for the Phase 3 clinical trial of nexiguran ziclumeran. The study is a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of nex-z in about 1,200 patients with transthyretin amyloidosis with cardiomyopathy.
The FDA in late October imposed clinical holds on the investigational new drug application for Intellia's Magnitude trial and Magnitude-2, a randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of nex-z in about 60 patients with hereditary ATTR amyloidosis with polyneuropathy. The pause followed the observation of grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in Magnitude.
The company said it has aligned with the FDA on mitigation measures for the trials 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.