Intellia announces ‘positive’ follow-up data from ongoing Phase 1 nex-z trial

Intellia Therapeutics (NTLA) announced “positive” follow-up data from the ongoing Phase 1 clinical trial of its investigational product nexiguran ziclumeran in patients with transthyretin amyloidosis with cardiomyopathy. Results were shared today in a late-breaking oral presentation at the American Heart Association Scientific Sessions 2025 in New Orleans, Louisiana. “These longer-term data showed a consistent and durable reduction in TTR and stability or improvement in multiple markers of cardiomyopathy following a single dose of nex-z. It’s remarkable that even in patients with advanced heart failure, a population that declines rapidly, disease stabilization or improvement was observed out to 24 months in a majority of participants. We look forward to seeing how these data mature in longer-term follow up. In addition, we are working diligently to address the ongoing clinical hold the FDA placed on our MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials,” said Intellia President and Chief Executive Officer John Leonard, M.D.