Intellia Therapeutics (NASDAQ:NTLA) announced today that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on its MAGNITUDE-2 Phase 3 clinical trial.
The decision allows the company to immediately resume patient enrollment and dosing for nexiguran ziclumeran (nex-z), a CRISPR-based investigational therapy for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
Intellia has aligned with the FDA on certain protocol modifications, including enhanced liver safety monitoring, and has increased the target enrollment from approximately 50 to approximately 60 patients to strengthen the study’s statistical power.
Engagement with the FDA remains ongoing regarding the related MAGNITUDE Phase 3 trial in transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
The company expects to provide an update on the path forward for that study following further alignment with regulators.
Nexiguran ziclumeran is an in vivo CRISPR-based gene editing therapy designed to knock out the TTR gene and reduce production of transthyretin protein, addressing the underlying cause of ATTR amyloidosis.
The lift of the clinical hold follows a voluntary pause and subsequent FDA review prompted by observations in earlier studies.
Intellia emphasized that the safety profile of nex-z remains consistent with prior data, and the modifications are intended to further ensure patient safety while advancing the program toward potential registration.
The company continues to prioritize the development of nex-z as a potential one-time treatment for ATTR amyloidosis and will provide additional updates on enrollment progress and timelines as the trials advance.