The FDA has lifted a clinical hold on Intellia Therapeutics’ Magnitude-2 phase 3 trial of nexiguran ziclumeran, or nex-z: a CRISPR-based gene therapy for patients with hereditary transthyretin amyloidosis with polyneuropathy.
The hold was initiated Oct. 29 following a serious safety event in a separate study, Magnitude, which is testing nex-z in patients with transthyretin amyloidosis with cardiomyopathy. A patient in that trial experienced grade 4 liver toxicity and elevated bilirubin after receiving the therapy Sept. 30 and later died. Intellia paused enrollment and dosing in both trials after the event.
After reaching alignment with the FDA on study modifications and enhanced liver safety monitoring, Intellia is resuming enrollment for Magnitude-2, according to a Jan. 27 news release. The study will now enroll about 60 patients, up from 50, and is evaluating a single 55-milligram dose of nex-z. Primary endpoints include changes in modified neuropathy impairment scores and serum TTR levels.
The Magnitude trial for cardiomyopathy remains on hold while FDA discussions continue, according to the drugmaker.
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