Intellia Therapeutics, Inc. (NTLA) announced that the U.S. FDA has lifted the clinical hold on its MAGNITUDE-2 Phase 3 trial evaluating nexiguran ziclumeran (nex-z) in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), allowing the company to resume patient enrollment and dosing.
The phase 3 MAGNITUDE and MAGNITUDE-2 trials were originally placed on clinical hold last October, after a patient in the MAGNITUDE study experienced Grade 4 elevations in liver transaminases and increased total bilirubin, triggering protocol-defined pausing criteria. The FDA required Intellia to address the safety event and propose risk-mitigation steps before allowing the program to proceed.
MAGNITUDE is a phase 3 clinical trial evaluating the efficacy and safety of nex-z in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
Intellia has now aligned with the FDA on a series of study modifications and enhanced safety measures, including more frequent liver laboratory monitoring, which formed the basis for the FDA's decision to lift the hold for MAGNITUDE-2.
The company is working with investigators, ethics committees, and international regulators to restart enrollment as quickly as possible. Engagement with the FDA remains ongoing for the separate MAGNITUDE Phase 3 trial in ATTR-CM, which continues to remain on hold pending further alignment on the development path.
MAGNITUDE-2 is a randomized, double-blind, placebo-controlled Phase 3 study evaluating a single 55 mg infusion of nex-z in adults with ATTRv-PN. As part of the protocol amendment, the target enrollment has been increased from approximately 50 to 60 patients. The trial's primary endpoints include changes in modified neuropathy impairment score and serum transthyretin (TTR) levels.
Nex-z, based on CRISPR/Cas 9 gene-editing technology, is designed to permanently inactivate the TTR gene and has the potential to become the first one-time treatment for ATTRv-PN and ATTR-CM. Interim Phase 1 data previously showed deep and durable reductions in serum TTR levels following a single dose.
Nex-z holds Orphan Drug and RMAT designations from the FDA, as well as Orphan Drug Designation from the European Commission.
NTLA has traded between $5.90 and $28.25 over the past year. The stock closed yesterday's trading at $13.95, down 4.65% and is currently trading in the pre-market at $17.40, up 25%.
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