Intellia Therapeutics Receives FDA Clearance to Resume Key Clinical Trial

Intellia Therapeutics has achieved a significant regulatory milestone, with the U.S. Food and Drug Administration (FDA) lifting a clinical hold on one of its crucial late-stage programs. This development allows the company to move forward with a pivotal Phase 3 study, though certain restrictions remain in place and a related trial continues to be paused.

The biotech firm announced this week that the FDA has removed the clinical hold on its MAGNITUDE-2 trial. This Phase 3 study is evaluating the investigational therapy nexiguran ziclomeran (nex-z) for treating patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN), a rare and progressive genetic nerve disorder.

With the hold now lifted, Intellia plans to restart patient enrollment "as quickly as possible." The regulatory clearance, however, comes with modified trial parameters. The company has agreed to implement enhanced liver function monitoring as an additional safety measure. Furthermore, the target enrollment for the study has been increased from approximately 50 to around 60 patients.

Background on the FDA's Initial Action

The regulatory agency originally imposed the clinical hold in late October 2025. The action affected two studies involving the same drug candidate. Alongside MAGNITUDE-2, the related MAGNITUDE trial was also paused. That study is testing nex-z for a different manifestation of the disease: transthyretin amyloidosis with cardiomyopathy (ATTR-CM), which involves heart complications.

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The hold was triggered by a serious adverse event reported in the MAGNITUDE trial, where one participant developed severe liver toxicity and subsequently died. While the FDA has now granted clearance for the MAGNITUDE-2 study (polyneuropathy), the MAGNITUDE trial (cardiomyopathy) remains on hold. Intellia stated that discussions with the agency regarding the path forward for that study are ongoing.

Market Implications and Sector Significance

The partial resumption is viewed as a positive step not only for Intellia but for the broader gene-editing sector, where regulatory decisions concerning novel technologies like CRISPR are closely scrutinized. The restart of this Phase 3 program refocuses attention on nex-z as a potential one-time treatment for ATTRv-PN.

The market responded favorably to the news: The company's share price advanced following the announcement. Nonetheless, the continued suspension of the second trial arm (ATTR-CM) represents a remaining area of uncertainty for investors and the company's development pipeline.

Key Trial Updates:

• The MAGNITUDE-2 Phase 3 study clinical hold has been lifted by the FDA.
• Patient recruitment is slated to recommence at the earliest opportunity.
• Stricter safety protocols focusing on liver values will be enforced.
• The study's participant target grows from about 50 to 60 patients.
• A second, connected study (MAGNITUDE for cardiac involvement) remains paused.