FDA places clinical holds on nex-z Phase 3 trials after a Grade 4 liver transaminase event

Intellia Therapeutics/Nex-z (NTLA-2001)

Intellia disclosed that the FDA verbally placed clinical holds on both MAGNITUDE and MAGNITUDE-2 Phase 3 trials of nex-z following Grade 4 liver transaminase elevations and elevated bilirubin in a MAGNITUDE trial patient. Intellia had already paused dosing on October 27.

• Holds placed on both MAGNITUDE (cardiomyopathy) and MAGNITUDE-2 (polyneuropathy) trials.
• Triggering event: Grade 4 liver transaminases and elevated total bilirubin in one MAGNITUDE patient.
• Company had initiated a dosing pause on October 27 before the FDA's verbal hold notice.
• nex-z (nexiguran ziclumeran) is an in vivo CRISPR therapy for transthyretin amyloidosis.