Particle.news
Download on the App Store

Intellia Pauses Phase 3 Trials of CRISPR Therapy After Severe Liver Event

A protocol-triggering Grade 4 liver event in a recently dosed participant prompted regulatory engagement alongside expert review.

Overview

  • The company halted enrollment and dosing across the MAGNITUDE (ATTR-CM) and MAGNITUDE-2 (ATTR-PN) Phase 3 studies of nex-z.
  • An enrollee treated on Sept. 30 developed Grade 4 transaminase elevations with increased bilirubin reported on Oct. 24 and was hospitalized for medical care.
  • More than 650 patients are enrolled in MAGNITUDE and 47 in MAGNITUDE-2, with over 450 participants estimated to have already received nex-z.
  • Intellia is consulting external experts, engaging regulators, evaluating risk-mitigation steps, and adding more frequent lab monitoring while it investigates potential causes.
  • The update follows a separate Grade 4 liver event reported in May that did not require hospitalization, shares fell nearly 50% on Monday, and a webcast was scheduled for 8:30 a.m. ET.