Intellia reports grade 4 event in late-stage ATTR amyloidosis trial

Intellia Therapeutics revealed that a patient had experienced a grade 4 adverse event in its global phase 3 MAGNITUDE study of CRISPR-based candidate, nex-z, for patients with ATTR amyloidosis with cardiomyopathy.

In an SEC filing, Intellia said that while over two hundred patients have been dosed with nex-z in the MAGNITUDE study, there has been a single, recent, asymptomatic patient with grade 4 liver transaminase elevations based on laboratory tests. According to Intellia, the issues “appear to be resolving without hospitalization or medical intervention and have fallen to grade 3 ALT and grade 2 AST elevations.”

Intellia is continuing to enroll patients in the trial, anticipating enrollment completion by early 2027.

Additionally, Intellia reported that its phase 3 MAGNITUDE-2 study of nex-z in hereditary transthyretin amyloidosis with polyneuropathy is on track, with the company expecting to complete the study to support a potential BLA submission by 2028 and potential U.S. commercial launch in 2029.

Earlier this year, Intellia announced a strategic reorg in order to advance its two late-stage candidates, nex-z and NTLA-2002, which is currently being evaluated for hereditary angioedema in the phase 3 HAELO study. The reorg will result in a workforce reduction of approximately 27% over the course of 2025.