Intellia Therapeutics' Rare Heart Disease Candidate Reports Liver Enzyme Spike In Phase 3 Trial, But Stays On Course

Intellia Therapeutics Inc NTLA said on Wednesday that a single, recent, asymptomatic patient has had grade 4 (severe) liver transaminase elevations (liver enzymes) based on laboratory tests in its Phase 3 MAGNITUDE study.

The trial is evaluating nexiguran ziclumeran (nex-z, also known as NTLA-2001) in transthyretin amyloidosis with cardiomyopathy.

These elevations appear to be resolving without hospitalization or medical intervention and have fallen to grade 3 ALT and grade 2 AST elevations.

The company has enrolled approximately 365 patients in the MAGNITUDE study out of an expected total enrollment of approximately 765.

The company expects to complete enrollment by early 2027. Over 200 patients have been dosed with nex-z in the MAGNITUDE study.

Also Read: Why Is Intellia Therapeutics Stock Trading Lower On Friday?

In an SEC filing, the company reported that adverse events have been similar to those reported in the Phase 1 study of Nex-z for ATTR amyloidosis and have included infusion-related reactions and asymptomatic liver transaminase elevations.

William Blair writes, “We remain positive on the risk/benefit given the two reported grade 4 LFT (liver function test) events with nex-z have been asymptomatic to date, avoided hospitalization, and are likely to resolve without medication, in our view.”

The regulatory filing states that enrollment in the global Phase 3 HAELO study of NTLA-2002 for hereditary angioedema is on track. 

The company expects to complete enrollment in the third quarter of 2025 and submit a biologics license application (BLA) in the second half of 2026 to support plans for a potential U.S. commercial launch in 2027.

Enrollment in the global Phase 3 MAGNITUDE-2 study of nex-z for ATTRv-PN is also progressing well. The study is designed to measure clinical outcomes and evaluate how a single dose of Nex-z can reduce serum TTR.

Intellia expects to complete MAGNITUDE-2 by 2028 to support a potential BLA submission and a potential U.S. commercial launch in 2029.

HC Wainwright reiterated Intellia Therapeutics with a Buy and maintains a $30 price target.

Price Action: At the last check on Thursday, NTLA stock was down 22.6% at $7.48 during the premarket session.

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