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

Core Viewpoint - Intellia Therapeutics is conducting a Phase 3 study (MAGNITUDE) for its drug nexiguran ziclumeran (nex-z) in treating transthyretin amyloidosis with cardiomyopathy, with some patients experiencing severe liver enzyme elevations that are resolving without intervention [1][4]. Group 1: Study Progress and Patient Enrollment - The MAGNITUDE study has enrolled approximately 365 patients out of an expected total of 765, with completion of enrollment anticipated by early 2027 [2]. - Over 200 patients have been dosed with nex-z in the MAGNITUDE study [2]. - The company is also progressing well in the global Phase 3 MAGNITUDE-2 study of nex-z for ATTRv-PN, which aims to measure clinical outcomes and evaluate the drug's effect on serum TTR [5]. Group 2: Adverse Events and Safety Profile - Adverse events reported in the MAGNITUDE study have been similar to those in the Phase 1 study, including infusion-related reactions and asymptomatic liver transaminase elevations [3]. - The two reported grade 4 liver function test events have been asymptomatic, avoided hospitalization, and are likely to resolve without medication, indicating a positive risk/benefit outlook [4]. Group 3: Future Plans and Market Outlook - The company expects to submit a biologics license application (BLA) in the second half of 2026 to support a potential U.S. commercial launch in 2027 [5]. - Completion of the MAGNITUDE-2 study is expected by 2028 to support a potential BLA submission and a U.S. commercial launch in 2029 [6]. - HC Wainwright has reiterated a Buy rating for Intellia Therapeutics with a price target of $30 [6].