- Priority programs – NTLA-2002 for hereditary angioedema (HAE) and nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis – set foundation for significant, near-term value creation
- Phase 3 HAELO study evaluating NTLA-2002 for HAE to complete enrollment in the second half of 2025; Company plans to submit a Biologics License Application in the second half of 2026
- More than 550 patients expected to be enrolled by year end within the ongoing MAGNITUDE study for nex-z in ATTR-CM – the program remains ahead of internal enrollment estimates
- Pipeline priorities result in NTLA-3001 discontinuation and select, research-focused investment
- Anticipated cost savings, including a net workforce reduction of approximately 27% in 2025, support company operations into 1H 2027 and through anticipated, first commercial launch in the U.S.
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