Leerink notes that Intellia Therapeutics (NTLA) announced a protocol-specified pause of Phase 3 MAGNITUDE and MAGNITUDE-2 studies of nex-z in ATTR, following report of G4 liver transaminases and increased total bilirubin in an ATTR-CM patient in his early 80s, treated September 30, 2025. The patient is hospitalized and being closely monitored. Management does not believe the safety signal is LNP-related given the onset timing and hypothesizes that it may be a delayed response specific to the targeted gene. If correct, this would be concerning for this program, but with more limited read-across to the broader pipeline/gene editing space, Leerink adds. While lonvo-z for hereditary angioedema is expected to launch first, nex-z represents a key driver for the firm’s model, and Leerink is not surprised to see shares down 40% on this headline. The firm has an Outperform rating on the shares.
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