Shots:
- Intellia has reported P-I trial data assessing nexiguran ziclumeran (nex-z) in ATTRv-PN; results were presented at the International ATTR Amyloidosis Meeting 2025 & published in The NEJM
- Single dose of nex-z achieved mean TTR reductions of 92% at 24mos. & 90% in 12 pts followed for 36mos. At 24mos., 13/18 pts met ≥4-point mNIS+7 improvement, incl. most progressed on patisiran. Across all 36 pts, mBMI, QoL-DN & NfL trended toward improvement & 89% showed improved or stable PND scores over 24mos.
- The P-III (MAGNITUDE-2) trial started dosing in Apr 2025 & is rapidly enrolling ~50 pts, with enrollment expected to be complete by H1’26 & a BLA submission for ATTRv-PN planned by 2028
Ref: Intellia Therapeutics | Image: Intellia Therapeutics | Press Release
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