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Intellia Therapeutics
NEWS

Intellia Reports Long-Term P-I Trial Data of Nexiguran Ziclumeran for Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN)

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 NEJMSingle 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.…
September 26, 2025

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Intellia Therapeutics
NEWS

Intellia Therapeutics Reports the First Patient Dosing in P-III (MAGNITUDE-2) Trial of Nexiguran Ziclumeran for ATTRv-PN

Shots:Intellia has reported the first pts dosing in its P-III (MAGNITUDE-2) trial assessing nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN); BLA submission is expected in 2028Trial will assess nex-z (55mg infusion) vs PBO in ~50 ATTRv-PN pts to evaluate modified neuropathy impairment score & change in serum TTR levels as…
April 7, 2025

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AstraZeneca & Ionis
NEWS

AstraZeneca and Ionis’ Wainzua (Eplontersen) Receives the EC’s Approval to Treat Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

Shots:The EC has approved Wainzua to treat ATTRv-PN (stage 1/2 polyneuropathy) in adults pts based on P-III (NEURO-TTRansform) trial assessing eplontersen vs external PBO over 66wks., with follow-up to Wk. 85 & an end-of-trial evaluation. Eligible pts could then enter an ongoing OLE study Trial showed sustained benefits in co-1EPs of serum transthyretin…
March 11, 2025

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