Intellia Therapeutics has concluded patient enrolment in MAGNITUDE-2, a Phase III clinical study investigating nexiguran ziclumeran, known as nex-z, in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
The investigational in vivo CRISPR-based candidate works by disabling the TTR gene inside liver cells.
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This action prevents the formation of TTR protein, with the company aiming to halt or reverse disease progression after a single administration.
The double-blind, randomised, placebo-controlled trial recruited around 60 adult participants to assess the candidate’s safety and efficacy.
Participants were assigned on an equal basis to receive either a single 55mg infusion of nex-z or a placebo.
The study will track two primary outcomes: adjustments in serum TTR concentrations at day 29 and changes in the modified neuropathy impairment score at month 18.
Patients who reach the 18-month mark can opt to participate in a blinded crossover.
Intellia president and CEO John Leonard said: “We are grateful to...