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 the patients, caregivers and investigators who are participating in MAGNITUDE-2.
“Completing enrolment in this trial is a pivotal step toward bringing a potentially transformative one-time treatment to people living with ATTRv-PN, a debilitating and life-threatening disease with significant unmet need.”
The therapy is also under clinical assessment in MAGNITUDE, a separate Phase III randomised, double-blind, placebo-controlled trial involving patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
Intellia directs research, development, and commercialisation efforts for nex-z under a multi-target partnership with Regeneron Pharmaceuticals.
The therapy received orphan drug designation and regenerative medicine advanced therapy designation from the US Food and Drug Administration (FDA), alongside an orphan drug designation from the European Commission.
In March, the FDA released the clinical hold on a second trial evaluating Intellia Therapeutics’ ATTR-CM gene therapy.
