Nexiguran Ziclumeran (nex-z) - (MAGNITUDE-2)

Nov. 4, 2026: Intellia Therapeutics (NASDAQ: NTLA) announced completion of enrollment in MAGNITUDE-2, a Phase 3 randomized, double-blind, placebo-controlled trial of nex-z (nexiguran ziclumeran) in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). About 60 adult patients were randomized 1:1 to a single 55 mg infusion of nex-z or placebo. The primary endpoints are change in modified neuropathy impairment score at month 18 and change in serum TTR levels at day 29; patients may opt into blinded crossover after month 18.

Nex-z is an investigational in vivo CRISPR/Cas9 therapy designed to inactivate the TTR gene in the liver and reduce transthyretin protein after a one-time treatment. The company also said nex-z is being studied in MAGNITUDE, a separate Phase 3 trial in transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

The release also notes nex-z has received FDA Orphan Drug and RMAT designations and European Commission Orphan Drug Designation. Intellia is leading development and commercialization under a collaboration with Regeneron Pharmaceuticals.