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Intellia Therapeutics Announces First Patient Dosed in the MAGNITUDE-2 Phase 3 Study of Nexiguran Ziclumeran (nex-z), a One-Time Gene Editing-Based Treatment for Transthyretin (ATTR) Amyloidosis with Polyneuropathy
CAMBRIDGE, Mass., April 03, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., April 03, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the first patient has been dosed in MAGNITUDE-2, a global, pivotal Phase 3 trial of nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). “We are pleased to have dosed the first patient with a treatment that has such strong potential to redefine the treatment paradigm for those living with ATTR with polyneuropathy. This is a debilitating, progressive disease that leaves people feeling increasingly helpless,” said Dr. Paulo Sgobbi, Medical Director, PSEG Clinical Research Center. “Through nex-z’s potential to favorably impact disease progression, patients living with ATTR polyneuropathy could experience life-changing benefit while being freed from the existing chronic treatment regimen of pills, injections and infusions.” “This milestone marks important progress toward our goal of completing the MAGNITUDE-2 clinical program and we are optimistic the study will enable us to demonstrate nex-z’s potential to be the first to halt or reverse disease progression with a single dose in hereditary ATTR with polyneuropathy,” said Intellia President and Chief Executive Officer John Leonard, M.D. The Phase 3 MAGNITUDE-2 study is informed by Intellia’s Phase 1 data, showing that a single dose of nex-z led to consistently rapid, deep and durable reduction in serum TTR. Intellia expects to present longer-term data from the Phase 1 studies of nex-z for both polyneuropathy and cardiomyopathy later this year. The company plans to submit a biologics licensing application (BLA) for ATTRv-PN by 2028. About the MAGNITUDE-2 StudyThe pivotal Phase 3 MAGNITUDE-2 clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nexiguran ziclumeran (nex-z) in approximately 50 patients with transthyretin amyloidosis with polyneuropathy (ATTRv-PN). The primary endpoint of the study includes a modified neuropathy impairment score and change in serum TTR levels. Adult patients with ATTRv-PN will be randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo. For more information on MAGNITUDE-2 (NCT06672237), please visit clinicaltri...