Drugs

Intellia Gets Hold Lifted on MAGNITUDE Phase 3 Trial in ATTR-CM

FDA has lifted a clinical hold against Intellia Therapeutics and its MAGNITUDE Phase 3 trial of nexiguran ziclumeran (nex-z), an experimental CRISPR-based therapy for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The agency originally placed clinical holds on the MAGNITUDE and MAGNITUDE-2 trials in 10/2025 after a patient in MAGNITUDE experienced Grade 4 liver transaminase elevations and increased bilirubin, meeting the protocol-defined pausing criteria. Intellia says it has since worked with regulators to implement safety mitigation measures, including enhanced liver monitoring, short-term steroid protocols for elevated liver enzymes, and revised patient exclusion criteria. For MAGNITUDE, additional exclusions now cover patients with recent cardiovascular instability or ejection fraction below 25% at screening, it says.

“Aligning with the FDA on the path forward for MAGNITUDE allows us to continue evaluating nex-z in a broad ATTR-CM population while further enhancing patient safety,” the company says. “With the MAGNITUDE-2 hold already resolved in January for hereditary ATTR with polyneuropathy, we are focused on completing enrollment in both trials and are grateful to the investigators and patients involved.”

MAGNITUDE is a randomized, double-blind, placebo-controlled trial enrolling about 1,200 patients with ATTR-CM. Participants are randomized 2:1 to receive a single 55 mg infusion of nex-z or placebo. The trial’s primary endpoint is a composite measure of cardiovascular-related events, including mortality.

MAGNITUDE-2, targeting hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN), enrolls roughly 60 patients and evaluates efficacy via changes in modified neuropathy impairment score and serum transthyretin (TTR) levels. Patients are randomized 1:1 to nex-z or placebo.

Intellia says Nex-z leverages CRISPR/Cas9 gene editing to inactivate the TTR gene, aiming to provide a one-time therapy for ATTR-CM and ATTRv-PN.

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome