Cliramitug Phase 1 Long-Term Follow-Up Data Published in Nature Medicine
Neurimmune announced today that safety and efficacy data of the long-term follow up of the NI006-101 trial of cliramitug (formerly NI006, ALXN2220) for the treatment of amyloid transthyretin cardiomyopathy (ATTR-CM) have been published in Nature Medicine by Peter C. Kahr et al.1
ATTR-CM is a systemic and potentially life-threatening disease caused by the deposition of misfolded transthyretin (TTR), which presents with progressive heart failure. Despite advances in slowing disease progression, there is currently no available treatment option designed to deplete TTR from the heart to revert cardiac dysfunction.
Cliramitug is an investigational human monoclonal antibody designed to deplete amyloid deposits from the affected heart in ATTR-CM.2 In Study NI006-101, treatment with cliramitug for more than a year was not associated with apparent drug-related serious adverse events, and surrogate markers of cardiac amyloid load indicated cardiac ATTR depletion along with improvement of cardiac biomarkers and function.3
The new data demonstrated dose- and time-dependent effects on amyloid depletion, cardiac biomarkers and cardiac structure and function in participants who continued treatment in the NI006-101 open-label extensions. Continued treatment and up-titration in participants with lower prior exposure led to further reductions of cardiac amyloid load as measured by cardiac extracellular volume on MRI and tracer uptake on bisphosphonate scintigraphy. Improvements were also observed in cardiac biomarkers NT-proBNP and troponin T, and in left ventricular relaxation, filling pressures and wall thickness measured by echocardiography.
“The time- and dose-dependent improvements demonstrated across structural, functional and biomarker endpoints are consistent with our previous observations and support the therapeutic potential of cliramitug as a novel amyloid-depleting therapy for ATTR-CM,” said Christoph Hock, MD, Chief Medical Officer of Neurimmune. “Together with our clinical investigators and collaboration partners at Alexion, AstraZeneca Rare Disease, we are excited to share the detailed results of the Phase 1 open-label extensions with the broader scientific community.”
In 2022, Neurimmune entered into an exclusive global collaboration and license agreement with Alexion, AstraZeneca Rare Disease for cliramitug. Neurimmune is responsible for the Phase 1 study (NI006-101) and the Phase 2 follow-on study (NI006-102, EXCEL), on behalf of Alexion. Alexion is responsible for further clinical development, manufacturing, and commercialization of cliramitug, including the ongoing DepleTTR-CM Phase 3 trial to evaluate the safety and efficacy of cliramitug in adults with ATTR-CM.
1 Kahr et al., Nature Medicine 2026; in print. https://doi.org/10.1038/s41591-026-04487-3
2 Michalon et al., Nat Commun 2021; 12(1):3142.
3 Garcia-Pavia et al., N Engl J Med 2023; 389(3):239.
About Neurimmune
Neurimmune is a clinical-stage biopharmaceutical company building next-generation antibody therapeutics. The company pioneers amyloid depletion as a novel therapeutic mechanism to treat CNS and related protein aggregation diseases including Alzheimer’s disease, amyotrophic lateral sclerosis and ATTR cardiomyopathy. Neurimmune discovered the anti-ATTR antibody cliramitug (formerly NI006, ALXN2220, Phase 3) for ATTR cardiomyopathy, the anti-misfolded SOD1 antibody AP-101 (Phase 2) for ALS, the anti-NogoA antibody NG004 (Phase 1) for spinal cord injury and the anti-amyloid light chain antibody NI009 (preclinical) for AL amyloidosis.
Contact for Media
Cameron Hayes (US)
cameron.hayes@dgagroup.com
Martin Meier-Pfister (Switzerland)
media@neurimmune.com