Neurimmune today announced that the U.S. Food and Drug Administration (FDA) has granted Alexion, AstraZeneca Rare Disease Fast Track designation for the development of ALXN2220 (formerly NI006) for the treatment of transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
ATTR-CM is a systemic, progressive and life-threatening disease caused by the deposition of misfolded transthyretin (TTR), which can lead to heart failure. Despite advances in slowing disease progression, there is no available treatment option designed to deplete TTR from the heart to revert cardiac dysfunction. The Fast Track designation was granted based on the results of a Phase 1b trial conducted by Neurimmune and published in The New England Journal of Medicine in 2023.1
"In our Phase 1b trial intravenously administered ALXN2220 reduced cardiac tracer uptake and extracellular volume. The reduction of these surrogate markers of cardiac amyloid load suggests that ALXN2220 can induce the depletion of cardiac amyloid deposits by the immune system. This is a novel therapeutic mechanism with potential to restore cardiac function in patients suffering from ATTR amyloidosis,” said Christoph Hock, MD, Chief Medical Officer of Neurimmune. “We look forward to Alexion’s continued development of ALXN2220 and are very pleased the FDA’s Fast Track designation will help accelerate this effort.”
ALXN2220 is an investigational human monoclonal antibody currently being evaluated in the Phase 3 DepleTTR-CM trial to assess its efficacy as an add-on treatment to the standard of care for patients with ATTR-CM. The primary endpoint is a composite of all-cause mortality and cumulative frequence of cardiovascular clinical events.
The Fast Track designation is granted to expedite the review of drugs to treat serious conditions and address an unmet medical need. The designation allows for more frequent interactions with the FDA, and potentially in the future, Accelerated Approval and Priority Review in the U.S.
In 2022, Neurimmune entered into an exclusive global collaboration and license agreement with Alexion, AstraZeneca Rare Disease for ALXN2220. Neurimmune is responsible for the completion of the Phase 1b clinical study on behalf of Alexion, with Alexion incurring certain trial costs. Aside from the Phase 1b trial, Alexion is responsible for further clinical development, manufacturing, and commercialization of ALXN2220.
1Garcia-Pavia et al., N Engl J Med 2023
About Neurimmune
Neurimmune is a biopharmaceutical company translating human immune memory into antibody therapeutics. Neurimmune develops drug candidates for CNS and related protein aggregation diseases including Alzheimer’s disease, amyotrophic lateral sclerosis, frontotemporal dementia and ATTR cardiomyopathy. With its Reverse Translational MedicineTM technology, Neurimmune discovered the brain amyloid depleter NI101SQ for Alzheimer’s disease, anti-SOD1 antibody AP-101 for ALS and the anti-ATTR antibody ALXN2220 (formerly NI006) for ATTR cardiomyopathy, programs being currently evaluated in clinical trials.
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