Neurimmune today announced that the translational PK/PD model relating to the human antibody ALXN2220 (formerly NI006) have been published in the current issue of Clinical Pharmacology & Therapeutics. The manuscript “Prediction of cardiac ATTR depletion by NI006 (ALXN2220) using mechanistic PK/PD modeling” describes the mechanistic pharmacokinetic and pharmacodynamic model used to predict drug exposure and its effects on cardiac transthyretin amyloid load.1
ALXN2220 is an investigational human monoclonal antibody designed to deplete amyloid deposits in transthyretin amyloidosis with cardiomyopathy (ATTR-CM).2,3 The mechanistic PK/PD model described by Michalon et al. provided important predictions to select a safe and potentially efficacious dose range and treatment duration for a proof-of-concept clinical Phase 1b study of ALXN2220. The model enabled the integration of preclinical results into the clinical trial design and provided predictions that were in good agreement with the observed primary results of the Phase 1b study recently published in The New England Journal of Medicine.
“We are excited about the predictive accuracy of our model which supported the translation of our preclinical findings on ALXN2220-mediated TTR amyloid depletion into the design of a successful Phase 1b clinical trial of ALXN2220,” said Jan Grimm, PhD, Chief Scientific Officer of Neurimmune. “We would like to thank Alexion and Lyo-X for the close collaboration and look forward to Alexion’s continued clinical development of this novel treatment with the potential to transform the course of ATTR amyloidosis.”
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.
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. Aside from the Phase 1b trial, Alexion is responsible for further clinical development, manufacturing, and commercialization of ALXN2220. Alexion, AstraZeneca Rare Disease, is currently conducting the Phase 3 DepleTTR-CM clinical study to assess the efficacy and safety of ALXN2220 as an add-on treatment to the standard of care for patients with ATTR-CM.
1 Michalon et al., Clin Pharm Ther 2024; in press, doi:10.1002/cpt.3455
2 Garcia-Pavia et al., N Engl J Med 2023
3 Michalon et al., Nat Commun 2021; 12(1):3142
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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john.capodanno@dentonsglobaladvisors.com
Martin Meier-Pfister (Switzerland)
media@neurimmune.com
