Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1056/NEJMc2309584 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Prediction of Cardiac ATTR Depletion by NI006 (ALXN2220) Using Mechanistic PK/PD Modeling.
Clin Pharmacol Ther
January 2025
Neurimmune, Schlieren, Switzerland.
NI006 (aka ALXN2220) is a therapeutic antibody candidate in phase III clinical development for the depletion of amyloid transthyretin (ATTR) in patients with ATTR cardiomyopathy, an infiltrative cardiomyopathy leading to increased left ventricular wall thickness (LVWT). The mode-of-action consists in removal of disease-causing amyloid accumulations by activating phagocytic immune cells, a mechanism without precedent in cardiology. To select a safe and potentially efficacious dose range and treatment duration for a combined first-in-human and proof-of-concept clinical phase Ib study, we developed a mechanistic pharmacokinetic and pharmacodynamic (PK/PD) model that can predict NI006 exposure, its effects on cardiac amyloid load and on LWVT, which is a predictor of heart failure in this disease.
View Article and Find Full Text PDFRecent advances in the diagnostic methods and therapeutic strategies of transthyretin cardiac amyloidosis.
World J Cardiol
July 2024
Department of Cardiology, School of Medicine, University Hospital of Larissa, Larissa 41110, Greece.
Article Synopsis
- Cardiac amyloidosis is a serious condition where abnormal amyloid fibrils accumulate in the heart, causing it to function poorly. It's mainly classified into two types: immunoglobulin light chain amyloidosis and transthyretin amyloidosis (ATTR), which comes in non-hereditary and hereditary forms.
- The rise in cases of ATTR cardiac amyloidosis is partly due to better diagnostic methods, and survival rates are improving thanks to new treatment options like tafamidis and emerging therapies currently in trials.
- New treatments such as acoramidis, eplontersen, vutrisiran, patisiran, and an experimental antibody NI006 are being tested to enhance cardiac function, with results expected in a few
Antibody NI006 for Cardiac Transthyretin Amyloid Depletion. Reply.
N Engl J Med
September 2023
Neurimmune, Schlieren, Switzerland.
Antibody NI006 for Cardiac Transthyretin Amyloid Depletion.
N Engl J Med
September 2023
St. Bartholomew's Hospital, London, United Kingdom
Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid.
N Engl J Med
July 2023
From Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV, and Centro Nacional de Investigaciones Cardiovasculares, Madrid, and Universidad Francisco de Vitoria, Pozuelo de Alarcón - all in Spain (P.G.-P.); the Department of Cardiology, University Hospital Heidelberg, Heidelberg (F.S.), and Cardiovascular Center Darmstadt, Darmstadt (A.V.K.) - both in Germany; the Department of Cardiology, University of Rennes, Centre Hospitalier Universitaire (CHU) de Rennes, INSERM, LTSI-UMR 1099, Rennes (E.D.), Service de Cardiologie, CHU de Toulouse-Hôpital Rangueil, Toulouse (O.L.), and the Cardiology Department and French National Reference Center for Cardiac Amyloidosis, Hôpitaux Universitaires Henri Mondor, Assistance Publique-Hôpitaux de Paris, and Institut Mondor de Recherche Biomédicale, INSERM, Université Paris Est Créteil, Créteil (T.D.) - all in France; the Department of Cardiology, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands (P.M.); Alexion-AstraZeneca Rare Disease, Boston (M.F.M.); and Neurimmune (A.M., R.J.A.F., J.G., R.M.N., C.H., P.C.K.) and the Institute for Regenerative Medicine (R.M.N., C.H.) and the Center for Molecular Cardiology (P.C.K.), University of Zurich - all in Schlieren, Switzerland.
Article Synopsis
- Transthyretin amyloid (ATTR) cardiomyopathy is a serious heart disease caused by misfolded proteins, and current treatments do not effectively remove these proteins from the heart.
- A phase 1 clinical trial tested NI006, a recombinant human antibody, on 40 patients to evaluate its safety and effects on heart function, using a double-blind method with varying doses.
- Results showed no serious side effects, and at higher doses, NI006 appeared to reduce markers of cardiac amyloid load and improve heart-related indicators over 12 months.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!