Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, at Alexion, discusses positive data for an investigational treatment for ATTR with cardiomyopathy (ATTR-CM).
AATR-CM is a rare amyloidosis caused by aging or genetic mutations in TTR protein. This results in the misfolding of TTR protein and accumulation of amyloid fibers in the cardiac myocardium. TTR fibrils build up in tissues, such as the peripheral nerves and heart, as well as the gastrointestinal tract, eyes, kidneys, central nervous system, thyroid, and bone marrow. This accumulation causes progressive dysfunction of these tissues. The disease eventually leads to heart failure.
ATTR-CM is frequently misdiagnosed due to the commonality of its symptoms with other diseases. It is common for the disease to go undiagnosed until significant damage has been done to the affected tissues.
ALXN2220 is an investigational human monoclonal antibody that targets misfolded TTR, addressing the underlying pathology of ATTR-CM. The therapy enables the removal of fibril deposits in the heart in combination with standard of care treatments. ALXN2220 has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) following results from a phase 1 trial evaluating the therapy. Within this trial, the therapy illustrated no serious adverse events or safety signals. Additionally, it was observed that cardiac tracer uptake and extracellular volume were reduced on doses of at least 10 mg per kilogram. Median N-terminal pro-B-type natriuretic peptide and troponin T levels were also reduced.
A phase 3 trial evaluating the clinical benefit of ALXN2220 as a standard of care treatment for ATTR-CM is ongoing. The primary endpoint is a composite of all-cause mortality and the cumulative frequency of cardiovascular clinical events. For more information, click here.
To learn more about rare cardiovascular disease, visit https://checkrare.com/diseases/heart-diseases/