Prothena announces review on birtamimab mechanism

Prothena Corporation announced the publication related to the mechanism of action, pharmacological characteristics, and clinical utility of birtamimab, a potential best-in-class anti-amyloid treatment for AL amyloidosis. The publication can be found in the latest issue of Leukemia & Lymphoma, an international peer-reviewed journal that publishes research on all aspects of hematological malignancies. AL amyloidosis is an idiopathic, rare, progressive and often fatal disease characterized by amyloid deposits that build up in vital organs leading to damage, dysfunction and failure. The risk of early mortality is especially high in patients with significant cardiac involvement as there are no current treatment options that target existing amyloid deposits in these patients. Birtamimab binds to a highly conserved cryptic epitope which is only exposed in misfolded kappa and lambda light chain protein. Birtamimab binds and neutralizes soluble, toxic LC aggregates and binds and clears insoluble AL amyloid deposits, without affecting normally folded LC proteins. This mechanism of action is complementary to the current standard of care therapy for AL amyloidosis which does not target the existing toxic LC aggregates and amyloid deposits. “Birtamimab was designed to clear deposited amyloid from vital organs and is the only treatment that has shown a significant survival benefit in patients with Mayo Stage IV AL amyloidosis, which was demonstrated in a post hoc analysis of the Phase 3 VITAL placebo-controlled clinical trial,” said Hideki Garren, M.D., Ph.D., Chief Medical Officer, Prothena. “Birtamimab is being evaluated in patients with Mayo Stage IV AL amyloidosis, who are at high risk of early mortality, in the ongoing confirmatory Phase 3 AFFIRM-AL clinical trial.”