The Vital Amyloidosis Study: A Randomized, Double-Blind, Placebo-Controlled, Global, Phase 3 Study Of Neod001 In Patients With Al Amyloidosis And Cardiac Dysfunction

TPS8614 Background: Light chain (AL) amyloidosis is a rare disease caused by the deposition of misfolded proteins that cause dysfunction of vital organs (eg, heart and kidneys). There are no approved therapies. Current therapeutic approaches target the plasma cells that produce the pathogenic light chain proteins and are typically associated with significant adverse effects. Therefore, there is a substantial need for a safe and effective therapy that specifically targets the misfolded light chain proteins responsible for the underlying organ dysfunction. NEOD001 is a monoclonal antibody that targets these misfolded proteins, and it is hypothesized to neutralize circulating soluble protein aggregates and clear insoluble aggregates from organs. In an ongoing phase I/II study in 27 patients with AL amyloidosis and persistent organ dysfunction, NEOD001 was safe and well tolerated, with monthly infusions producing meaningful decreases in cardiac and renal biomarkers by best response. Supported by these positiv...