Rusfertide is an injectable hepcidin mimetic peptide that is currently under clinical investigation for polycythema vera (PV). Rusfertide acts by binding to the iron exporter Ferroportein (FPN), inducing internalization and proteolysis of the transmembrane protein and consequently limiting or preventing iron release. Hepcidin mimetic therapies will benefit disease indications influenced by the modulation of iron homeostasis (e.g., Polycythemia Vera- PV) and in conditions with aberrant iron regulation (e.g., Hereditary Hemochromatosis- HH).
Oral hepcidin mimetic drugs may offer alternate route of administration for patients with chronic disease conditions.1 Herein we describe a new class of oral hepcidin mimetic peptides with improved systemic pharmacodynamic activity in regulating serum iron parameters.
[1]. Ginzburg YZ, Leukemia 2018; Taranath R, Blood 2020 136 Suppl 1: 49