PN-8047

PN-8047 is an orally bioavailable small molecule hepcidin functional mimetic currently in pre-clinical development by Protagonist Therapeutics. As a mimetic of the peptide hormone hepcidin, it is designed to regulate iron homeostasis by binding to ferroportin, the sole cellular iron exporter, leading to its internalization and degradation. This mechanism effectively restricts iron absorption from the diet and the release of recycled iron from macrophages, thereby lowering systemic iron levels. PN-8047 is being developed as a potential oral alternative or complement to injectable hepcidin mimetics like rusfertide for the treatment of iron overload disorders and conditions characterized by excessive erythropoiesis, such as polycythemia vera and hereditary hemochromatosis.