Rusfertide is a peptide mimetic of natural hepcidin, which targets and degrades ferroportin, reduces serum iron and transferrin-saturation, and thus regulates the production of red blood cells. Rusfertide ameliorates the polycythemia vera, β-thalassemia and hereditary hemochromatosis.
Rusfertide limits the iron toxicity in red blood cells (RBCs) (1 mg/kg, s.c., once every two days, for 49 days) and transferrin-saturation (2.5 mg/kg, s.c., once every two days, for 2 weeks), improves oxygen carrying capacity of RBCs, attenuates the anemia and iron deposition in mice models for β-thalassemia and hereditary hemochromatosis[1].
References:
[1]. Taranath R, et al., Regulation of iron homeostasis by PTG-300 improves disease parameters in mouse models for beta-thalassemia and hereditary hemochromatosis[J]. Blood, 2019, 134: 3540. [2]. Kremyanskaya M, et al., PTG-300 eliminates the need for therapeutic phlebotomy in both low and high-risk polycythemia vera patients[J]. Blood, 2020, 136: 33-35.