VIT-2763, an oral ferroportin inhibitor, inhibits hepcidin binding to ferroportin and blocks iron efflux. VIT-2763 has the potential in the treatment of β-thalassemia. In Vitro VIT-2763 dose dependently reduces the fluorescence polarization signal, indicating that VIT-2763 displaces TMR-hepcidin from ferroportin (IC 50 of 24 ± 13 nM). VIT-2763 induces BLA reporter gene activity with an average EC 50 of 140 ± 50 nM, as a consequence of increasing intracellular iron concentrations caused by blocked iron export in HEK293 cells. VIT-2763 (100 nM) triggers ubiquitination and subsequent internalization and degradation of ferroportin. MCE has not independently confirmed the accuracy of these methods. They are for reference only. Cell Viability Assay. Cell Line: J774 cells. Concentration: 100 nM. Incubation Time: 10, 20, 40, 60, or 120 minutes. Result: Induced ferroportin internalization and ubiquitination. In Vivo VIT-2763 (30, 100 mg/kg, orally twice daily for 36 days) decreases serum iron and prevented liver iron loading in Hbb th3/+ mice . VIT-2763 did not change the total liver iron . VIT-2763 (30, 100 mg/kg, orally twice daily for 36 days) significantly corrects anemia and improved RBC parameters in Hbb th3/+ mice. VIT-2763 decreases the percentage of ROS-positive RBCs in Hbb th3/+ mice from 67% to 30% . VIT-2763 decreases apoptosis and extends the life span of RBCs in Hbb th3/+ mice . MCE has not independently confirmed the accuracy of these methods. They are for reference only. Animal Model: Hbb th3/+ mice . Dosage: 30, 100 mg/kg. Administration: Orally twice daily for 36 days. Result: Significantly decreased serum iron levels by 77% (30 mg/kg) and 84% (100 mg/kg), Significantly increased Hb levels (as of day 8 of treatment), RBC counts, mean corpuscular Hb concentration (MCHC), and significantly lowered reticulocyte counts, mean corpuscular Hb (MCH), mean corpuscular volume (MCV), and RBC distribution width (RDW) in Hbbth3/+ mice, as compared with the Hbbth3/+ vehicle group. Form:Solid