Vifor Pharma reported positive phase-I trial results for its oral ferroportin inhibitor. The trial which was conducted amongst healthy volunteers over a period of 9 months, concluded in October 2018. Trial subjects received single oral doses of VIT-2763 (ferroportin inhibitor) from 5 mg to 240 mg, or multiple doses from 60 mg to 120 mg once or twice daily over 7 days. In all treated trial participants, the ferroportin inhibitor was well tolerated with mild to moderate side effects that were transient and self-limiting. The human pharmacokinetic profile exhibited dose-linear exposure, following which serum iron was lowered and remained below baseline values up to 24 hours post-dose. This iron-lowering pharmacodynamic effect is consistent with observations in preclinical models. Following these positive phase-I results, Vifor Pharma intends to start a phase-II proof-of-concept trial in the second half of 2019. This will be a phase-II randomized, controlled, multi-national trial conducted in patients with beta-thalassemia and documented iron overload. VIT-2763 is an oral inhibitor of ferroportin, the only known mammalian iron exporter and essential for transport of iron from one cell type to another. Preclinical models had already shown that VIT-2763 decreases serum iron levels in a dose-dependent manner1. Beta-thalassemia is an inherited, rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body potentially causing anaemia. Affected individuals may experience classic signs of anaemia including fatigue, weakness and shortness of breath. Severe forms can cause serious, even life-threatening complications if left untreated. Beta-thalassemia is often treated with blood transfusions which may lead to excess levels of iron in the body (iron overload). Treatment with VIT-2763 can improve iron metabolism and the production of red blood cells, erythropoiesis.