KER-047

KER-047 is an investigational, oral small molecule designed to selectively and potently inhibit ALK2 (activin receptor-like kinase 2, also known as ACVR1), a member of the Transforming Growth Factor-Beta (TGF-β) receptor family. Developed by Keros Therapeutics, it was intended for the treatment of diseases related to iron imbalance such as anemia—including iron deficiency anemia and IRIDA (iron-refractory iron deficiency anemia)—as well as fibrodysplasia ossificans progressiva (FOP), a rare musculoskeletal disorder. The drug works by inhibiting ALK2 signaling, which reduces elevated hepcidin levels and improves iron absorption and recycling in conditions like IRIDA. Clinical studies showed that administration of KER-047 led to decreased serum hepcidin and ferritin with increased serum iron and transferrin saturation[1][3][4][6][8]. However, development has been discontinued for all indications.