itacnosertib

Itacnosertib (TP-0184) is an orally bioavailable small molecule inhibitor that targets activin A receptor type 1 (ACVR1, also known as activin receptor-like kinase 2 or ALK2), as well as ALK5 and Janus kinase 2 (JAK2). It acts by binding to and inhibiting the activity of these serine/threonine receptor kinases, thereby blocking downstream signaling pathways such as BMP/SMAD. This inhibition leads to growth arrest in tumor cells overexpressing ACVR1/ALK2, particularly in FLT3-mutated acute myeloid leukemia (AML) and cancers with ACVR1 mutations such as diffuse intrinsic pontine glioma (DIPG). In preclinical models, TP-0184 has shown potential antineoplastic activity and can reduce hepcidin expression—restoring plasma iron levels—which may be beneficial for treating anemia of chronic disease. The drug is being developed primarily for the treatment of anemia in patients with low or intermediate risk myelodysplastic syndromes (MDS), but is also under investigation for pediatric brain tumors like DIPG[1][3][4][5][6][7].

Other names

1628870-27-8UNII-22Z53X5LHFUNII22Z53X5LHFUNII 22Z53X5LHF