Mitapivat

Mitapivat is an allosteric activator of erythrocyte pyruvate kinase (PKR). It increases PKR activity and ATP levels in red blood cells isolated from patients with pyruvate kinase deficiency when used at a concentration of 10 µM. Mitapivat increases hematocrit and hemoglobin levels, decreases osmotic fragility, and reduces splenic erythrophagocytosis in a model of hereditary spherocytosis using protein 4. knockout (4.2-/-) mice. Formulations containing mitapivat have been used in the treatment of hemolytic anemia in patients with pyruvate kinase deficiency. InChI: InChI=1S/C24H26N4O3S/c29-24(28-15-13-27(14-16-28)17-18-6-7-18)20-8-10-21(11-9-20)26-32(30,31)22-5-1-3-19-4-2-12-25-23(19)22/h1-5,8-12,18,26H,6-7,13-17H2, InChIKey: XAYGBKHKBBXDAK-UHFFFAOYSA-N, SMILES: O=C(N1CCN(CC2CC2)CC1)C3=CC=C(NS(C4=CC=CC5=CC=CN=C45)(=O)=O)C=C3