Agios Pharmac

Agios Pharmaceuticals, Inc., a biopharmaceutical company, discovers and develops medicines in the field of cellular metabolism in the United States. Its lead product includes PYRUKYND (mitapivat), an activator of wild-type and mutant pyruvate kinase (PK), enzymes used for the treatment of hemolytic anemias in adults with PK deficiency. The company’s PYRUKYND product is also used for the treatment of sickle cell disease that is in phase 3 clinical trial; for the treatment of PK deficiency in pediatric patients; and AQVESME for the treatment of adult patients with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. In addition, it developed tebapivat, a PK activator for the treatment of lower-risk myelodysplastic syndrome and sickle cell disease; AG-181, a phenylalanine hydroxylase stabilizer for the treatment of phenylketonuria (PKU); and AG-236, an siRNA in-licensed from Alnylam, targeting the transmembrane serine protease 6 (TMPRSS6) gene for the treatment of polycythemia vera.