Agios' blood disorder drug meets goals of late-stage study

Agios Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is focused on developing and delivering transformative therapies for patients living with rare diseases. It markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for debilitating hemolytic anemia. Its lead product candidate in its genetically defined disease (GDD) portfolio, PYRUKYND (Mitapivat), is an activator of both wild-type and a variety of mutant pyruvate kinase (PK) enzymes, for the treatment of hemolytic anemias. The Mitapivat is an orally available small molecule and a potent activator of the pyruvate kinase R (PKR) enzymes. In addition, it is also evaluating mitapivat for the treatment of alpha- and beta- thalassemia and sickle cell disease (SCD) in ongoing clinical trials. The Company is also developing AG-946, a clinical-stage oral activator of PKR enzymes for the treatment of hemolytic anemias and other indications, including SCD.