Description
Agios Pharmaceuticals is a biopharmaceutical company. The Company's 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 a- and b-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 and anemia associated with low-to intermediate-risk myelodysplastic syndrome (L-IR MDS), which is in Phase I clinical study.
