AG-946

About Agios Pharmaceuticals

Agios Pharmaceuticals is a clinical-stage biopharma company with a singular focus on pioneering therapies for rare genetic diseases by targeting cellular metabolism. The company successfully pivoted from oncology, having discovered IDH mutations and launched two approved cancer drugs, to concentrate its resources on rare metabolic disorders following the $2 billion divestiture of its oncology portfolio to Servier. Its current strategy centers on advancing its lead asset, mitapivat, a pyruvate kinase activator, through late-stage trials for pyruvate kinase deficiency, thalassemia, and sickle cell disease, while cultivating a research engine for novel metabolic targets.

Therapeutic Areas