AGIO

Agios Pharmaceuticals, Inc. operates as a biopharmaceutical company focused on discovering and developing novel therapies for genetically defined diseases. Headquartered in Cambridge, Massachusetts, the company specializes in cellular metabolism and adjacent areas of biology, with particular emphasis on oncology and rare genetic diseases. Agios markets PYRUKYND (mitapivat), an oral medication approved for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency, and has expanded its indication to include thalassemia. The company previously developed TIBSOVO (ivosidenib) for IDH1-mutant cancers and IDHIFA (enasidenib) for IDH2-mutant acute myeloid leukemia, though it divested these oncology assets to Servier in 2020 for $1.8 billion upfront plus potential milestone payments. This strategic shift allowed Agios to concentrate resources on its rare disease portfolio. The company maintains a pipeline of genetically validated targets in metabolic disorders and continues advancing mitapivat in additional indications, including sickle cell disease. Agios employs approximately 300 people and collaborates with various academic institutions and healthcare organizations to advance its research programs. The company's approach centers on targeting fundamental biological pathways disrupted by genetic mutations, utilizing its expertise in cellular metabolism to develop first-in-class and best-in-class therapies for patients with significant unmet medical needs.