AGIOS PHARMACEUTICALS Business Summary

Agios Pharmaceuticals is a commercial-stage biopharmaceutical company specializing in cellular metabolism and rare hematologic diseases. The company generates revenue primarily through the global sale of its first-in-class pyruvate kinase (PK) activators, notably PYRUKYND (mitapivat) for PK deficiency and the newly launched AQVESME (mitapivat) for alpha- and beta-thalassemia, which received FDA approval in late 2025. Its business model is centered on a "PK activation" franchise, leveraging small-molecule therapeutics to address hemolytic anemias. As of February 2026, the company is expanding its commercial footprint into sickle cell disease (SCD) and lower-risk myelodysplastic syndromes (MDS), supported by a robust pipeline including tebapivat and AG-236. Agios competes directly with Vertex Pharmaceuticals, bluebird bio, and Bristol Myers Squibb in the hemoglobinopathy market; it distinguishes itself by offering oral, non-genetic small molecules as a more accessible alternative to complex gene therapies and chronic transfusion protocols. Leadership is directed by CEO Brian Goff, who joined in 2022 with extensive rare disease experience from Alexion and Baxter, alongside CFO Cecilia Jones and Dr. Sarah Gheuens, the Chief Medical Officer and Head of R&D. The company was founded by a distinguished scientific team including Dr. Lewis Cantley, Dr. Tak Mak, and Dr. Craig Thompson, pioneers in the field of cancer metabolism. Agios maintains a strong institutional backing from major investors such as Farallon Capital Management, Vanguard, and BlackRock, with notable board representation from industry veterans like former CEO Dr. Jackie Fouse and independent director Dr. David Scadden. As of early 2026, the company maintains a formidable liquidity position of approximately $1.2 billion, providing the capital necessary to scale its thalassemia launch and finalize regulatory submissions for its sickle cell disease indications.