Agios Pharmaceuticals (NASDAQ:AGIO) is a biopharmaceutical company headquartered in Cambridge, Massachusetts, that specializes in the discovery and development of therapies targeting cellular metabolism in both oncology and rare genetic diseases. The company’s scientific approach centers on modulating metabolic processes to restore normal cellular function or to inhibit disease-specific metabolic pathways. Since its founding in 2008, Agios has built a research platform that integrates molecular biology, genetics, and chemistry to advance precision medicines from early research through regulatory approval.
Agios’ commercial portfolio includes Tibsovo® (ivosidenib), an oral inhibitor of mutant IDH1 used to treat certain forms of acute myeloid leukemia (AML) and cholangiocarcinoma, and Pyrukynd® (mitapivat), a first-in-class pyruvate kinase activator approved for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. These products reflect the company’s commitment to addressing areas of significant unmet medical need through tailored therapies that leverage its expertise in enzyme regulation.
Beyond its marketed therapies, Agios maintains a robust pipeline of investigational candidates targeting metabolic drivers of cancer and rare diseases. Research collaborations with global pharmaceutical partners support late-stage development in additional hematologic malignancies and solid tumors, as well as expansion into neurometabolic disorders. The company’s research operations and strategic partnerships span North America, Europe, and Asia, reflecting its goal of making novel metabolic medicines accessible to patients worldwide.
Co-founded by leaders in metabolic biology and biochemistry, Agios completed its initial public offering in 2013 and has since evolved into a wholly owned affiliate of Servier following a strategic transaction in early 2023. Under the leadership of President and Chief Executive Officer Brian Goff, Agios continues to invest in scientific innovation, manufacturing capabilities, and global regulatory activities, positioning itself at the forefront of metabolic therapy development.
