Agios’ mission is to create differentiated, small molecule medicines for people living with genetically defined diseases. Our scientific focus is on discovering and developing innovative therapies based on our core expertise in cellular metabolism. We’re a company that cares about our work, each other, and the people who are counting on us the most. We’re inspired to think big and welcome the different perspectives and backgrounds needed to deliver extraordinary results.
Our Focus Areas
We are driven by a commitment to people living with genetically defined diseases and are accelerating and expanding our portfolio of investigational medicines for these conditions. Genetically defined diseases range from a broad group of more than 600 rare diseases caused by mutations of single genes to conditions resulting from alterations in multiple genes (polygenic diseases) that affect up to millions of patients worldwide. In these disorders, the genetic defect(s) lead to a deficient expression or function in one or several gene products, which manifest in organ dysfunction and frequently result in inexorable deterioration until death or to significant irreversible lifelong disability and or suffering.
Our therapeutic focus is defined by a combination of our most differentiated foundational elements
The lead product candidate in our clinical portfolio, mitapivat, is an activator of both wild-type and mutant pyruvate kinase-R, or PKR, for the potential treatment of hemolytic anemias. We are currently evaluating mitapivat for the treatment of pyruvate kinase, or PK, deficiency, thalassemia and sickle cell disease. We are also developing AG-946, a next-generation PKR activator, for the potential treatment of hemolytic anemias and other indications.
We continue to foster a productive research engine yielding new insights and potential therapeutic approaches – across both PK activation and non-PK activation opportunities. Our growing late-stage research pipeline is a rich and sustainable portfolio of genetically defined disease targets with clear disease area applications, including hereditary and acquired anemias, myopathies, retinal diseases and diseases of inborn errors of metabolism such as aminoacidurias, aminoacidemias and others.
Since our founding in 2008, we have pioneered a novel path to treating serious and challenging diseases by targeting cellular metabolism. In our first decade, we brought two precision oncology medications from our own labs to patients with acute myeloid leukemia.
During that same time period, our team discovered and developed six additional investigational new drug candidates, including mitapivat, the first PKR activator in development as a potential treatment for a rare hemolytic anemia known as PK deficiency. We are leading the way in advancing PKR activation for additional hemolytic anemias, including thalassemia and sickle cell disease.
In late 2020, we announced that we will move forward with a singular focus on accelerating and expanding our genetically defined disease portfolio, including the mitapivat clinical programs and a robust pipeline of therapeutic candidates. To enable this renewed focus, we sold our commercial, clinical and research-stage oncology portfolio to Servier, an independent global pharmaceutical company that is committed to the cancer community and to investing in our oncology assets and our employees who support these programs.
This transformation allows our oncology portfolio to grow and thrive with Servier and provides Agios with the resources required to optimize the development of our promising genetically defined disease therapies, ultimately enabling the greatest overall positive impact for patients.