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Mitapivat

Non-Transfusion-Dependent Thalassemia

Phase 3Active (ENERGIZE trial)

Key Facts

Indication
Non-Transfusion-Dependent Thalassemia
Phase
Phase 3
Status
Active (ENERGIZE trial)
Company

About Agios Pharmaceuticals

Founded in 2008, Agios Pharmaceuticals is a leader in the science of cellular metabolism, initially making its mark with the discovery of IDH inhibitors leading to two approved cancer drugs. The company has since strategically pivoted to focus exclusively on rare diseases, particularly hemolytic anemias, leveraging its pioneering PK activation platform. With one approved rare disease therapy and a robust late-stage pipeline, Agios is positioned to address significant unmet needs in thalassemia and sickle cell disease, supported by a strong corporate culture centered on patient connections.

View full company profile

About Agios Pharmaceuticals

Founded in 2008, Agios Pharmaceuticals is a leader in the science of cellular metabolism, initially making its mark with the discovery of IDH inhibitors leading to two approved cancer drugs. The company has since strategically pivoted to focus exclusively on rare diseases, particularly hemolytic anemias, leveraging its pioneering PK activation platform. With one approved rare disease therapy and a robust late-stage pipeline, Agios is positioned to address significant unmet needs in thalassemia and sickle cell disease, supported by a strong corporate culture centered on patient connections.

View full company profile

About Agios Pharmaceuticals

Founded in 2008, Agios Pharmaceuticals is a leader in the science of cellular metabolism, initially making its mark with the discovery of IDH inhibitors leading to two approved cancer drugs. The company has since strategically pivoted to focus exclusively on rare diseases, particularly hemolytic anemias, leveraging its pioneering PK activation platform. With one approved rare disease therapy and a robust late-stage pipeline, Agios is positioned to address significant unmet needs in thalassemia and sickle cell disease, supported by a strong corporate culture centered on patient connections.

View full company profile