ipeline Highlights Mitapivat First-in-class PK activator with the potential to transform care across multiple rare hematologic diseases. Approved in the U.S. for PK deficiency (2022) and thalassemia (2025). In July 2026, the FDA accepted the supplemental New Drug Application for sickle cell disease with Priority Review. If approved, mitapivat has the potential to become the first oral PK activator for sickle cell disease, expanding treatment options for patients. Additionally, Agios dosed the first patient in the REIGNITE Phase 3 trial, the confirmatory clinical trial required to be conducted under the U.S. accelerated approval pathway. This global trial is designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with sickle cell disease aged 12 years or older. Agios also filed for regulatory approval of mitapivat for sickle cell disease in Saudi Arabia. Cevidoplenib Added to the Agios pipeline through an exclusive global licensing agreement with Oscotec in 2026. A highly selective, next-generation oral SYK inhibitor being developed for ITP. Planned Phase 3 development in 2028, following completion of additional CMC AG-236 An investigational siRNA targeting TMPRSS6 for polycythemia vera. Designed to address the underlying biology of disease characterized by excessive RBC production Phase 2 portion of the Phase 2/3 program is expected to begin in the second half of 2026 AG-181 An investigational phenylalanine hydroxylase (PAH) stabilizer for phenylketonuria (PKU). Designed to address the underlying metabolic defect responsible for PKU. The first patient has been dosed in the Phase 1b study, with data expected in the second half of 2026
ipeline Highlights
Mitapivat
First-in-class PK activator with the potential to transform care across multiple rare hematologic diseases.
Approved in the U.S. for PK deficiency (2022) and thalassemia (2025).
In July 2026, the FDA accepted the supplemental New Drug Application for sickle cell disease with Priority Review. If approved, mitapivat has the potential to become the first oral PK activator for sickle cell disease, expanding treatment options for patients.
Additionally, Agios dosed the first patient in the REIGNITE Phase 3 trial, the confirmatory clinical trial required to be conducted under the U.S. accelerated approval pathway. This global trial is designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with sickle cell disease aged 12 years or older.
Agios also filed for regulatory approval of mitapivat for sickle cell disease in Saudi Arabia.
Cevidoplenib
Added to the Agios pipeline through an exclusive global licensing agreement with Oscotec in 2026.
A highly selective, next-generation oral SYK inhibitor being developed for ITP.
Planned Phase 3 development in 2028, following completion of additional CMC
AG-236
An investigational siRNA targeting TMPRSS6 for polycythemia vera.
Designed to address the underlying biology of disease characterized by excessive RBC production
Phase 2 portion of the Phase 2/3 program is expected to begin in the second half of 2026
AG-181
An investigational phenylalanine hydroxylase (PAH) stabilizer for phenylketonuria (PKU).
Designed to address the underlying metabolic defect responsible for PKU.
The first patient has been dosed in the Phase 1b study, with data expected in the second half of 2026
Created using ChatSlide
Agios Pipeline focuses on hematology, leveraging PK activation to address rare diseases. Mitapivat, a key product, has gained U.S. approvals for PK deficiency and thalassemia, with a priority review for sickle cell expected in 2026. The company is also diversifying its portfolio with next-wave assets like Cevidoplenib, an oral SYK inhibitor for ITP, which is set for Phase 3 trials in 2028 following a global license acquisition.