Clinical Trial SuccessPhase 2 data from the RISE UP study in sickle cell disease have shown highly encouraging results, with superior hemoglobin increase and reduction in vaso-occlusive crises compared to competitors.
New IndicationsMitapivat, the lead asset of Agios, is expected to have three approved indications by the end of 2026, expanding its potential market significantly.
Regulatory ProgressAgios has completed thalassemia regulatory filings for mitapivat in the EU, Saudi Arabia, and UAE.