PYRUKYND Revenue Growth
Fourth quarter 2024 net PYRUKYND revenue was $10.7 million, an increase of 51% compared to $7.1 million in the fourth quarter of 2023.
Successful Pediatric Trials
Top line results from the ACTIVATE KIDS Phase III trial showed a 31.6% hemoglobin response in the mitapivat arm compared to 0% in the placebo arm.
Thalassemia and Sickle Cell Disease Pipeline Progress
Potential approval and launch of PYRUKYND in thalassemia expected in September 2025, with sickle cell disease in 2026. Phase III RISE up study for sickle cell disease completed enrollment.
Strong Financial Position
Agios ended the fourth quarter with cash, cash equivalents, and marketable securities of approximately $1.5 billion.
Regulatory and Clinical Milestones
The September 7 PDUFA goal date for PYRUKYND in thalassemia and the Phase III readout of RISE study in sickle cell disease by the end of 2025.