Strong Cash Position and Milestone Payments
Agios received $1.1 billion in milestone payments following FDA approval of vorasidenib, enhancing the company's cash base to approximately $1.7 billion.
Positive Phase III Data and Advancements
Agios reported positive data in Phase III programs for mitapivat in PKD and thalassemia, and completed enrollment in the Phase III RISE UP sickle cell disease study.
Orphan Drug Designation for Tebapivat
The FDA granted orphan drug designation to tebapivat for the treatment of myelodysplastic syndromes, supporting Agios' potential in this market.
Commercial Preparation for Thalassemia Launch
Agios is actively preparing for a potential U.S. launch of PYRUKYND in thalassemia in 2025, targeting approximately 65% of the adult thalassemia patient population.
Increased Net Revenue from PYRUKYND
Third quarter 2024 net PYRUKYND revenue was $9 million, marking a 22% increase compared to the third quarter of 2023.