Agios to Receive $1.1 Billion in Milestone Payments Following FDA Approval of Vorasidenib

Core Insights - Agios Pharmaceuticals is set to receive a total of $1.1 billion in milestone payments following the FDA approval of vorasidenib for Grade 2 astrocytoma or oligodendroglioma, which includes a $905 million payment from Royalty Pharma and a $200 million payment from Servier [1][2][3] Financial Position - The anticipated payments will increase Agios' pro-forma cash position to $1.7 billion as of June 30, 2024, providing strong financial resources for future initiatives [1][2] - The company expects to leverage this cash position to prepare for potential launches of PYRUKYND® (mitapivat) in thalassemia in 2025 and sickle cell disease in 2026 [1][2] Business Transactions - Agios completed the sale of its oncology business to Servier on March 31, 2021, for $1.8 billion in upfront cash, with additional milestone payments tied to the FDA approval of vorasidenib [2][3] - Under the agreement with Royalty Pharma, Agios will receive an upfront payment of $905 million and retain a 3% royalty on U.S. net sales of vorasidenib exceeding $1 billion [3] Company Overview - Agios is a leader in pyruvate kinase (PK) activation, focusing on developing therapies for rare diseases, including a first-in-class PK activator for adults with PK deficiency [4] - The company is advancing a robust clinical pipeline targeting various conditions, including alpha- and beta-thalassemia, sickle cell disease, and MDS-associated anemia [4]