Agios to Get $1.1 Billion Post FDA Approval of Vorasidenib

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) has announced a significant milestone in its ongoing efforts to develop pioneering therapies for rare diseases. The company will receive a combined total of $1.1 billion in milestone payments following the recent approval of vorasidenib by the U.S. Food and Drug Administration (FDA). Vorasidenib is now approved for use in adults and pediatric patients aged 12 and older who have Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation post-surgery, including biopsy, sub-total resection, or gross total resection.

These payments consist of a $905 million inflow from Royalty Pharma, as per a royalty purchase agreement made public in May 2024, and a $200 million payment from Servier, linked to Agios’ divestiture of its oncology business in 2021.

Cecilia Jones, Chief Financial Officer at Agios, emphasized that the FDA’s approval marks the first for a Grade 2 glioma in over two decades. She credited both the executional excellence of the Servier team and Agios’ innovative approach to discovering therapeutics for high unmet medical needs. Jones noted that the $1.1 billion in milestone payments will significantly bolster Agios' financial position, enhancing the company’s ability to independently prepare for the anticipated launches of PYRUKYND® (mitapivat) for thalassemia in 2025 and sickle cell disease in 2026. The financial boost will also support the continued advancement of Agios' pipeline projects, aiming to build a multi-billion-dollar franchise.

The sale of Agios' oncology business to Servier was finalized on March 31, 2021. The agreement provided Agios with $1.8 billion in upfront cash along with a $200 million milestone payment contingent upon FDA approval of vorasidenib. Additionally, Agios had secured 15% royalties on potential U.S. net sales of vorasidenib from its first commercial sale until the loss of market exclusivity. In 2022, Agios had also obtained a one-time payment of $131.8 million by selling its rights to 5% royalties on U.S. net sales of Servier’s TIBSOVO® (ivosidenib tablets).

On May 28, 2024, Agios disclosed an agreement to sell its rights to 15% royalties on U.S. net sales of vorasidenib to Royalty Pharma. The deal entitles Agios to an upfront payment of $905 million immediately upon FDA approval. Royalty Pharma will receive the full 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion and a 12% royalty on annual U.S. net sales exceeding $1 billion. Agios will retain a 3% royalty on sales above the $1 billion mark.

Agios is at the forefront of PK activation and is committed to developing therapies that can transform the lives of patients suffering from rare diseases. In the United States, Agios markets a unique pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying treatment for this chronic and debilitating form of hemolytic anemia. Leveraging its deep scientific expertise in classical hematology and cellular metabolism, Agios is advancing a comprehensive clinical pipeline that includes investigational medicines targeting alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia, and phenylketonuria (PKU). Beyond its clinical pipeline, the company is also developing a preclinical TMPRSS6 siRNA aimed at treating polycythemia vera.