Agios Announces $905-Million Purchase Agreement for Vorasidenib Royalty

Agios Pharmaceuticals, Inc. recently announced it has agreed to sell its rights to its 15% royalty on potential US net sales of Servier’s vorasidenib to Royalty Pharma. Under the terms of the agreement, Agios will receive an upfront payment of $905 million upon approval of vorasidenib by the US FDA, and Royalty Pharma will receive the entirety of the 15% royalty on annual US net sales of vorasidenib up to $1 billion, and a 12% royalty on annual US net sales greater than $1 billion. Agios will retain a 3% royalty on annual US net sales greater than $1 billion.

Vorasidenib is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes for the treatment of IDH-mutant diffuse glioma. In 2021, Agios completed the sale of its oncology portfolio – including vorasidenib – to Servier. As part of that divestiture, Agios is owed a milestone payment of $200 million upon vorasidenib’s approval by the FDA, as well as a 15% royalty on U.S. net sales of vorasidenib. Agios continues to retain the right to the approval milestone from Servier. Servier announced the FDA has designated a Prescription Drug User Fee Act (PDUFA) action date of August 20, 2024.

“It’s an exciting time at Agios with multiple near-term catalysts that we believe have the potential to make a meaningful difference in patients’ lives and create significant shareholder value. With this transaction, we have added significant financial flexibility while retaining long-term value and have identified a partner in Royalty Pharma that shares our excitement about the potential of vorasidenib,” said Brian Goff, Chief Executive Officer at Agios. “This transaction will provide us with the financial independence to prepare for potential PYRUKYND (mitapivat) launches in thalassemia and sickle cell disease as we build a PK activation franchise with multi-billion-dollar potential, and to opportunistically expand our pipeline through both internally and externally discovered assets.”

Goldman Sachs & Co. LLC acted as exclusive financial advisor to Agios; WilmerHale served as legal advisor to Agios.

Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the US, Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, visit  www.agios.com.