Agios Pharmaceuticals (NASDAQ:AGIO) today released financial results and business updates for the fourth quarter and full year ended December 31, 2025, highlighting regulatory progress and commercial momentum in rare blood disorders.
Key clinical and regulatory developments include the FDA approval and U.S. availability of AQVESME (mitapivat) for thalassemia, expanding the company’s portfolio of first-in-class PK activator therapies.
PYRUKYND (mitapivat) generated fourth-quarter net revenue of $20 million ($16 million U.S., $4.0 million ex-U.S.) and full-year revenue of $54 million.
The company also recorded a fourth-quarter net loss of $108 million.
As of December 31, 2025, cash, cash equivalents, and marketable securities totaled $1.2 billion, providing a strong balance sheet to support ongoing clinical development and commercial execution.
Agios also plans a pre-sNDA meeting with the FDA for mitapivat in sickle cell disease in the first quarter of 2026 and expects topline data from the Phase 2 trial of tebapivat in H2 2026.