Agios Pharmaceuticals reported a sharp increase in third-quarter product revenue, led by growing sales of mitapivat, marketed as AQVESME in the U.S. and PYRUKYND outside the country. The company also updated its plans for mitapivat in sickle cell disease, outlined a substantial reduction in research and development staffing, and said its cash position supports its launches and pipeline programs.
Key details
-
Mitapivat revenue more than quadrupled year over year. Worldwide net revenue for AQVESME and PYRUKYND was $55.4 million in the third quarter, compared with $12.9 million a year earlier. U.S. revenue was $48.7 million, up 20% from the prior quarter; ex-U.S. revenue was $6.6 million, up 74%. The regional figures do not sum exactly to the reported total because of rounding.
-
AQVESME demand is expanding in the U.S. The drug launched in January 2026 for thalassemia. In the third quarter, U.S. physicians wrote 177 prescriptions with completed Agios start forms, bringing the cumulative number to 619 by Sept. 30. Another 48 prescriptions had start forms in process and were not included in that total. Agios said demand is reaching both transfusion-dependent patients and the larger non-transfusion-dependent population, and approximately 75% of patients remain on treatment after six months.
-
European and other international sales are developing. Ex-U.S. revenue growth was driven mainly by early access programs in Europe, following the European Union’s approval of PYRUKYND for thalassemia in May. Agios is pursuing international commercialization through partnerships with Avanzanite Bioscience in Europe and NewBridge Pharmaceuticals in the Gulf Cooperation Council region.
-
The FDA’s target date for a sickle cell decision is Feb. 1, 2027. The date applies to Agios’ supplemental application for mitapivat in sickle cell disease. The company said it is in labeling discussions with the FDA and is preparing for a potential launch.
-
Agios plans a significant reduction in R&D staffing. It expects to eliminate more than 100 R&D roles by the end of 2026, reducing the group’s headcount by more than 35% compared with 2025. The company expects the related cost savings to be realized in 2027.
-
The company reported $907 million in cash, cash equivalents and marketable securities at Sept. 30. Agios said the funds are sufficient to support its commercial launches and pipeline programs, and it is targeting non-GAAP profitability in 2028.
-
New research is headed to a major hematology meeting. Fourteen Agios abstracts were accepted for the 2026 American Society of Hematology meeting, including new clinical data on mitapivat in sickle cell disease and on AG-236 in polycythemia vera.
Agios is building its commercial business around mitapivat while preparing for a potential expansion into sickle cell disease. At the same time, the planned R&D workforce reductions are intended to lower costs, with the savings expected to begin in 2027.