Agios Pharmaceuticals reported a sharp increase in third-quarter product revenue, driven by the U.S. launch of AQVESME for thalassemia and growing sales of mitapivat outside the United States. The company also outlined a planned reduction in research and development staffing, updated the expected FDA decision timing for mitapivat in sickle cell disease, and said its cash position supports its commercial and research plans.
Commercial performance
- Mitapivat, sold as PYRUKYND and AQVESME, generated $55.4 million in worldwide net revenue in the quarter, compared with $12.9 million a year earlier. Agios said quarterly product revenue exceeded its total for 2025.
- U.S. net revenue was $48.7 million, up 20% from the prior quarter. The increase was driven by AQVESME, which launched in the United States in January for thalassemia.
- Revenue outside the United States was $6.6 million, up 74% sequentially. Agios attributed the increase primarily to early access programs in Europe, following the European Union’s approval of PYRUKYND for thalassemia in May.
- The company is advancing international access through partnerships with Avanzanite Bioscience in Europe and NewBridge Pharmaceuticals in the Gulf Cooperation Council region.
AQVESME uptake
- U.S. physicians wrote 177 AQVESME prescriptions with completed Agios start forms during the quarter, bringing the cumulative total to 619 as of September 30. Another 48 prescriptions had start forms in process and were not included in that count.
- Agios said demand is expanding among both transfusion-dependent and non-transfusion-dependent patients. It reported that about 75% of patients remain on treatment after six months.
- The prescription figures track completed or in-process start forms; they are not a count of patients currently receiving treatment.
Sickle cell disease program
- The FDA’s new target date for a decision on Agios’ supplemental application for mitapivat in sickle cell disease is February 1, 2027.
- Agios said it is in labeling discussions with the FDA and is preparing for a potential launch. The company described sickle cell disease as its largest indication opportunity for mitapivat.
- Fourteen Agios research abstracts were accepted for the 2026 American Society of Hematology meeting. They include new clinical data on mitapivat in sickle cell disease and on AG-236 in polycythemia vera.
Costs and financial position
- Agios expects to reduce its R&D workforce by more than 100 roles by year-end, a reduction of over 35% compared with 2025. It expects the related cost savings to be realized in 2027.
- The company had $907.0 million in cash, cash equivalents and marketable securities at September 30. Agios said the balance is sufficient to fund its commercial launches and pipeline programs.
- Agios is targeting non-GAAP profitability in 2028.
The operating changes point to a smaller R&D workforce and expected cost savings beginning next year, while the company continues to invest in commercializing mitapivat and developing its pipeline. The February 2027 FDA target date is a key near-term milestone for the sickle cell disease program.