Agios Q3 Mitapivat Revenue Hits Record as FDA Extends Sickle Cell Review

Agios reported record third-quarter 2026 mitapivat net revenue of $55.4 million, more than four times the year-earlier level, as the U.S. thalassemia launch broadened demand for AQVESME. The company also said it remains launch-ready for sickle cell disease, although the FDA extended the application’s review period by three months and set a new Feb. 1, 2027, target date.

Worldwide mitapivat net revenue increased 4.3-fold from $12.9 million in the third quarter of 2025 and exceeded the product’s revenue for all of 2025. U.S. net revenue reached $48.7 million, up 20% from the second quarter, driven by AQVESME demand across transfusion-dependent and non-transfusion-dependent thalassemia patients. Ex-U.S. revenue was $6.6 million, up 74% sequentially, reflecting early-access programs in Europe following the product’s second-quarter thalassemia approval.

Agios recorded 177 prescriptions with completed start forms during the quarter and 619 cumulatively since the U.S. launch. Another 48 prescriptions had start forms in process at quarter-end. The company said about 75% of patients remained on AQVESME after six months of treatment, supporting the development of a durable patient base.

Longer-term open-label extension data presented in 2026 indicated that roughly one-third of previous non-responders achieved a hemoglobin response with extended treatment. Mean response duration increased to 43.6 weeks from 17.9 weeks.

Expenses rise in commercial functions as R&D spending falls

Cost of sales increased to $2.5 million from $1.7 million a year earlier. Research and development expense declined to $73.5 million from $86.8 million, while selling, general and administrative expense rose to $56.5 million from $41.3 million as Agios supported its commercial activities and launch preparations.

The third-quarter net loss narrowed to $65.0 million from $103.4 million in the prior-year period. Agios ended September with $907.0 million in cash, cash equivalents and marketable securities, compared with $1.2 billion at the end of 2025.

Agios is reducing its R&D workforce by more than 35%, representing over 100 positions, during 2026 as it concentrates resources on rare hematology programs. The company is targeting non-GAAP profitability in 2028, based on an assumed 2027 sickle cell disease launch, revenue growth and cost discipline. That target excludes potential business development and certain items, including share-based compensation, depreciation, amortization and impairments, acquisition-related items and restructuring charges.

Sickle cell review moves to February 2027

The FDA changed mitapivat’s Prescription Drug User Fee Act goal date from Nov. 1, 2026, to Feb. 1, 2027, after classifying Agios’ response to an agency information request as a major amendment. The FDA had requested information to assess whether a Risk Evaluation and Mitigation Strategy would be necessary, and Agios responded with approximately nine months of additional safety data from open-label extension periods.

Agios said the additional data did not change its assessment of mitapivat’s benefit-risk profile. The company reported no hepatocellular injury cases similar in pattern or severity to those observed in thalassemia clinical trials. Labeling discussions with the FDA are continuing.

The company has trained field, medical, market-access and patient-support teams and staged manufacturing and supply for potential day-one availability. Its initial launch target comprises about 25,000 actively engaged patients aged 16 or older who need therapy, within an estimated U.S. diagnosed population of roughly 75,000 in that age group.

Open-label extension data from the Phase 3 RISE UP study showed that 62.7% of patients achieved a hemoglobin improvement, defined as an increase of at least 1.0 gram per deciliter across two consecutive visits. The longest ongoing response was 115 weeks, while 39.7% of initial non-responders later improved. The presentation also described emerging kidney and cardiac findings, though Agios characterized these as supportive data rather than established clinical outcomes.

Focused pipeline and capital allocation

Agios plans to advance AG-236, an investigational TMPRSS6-targeting therapy, through an operationally seamless Phase 2/3 program in polycythemia vera. Phase 1 healthy-volunteer data showed changes in hepcidin, transferrin saturation and hematocrit that were sustained after dosing, with certain iron-related effects lasting through six months. The company said the Phase 2/3 study was planned for the second half of 2026.

AG-181, a phenylalanine hydroxylase stabilizer being studied for phenylketonuria, remains in Phase 1b development. Healthy-volunteer data supported twice-daily dosing and showed no serious or Grade 3 or higher adverse events and no discontinuations. Proof-of-mechanism data in patients were expected in the second half of 2026.

Agios also plans to begin a Phase 3 study of cevidoplenib in immune thrombocytopenia in the first half of 2028. The company said it may consider strategic options for non-core assets, including AG-181 and potential monetization of its vorasidenib royalty.

The company’s outlook remains subject to regulatory, clinical and commercial uncertainty. Key risks identified in the presentation include the timing and outcome of FDA and other regulatory decisions, clinical-trial results and enrollment, manufacturing and commercial execution, unexpected cash requirements, competition, intellectual-property protection and uncertainty over potential milestone and royalty payments.