On November 5, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported financial results and updates for the third quarter ended September 30, 2026.

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"The third quarter was a record for our product revenue, outperforming our entire 2025 total in just three months. This growth was driven by the U.S. commercial launch of AQVESME in thalassemia, where broadening demand is translating into a durable base of patients on therapy," said Brian Goff, Chief Executive Officer, Agios. "This launch has built the commercial foundation for our largest indication opportunity with mitapivat – sickle cell disease. Our conviction in mitapivat for patients with sickle cell disease is grounded in the robust efficacy and safety data generated to date and we are launch-ready ahead of our PDUFA goal date of February 1, 2027. With this commercial infrastructure, alongside a strong balance sheet and a disciplined operating model, Agios is well positioned for sustained growth as a focused, rare hematology company."

Third Quarter 2026 and Recent Corporate Highlights
•Mitapivat (PYRUKYND and AQVESME) Commercial Performance and Update –
o$48.7 million in U.S. net revenue and $6.6 million in ex-U.S. net revenue in the third quarter of 2026
•U.S. net revenue increased 20% sequentially, driven by the U.S. commercial launch of AQVESME (mitapivat) in thalassemia in late January 2026.
•Ex-U.S. net revenue increased 74% sequentially, driven primarily by early access programs in Europe following the late May 2026 approval of PYRUKYND (mitapivat) in thalassemia in the European Union. This robust, early momentum underscores the strong global opportunity for mitapivat, which the company is advancing through capital-efficient partnerships with Avanzanite Bioscience B.V. in Europe and NewBridge Pharmaceuticals in the Gulf Cooperation Council (GCC) region.

oAQVESME demand continues to expand following the January 2026 U.S. commercial launch in thalassemia
•In the third quarter, 177 AQVESME prescriptions with completed Agios start forms (ASFs) were written by Risk Evaluation and Mitigation Strategy (REMS)-certified U.S. physicians, bringing cumulative prescriptions to 619 as of September 30, 2026.
•An additional 48 cumulative prescriptions had ASFs in process as of September 30, 2026, which are not included in the reported metric above.
•Demand continues to broaden, with deeper penetration in transfusion-dependent patients and expansion into the larger non-transfusion dependent population. Approximately 75% of patients remain on AQVESME after six months of treatment.

•Operating Model Update –
oOver the course of 2026, Agios reviewed its operating model, and will have reduced its R&D headcount by over 100 roles by year-end, representing a more than 35% decrease compared with 2025. The company expects the associated cost savings of these reductions to be realized in 2027.

Research and Development (R&D) Highlights
•Mitapivat (pyruvate kinase [PK] activator) –
oSickle Cell Disease –
•Agios announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) goal date for its mitapivat sickle cell disease supplemental New Drug Application (sNDA) by three months, from November 1, 2026, to February 1, 2027.
•The FDA recently requested additional information to evaluate whether a REMS is necessary for mitapivat in sickle cell disease. The FDA classified Agios’ response to this information request as a major amendment to the sNDA, resulting in the three-month review extension.
•Agios will continue its labeling discussions with the FDA as part of the ongoing sNDA review, including elements of a potential REMS.

•American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2026) –
oAgios announced that 14 abstracts led by Agios and external collaborators have been accepted for presentation and publication at ASH (Free ASH Whitepaper) 2026, taking place December 12-15, 2026, in New Orleans, Louisiana. These include three oral presentations featuring new data across mitapivat clinical programs.
oNew clinical data from the RISE UP Phase 3 trial of mitapivat in sickle cell disease shows durable hemoglobin improvements for up to 115 weeks (over two years) with continued treatment. Additional post-hoc RISE UP analyses show the potential for mitapivat to improve measures of kidney function and cardiac remodeling in this population, which may reflect its unique dual activation of two pyruvate kinase isoforms: PKR, expressed in red blood cells, and PKM2, expressed in organ tissues.
oThe first clinical data for AG-236 from a Phase 1 trial in healthy volunteers support every-six-month dosing in polycythemia vera, supporting a potential best-in-class profile.

Third Quarter 2026 Financial Results
For the quarter ended September 30, 2026, net loss was $65.0 million, compared to net loss of $103.4 million for the quarter ended September 30, 2025.
•Net product revenue from U.S. sales of mitapivat (PYRUKYND and AQVESME) for the third quarter of 2026 was $48.7 million, compared to $12.3 million for the third quarter of 2025.

•Net product revenue from ex-U.S. sales of mitapivat (PYRUKYND) for the third quarter of 2026 was $6.6 million, compared to $0.6 million for the third quarter of 2025.

•Cost of sales for the third quarter of 2026 was $2.5 million.

•Research and Development (R&D) expenses were $73.5 million for the third quarter of 2026, compared to $86.8 million for the third quarter of 2025, driven primarily by reductions in spend associated with mitapivat and tebapivat process development and certain clinical trials.

•Selling, General and Administrative (SG&A) expenses were $56.5 million for the third quarter of 2026, compared to $41.3 million for the third quarter of 2025, due to an increase in activities supporting the U.S. commercial launch of AQVESME in thalassemia and preparation for the potential U.S. commercial launch of mitapivat in sickle cell disease.

•Cash, cash equivalents and marketable securities were $907.0 million as of September 30, 2026, compared to $1.2 billion as of December 31, 2025. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to execute the U.S. commercial launch of AQVESME in thalassemia, prepare for the potential U.S. commercial launch of mitapivat in sickle cell disease, and progress the company’s pipeline. Agios’ goal is to achieve non-Generally Accepted Accounting Principles (GAAP) profitability in 2028.

Third Quarter 2026 Conference Call Information
Agios will host a conference call and live webcast today, November 5, 2026, at 8:00 a.m. ET to discuss the company’s third quarter 2026 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.

(Press release, Agios Pharmaceuticals, NOV 5, 2026, View Source [SID1234674295])