Agios Reports Third Quarter 2026 Financial Results and Provides Business Update
AQVESME growth accompanied a narrower quarterly loss, while the FDA extended the sickle cell disease application review by three months.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Rhea-AI Summary
Agios Pharmaceuticals (Nasdaq: AGIO) reported third-quarter 2026 worldwide mitapivat net revenue of $55.4 million, more than four times the prior-year quarter. Net loss narrowed to $65.0 million from $103.4 million. U.S. revenue rose 20% sequentially, while ex-U.S. revenue rose 74%. Cumulative AQVESME prescriptions with completed start forms reached 619, with another 48 in process.
The FDA extended its sickle cell disease application review deadline to February 1, 2027. New trial data showed sustained hemoglobin improvements for up to 115 weeks. Agios plans to reduce research and development staffing by over 100 roles by year-end, with savings expected in 2027. Cash and marketable securities totaled $907.0 million, down from $1,164.438 million at December 31, 2025. The company targets non-GAAP profitability in 2028, a measure outside generally accepted accounting principles.
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Hollow bars mark forward-looking points. How the balance works
Positive
- Moderate pointWorldwide mitapivat revenue reached $55.4 million in Q3 2026, versus $12.9 million a year earlier.
- Moderate pointQ3 net loss narrowed to $65.0 million from $103.4 million; loss per share improved to $1.09 from $1.78.
- Moderate pointQ3 research and development expense fell to $73.5 million from $86.8 million a year earlier.
- Moderate point. Forward-looking: it has not happened yet and may not happen.Non-GAAP profitability is Agios’ target for 2028.
- Minor pointU.S. revenue reached $48.7 million, versus $12.3 million a year earlier; sequential growth was 20%.
13 minor points
- Minor pointEx-U.S. revenue reached $6.6 million, versus $0.6 million a year earlier; sequential growth was 74%.
- Minor pointAQVESME prescriptions with completed start forms reached 619 cumulatively, including 177 in Q3; another 48 were in process.
- Minor pointApproximately 75% of patients remain on AQVESME after six months of treatment.
- Minor pointQ3 operating loss narrowed to $77.160 million from $116.869 million a year earlier.
- Minor pointNine-month revenue increased to $120.876 million from $34.061 million a year earlier.
- Minor pointNine-month net loss narrowed to $264.859 million from $304.742 million; loss per share improved to $4.46 from $5.27.
- Minor pointNine-month operating loss narrowed to $297.737 million from $350.554 million a year earlier.
- Minor pointOncology-business sale royalty income totaled $2.969 million in Q3 2026, versus none a year earlier.
- Minor pointRISE UP Phase 3 data showed durable hemoglobin improvements for up to 115 weeks with continued mitapivat treatment.
- Minor pointPost-hoc RISE UP analyses showed potential improvements in kidney-function and cardiac-remodeling measures.
- Minor pointAG-236 Phase 1 healthy-volunteer data support potential every-six-month dosing in polycythemia vera.
- Minor point. Forward-looking: it has not happened yet and may not happen.Staff-reduction cost savings are expected in 2027.
- Minor point. Forward-looking: it has not happened yet and may not happen.Available funds and anticipated revenue and interest income are expected to support launches and pipeline progress.
Negative
- Moderate pointFDA review deadline moved three months from November 1, 2026, to February 1, 2027.
- Moderate pointCash, cash equivalents and marketable securities fell to $907.018 million from $1,164.438 million at December 31, 2025.
- Moderate pointQ3 selling, general and administrative expense rose to $56.5 million from $41.3 million a year earlier.
- Moderate point. Forward-looking: it has not happened yet and may not happen.Research and development staffing will decline by over 100 roles by year-end, more than 35% versus 2025.
- Minor pointPotential risk-management program remains under FDA discussion during the sickle cell disease application review.
5 minor points
- Minor pointQ3 cost of sales increased to $2.525 million from $1.679 million a year earlier.
- Minor pointNine-month research and development expense increased to $255.389 million from $251.479 million a year earlier.
- Minor pointNine-month selling, general and administrative expense increased to $156.384 million from $128.670 million a year earlier.
- Minor pointNine-month cost of sales increased to $6.840 million from $4.466 million a year earlier.
- Minor pointNet interest income fell to $8.880 million in Q3 and $29.405 million over nine months, from $13.369 million and $43.969 million.
News Explained
FDA review and labeling discussions still include whether a REMS is needed, alongside expected 2027 savings from R&D cuts.
Agios says it will have reduced R&D headcount by over 100 roles by
The FDA requested additional information to assess whether a Risk Evaluation and Mitigation Strategy (REMS) is necessary, classified Agios’ response as a major amendment, and the sNDA review remains ongoing with labeling discussions that include a potential REMS.
Key Figures
- Worldwide mitapivat revenue
- $55.4 million
- Third quarter 2026
- Net loss
- $65.0 million
- Third quarter 2026; compared with $103.4 million in third quarter 2025
- Cash, cash equivalents and marketable securities
- $907.0 million
- As of September 30, 2026
- PDUFA goal date
- February 1, 2027
- Mitapivat sickle cell disease sNDA
- Cumulative AQVESME prescriptions
- 619 prescriptions
- With completed start forms as of September 30, 2026
- Patients remaining on treatment
- Approximately 75%
- After six months of AQVESME treatment
- Duration of hemoglobin improvements
- Up to 115 weeks
- RISE UP Phase 3 trial; continued mitapivat treatment
- Non-GAAP profitability target
- 2028
- Company target
Previous Earnings Reports
-
Mitapivat revenue rose to $44.7 million, while net loss narrowed year over year.
-
Worldwide mitapivat revenue reached $20.7 million, with AQVESME launch prescriptions reported.
-
PYRUKYND net product revenue reached $12.9 million, up 44% year over year.
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Key Terms
pdufa regulatory
rems regulatory
non-gaap financial
AI-generated analysis. How Rhea-AI works. Not financial advice.
- Mitapivat (PYRUKYND® and AQVESME®) delivered worldwide net revenues of
$55.4 million in third quarter of 2026, more than four times the$12.9 million reported in third quarter of 2025 - Strong U.S. demand for AQVESME in thalassemia continues, with 619 cumulative prescriptions with completed start forms from REMS-certified physicians, plus 48 additional prescriptions with start forms in process as of September 30, 2026
- New PDUFA goal date for mitapivat sickle cell disease sNDA set for February 1, 2027; company is launch-ready and in labeling discussions with FDA
- 14 abstracts accepted at ASH 2026, including new clinical data for mitapivat in sickle cell disease and for AG-236 in polycythemia vera, highlighting the breadth and depth of Agios’ rare hematology portfolio
$907.0 million in cash, cash equivalents, and marketable securities as of September 30, 2026, fully funding commercial launches and pipeline programs; company is targeting non-GAAP profitability in 2028
CAMBRIDGE, Mass., Nov. 05, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced financial results and updates for the third quarter ended September 30, 2026.
“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 –
$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.
- U.S. net revenue increased
- AQVESME 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 –
- Over 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.
- Over 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
Research and Development (R&D) Highlights
- Mitapivat (pyruvate kinase [PK] activator) –
- Sickle 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.
- Sickle Cell Disease –
- American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2026) –
- Agios announced that 14 abstracts led by Agios and external collaborators have been accepted for presentation and publication at ASH 2026, taking place December 12-15, 2026, in New Orleans, Louisiana. These include three oral presentations featuring new data across mitapivat clinical programs.
- New 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.
- The 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
- 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.
About Agios: Fueled by Connections to Transform Rare Diseases™
At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X.
Available Information about Agios
To achieve broad dissemination, Agios may disclose information to the public through a variety of disclosure channels including press releases, SEC filings, and public conference calls and webcasts. Some of the information distributed through these disclosure channels may be considered material information. Investors and others should note that Agios plans to use its website (www.agios.com) as a distribution channel to announce and give notice of Agios’ upcoming events and presentations (including, but not limited to, presentations at medical or healthcare conferences). Such information, which may be deemed material, will be available on the Investors section of the company’s website under the “Events & Presentations” tab. In addition, you may sign up to automatically receive email alerts about Agios’ upcoming events and presentations (“Calendar Alerts”) by visiting the “Email Alerts” option under the “IR Resources” tab of the Investors section of the company’s website and submitting your email address.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat), AQVESME® (mitapivat), and AG-236; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including mitapivat and AG-236; Agios’ expectations for the review of marketing applications for mitapivat by regulatory agencies, including the FDA; Agios’ strategic vision and goals; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236 and cevidoplenib, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
| Consolidated Balance Sheet Data | |||||||
| (in thousands) | |||||||
| (Unaudited) | |||||||
| September 30, 2026 | December 31, 2025 | ||||||
| Cash, cash equivalents, and marketable securities | $ | 907,018 | $ | 1,164,438 | |||
| Accounts receivable, net | 25,254 | 10,577 | |||||
| Inventory | 37,841 | 32,920 | |||||
| Total assets | 1,064,040 | 1,297,225 | |||||
| Stockholders' equity | 979,336 | 1,193,114 | |||||
| Consolidated Statements of Operations Data (in thousands, except share and per share data) (Unaudited) | |||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||
| Revenues: | |||||||||||||||
| Product revenue, net | $ | 55,385 | $ | 12,880 | $ | 120,876 | $ | 34,061 | |||||||
| Total revenue | 55,385 | 12,880 | 120,876 | 34,061 | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | $ | 2,525 | $ | 1,679 | $ | 6,840 | $ | 4,466 | |||||||
| Research and development | 73,487 | 86,796 | 255,389 | 251,479 | |||||||||||
| Selling, general and administrative | 56,533 | 41,274 | 156,384 | 128,670 | |||||||||||
| Total operating expenses | 132,545 | 129,749 | 418,613 | 384,615 | |||||||||||
| Loss from operations | (77,160 | ) | (116,869 | ) | (297,737 | ) | (350,554 | ) | |||||||
| Royalty income from gain on sale of oncology business | 2,969 | — | 2,969 | — | |||||||||||
| Interest income, net | 8,880 | 13,369 | 29,405 | 43,969 | |||||||||||
| Other income, net | 266 | 67 | 504 | 1,843 | |||||||||||
| Net loss | $ | (65,045 | ) | $ | (103,433 | ) | $ | (264,859 | ) | $ | (304,742 | ) | |||
| Net loss per share - basic and diluted | $ | (1.09 | ) | $ | (1.78 | ) | $ | (4.46 | ) | $ | (5.27 | ) | |||
| Weighted-average number of common shares used in computing net loss per share – basic and diluted | 59,707,904 | 58,139,277 | 59,329,729 | 57,846,173 | |||||||||||
Contacts:
Investor Contact
Morgan Sanford, Vice President, Investor Relations
Agios Pharmaceuticals
morgan.sanford@agios.com
Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
eamonn.nolan@agios.com
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What were Agios’ third-quarter 2026 revenue and net loss?
Agios reported $55.4 million in worldwide mitapivat net revenue and a $65.0 million net loss for the quarter ended September 30, 2026. Revenue was $12.9 million and net loss was $103.4 million in the prior-year quarter.
When is the FDA decision deadline for Agios’ mitapivat sickle cell disease application?
The FDA review goal date is February 1, 2027, extended from November 1, 2026. The application remains under review, and Agios is continuing labeling discussions with the agency.
Why did the FDA extend its review of Agios’ mitapivat sickle cell disease application?
The FDA classified Agios’ response to an additional-information request as a major amendment, triggering a three-month extension. The request concerned whether a Risk Evaluation and Mitigation Strategy, a drug-safety management program, is necessary for mitapivat in sickle cell disease.