Agios: FDA extends mitapivat review to Feb. 1, 2027
Mitapivat generated $48.7 million in U.S. net revenue and $6.6 million in ex-U.S. net revenue as its sickle cell review moved to a February 1, 2027 goal date.
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.
Form Type
8-K
Rhea-AI Filing Summary
Agios Pharmaceuticals, Inc. reported third-quarter 2026 net product revenue of $55.385 million, versus $12.880 million a year earlier, and a net loss of $65.045 million, versus $103.433 million. U.S. net revenue was $48.7 million, up 20% sequentially, driven by the AQVESME thalassemia launch; ex-U.S. net revenue was $6.6 million, up 74% sequentially, driven primarily by early access programs after European Union approval of PYRUKYND for thalassemia. Cash, cash equivalents and marketable securities totaled $907.018 million as of September 30, 2026.
The FDA extended the PDUFA goal date for mitapivat's sickle cell disease sNDA by three months, from November 1, 2026, to February 1, 2027, after classifying Agios' response to a request about whether a REMS is necessary as a major amendment; labeling discussions continue. Agios expects to have reduced R&D headcount by over 100 roles by year-end 2026, more than 35% compared with 2025, with associated savings expected in 2027. It targets non-GAAP profitability in 2028 and reported 14 abstracts accepted for ASH 2026.
2 points · 0 major0 major · 1 point
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.
Positive
- Moderate pointQuarterly net product revenue was $55.385 million, versus $12.880 million a year earlier.
- Moderate pointQuarterly net loss was $65.045 million, versus $103.433 million a year earlier.
Negative
- Moderate pointThe FDA extended mitapivat's sickle cell sNDA review by three months, moving its PDUFA goal date to February 1, 2027.
Filing Explained
The release reports
8-K Event Classification
2 items: 2.02, 9.01
2 items
Item 2.02
Results of Operations and Financial Condition
Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Key Figures
Net product revenue: $55.385 million
Net loss: $65.045 million
U.S. net revenue: $48.7 million
+4 more
7 metrics
Net product revenue
$55.385 million
Three months ended September 30, 2026; $12.880 million in 2025
Net loss
$65.045 million
Three months ended September 30, 2026; $103.433 million in 2025
U.S. net revenue
$48.7 million
Third quarter 2026; $12.3 million in third quarter 2025
Ex-U.S. net revenue
$6.6 million
Third quarter 2026; $0.6 million in third quarter 2025
Cash, cash equivalents and marketable securities
$907.018 million
As of September 30, 2026; $1,164.438 million as of December 31, 2025
Research and development expenses
$73.487 million
Three months ended September 30, 2026; $86.796 million in 2025
Selling, general and administrative expenses
$56.533 million
Three months ended September 30, 2026; $41.274 million in 2025
Key Terms
PDUFA goal date, supplemental New Drug Application (sNDA), Risk Evaluation and Mitigation Strategy (REMS), non-GAAP profitability
4 terms
PDUFA goal date regulatory
"new PDUFA goal date for mitapivat sickle cell disease"
The PDUFA goal date is the target deadline set by the U.S. Food and Drug Administration for completing its review of a new drug or biologic application. Investors watch it like a court date for a product: the outcome (approval, rejection, or request for more information) can sharply change a company’s revenue prospects and stock price, and the date gives a predictable event around which markets and planning can focus.
supplemental New Drug Application (sNDA) regulatory
"mitapivat sickle cell disease supplemental New Drug Application (sNDA)"
A supplemental new drug application (snda) is a formal request made to regulatory authorities to make changes to an already approved medication, such as adding new uses, adjusting dosages, or improving manufacturing processes. It’s similar to updating a product’s packaging or instructions after it has been approved for sale. For investors, an snda signals ongoing development or improvements that could impact a company’s future sales or regulatory approval prospects.
Risk Evaluation and Mitigation Strategy (REMS) regulatory
"whether a REMS is necessary for mitapivat in sickle cell disease"
A Risk Evaluation and Mitigation Strategy (REMS) is a formal safety program required by regulators for certain medicines or medical products to ensure benefits outweigh serious risks. It sets rules—such as training for prescribers, special distribution systems, or monitoring—to reduce harm. For investors, REMS can limit how widely and quickly a product can be sold, raise compliance costs, and affect legal risk, similar to how safety checks can slow and add expense to running a vehicle fleet.
non-GAAP profitability financial
"goal is to achieve non-GAAP profitability in 2028"
Earnings Snapshot
Net product revenue: $55.385 million · Guidance included
Three months ended September 30, 2026
Net product revenue
$55.385 million
from $12.880 million in the three months ended September 30, 2025
Net loss
$65.045 million
from $103.433 million in the three months ended September 30, 2025
Research and development expenses
$73.487 million
from $86.796 million in the three months ended September 30, 2025
Selling, general and administrative expenses
$56.533 million
from $41.274 million in the three months ended September 30, 2025
Guidance
Agios' goal is to achieve non-GAAP profitability in 2028.
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
How much revenue did AGIO report in the third quarter of 2026?
Agios reported $55.385 million in net product revenue for the third quarter of 2026, compared with $12.880 million in the third quarter of 2025. U.S. net revenue was $48.7 million and ex-U.S. net revenue was $6.6 million.
What is AGIO's new PDUFA goal date for mitapivat in sickle cell disease?
The FDA set February 1, 2027, as the PDUFA goal date after extending review by three months from November 1, 2026. The extension followed a request for information about whether a REMS is necessary and the FDA's classification of Agios' response as a major amendment.
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): November 5, 2026
(Exact Name of Registrant as Specified in Charter)
| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (IRS Employer Identification No.) | ||||||||||||
| (Address of Principal Executive Offices) | (Zip Code) | |||||||||||||
Registrant’s telephone number, including area code: (617 ) 649-8600
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
| Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) | ||||||||
| Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) | ||||||||
| Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) | ||||||||
| Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) | ||||||||
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading symbol(s) | Name of each exchange on which registered | ||||||||||||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter). | |||||
| Emerging growth company | |||||
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐ | |||||
| Item 2.02 | Results of Operations and Financial Condition. | ||||
On November 5, 2026, Agios Pharmaceuticals, Inc. issued a press release announcing its results for the quarter ended September 30, 2026 and other business highlights. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in this Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.
| Item 9.01 | Financial Statements and Exhibits. | ||||
(d) Exhibits.
| Exhibit No. | Description | ||||
| 99.1 | Press release issued November 5, 2026 | ||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | ||||
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AGIOS PHARMACEUTICALS, INC. | ||||||||
Date: November 5, 2026 | By: | /s/ Brian Goff | ||||||
Brian Goff Chief Executive Officer | ||||||||
Exhibit 99.1
Agios Reports Third Quarter 2026 Financial Results and Provides Business Update
•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. 5, 2026 – 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 –
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) 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 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.
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