Agios (NASDAQ: AGIO) doubles mitapivat revenue and funds 2026 pipeline catalysts
Filing Impact
Filing Sentiment
Form Type
8-K
Rhea-AI Filing Summary
Agios Pharmaceuticals reported first quarter 2026 results showing rapid growth in its rare disease franchise but continued investment-driven losses. Mitapivat (PYRUKYND® and AQVESME™) generated worldwide net revenues of $20.7 million, up from $8.7 million in the first quarter of 2025, supported by a strong U.S. launch of AQVESME in thalassemia with 242 prescriptions written as of March 31, 2026.
Net loss for the quarter was $99.1 million compared to $89.3 million a year earlier, reflecting higher R&D spending of $81.1 million and SG&A expenses of $48.3 million. Cash, cash equivalents and marketable securities totaled $1.0 billion as of March 31, 2026. The company plans to submit a supplemental NDA for mitapivat in sickle cell disease in the second quarter of 2026 and expects Phase 2 tebapivat readouts in myelodysplastic syndromes and sickle cell disease later in 2026.
Positive
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Insights
Agios is rapidly scaling mitapivat revenue while investing heavily in its hematology pipeline.
Agios Pharmaceuticals reported Q1 2026 net product revenue of $20.746M, more than double Q1 2025, driven by the U.S. launch of AQVESME in thalassemia and ex-U.S. PYRUKYND sales. This shows early commercial traction in rare hematologic diseases.
The company posted a net loss of $99.111M, up from $89.289M, as R&D rose to $81.148M and SG&A to $48.304M, reflecting broader clinical activity and launch spending. Cash, cash equivalents and marketable securities of $1.045B as of March 31, 2026 provide a substantial runway.
Key upcoming catalysts include a planned mitapivat sNDA filing for sickle cell disease in Q2 2026 and Phase 2 tebapivat readouts in lower-risk myelodysplastic syndromes in the first half of 2026 and sickle cell disease in the second half of 2026. Outcomes from these milestones and future quarters’ revenue trends will further clarify the trajectory toward sustainable growth.
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, and exhibit attachments filed with this report.
Key Figures
Worldwide mitapivat net revenue: $20.7 million
Total product revenue: $20.746 million
Net loss: $99.111 million
+5 more
8 metrics
Worldwide mitapivat net revenue
$20.7 million
First quarter 2026, vs. $8.7 million in Q1 2025
Total product revenue
$20.746 million
Three months ended March 31, 2026
Net loss
$99.111 million
Three months ended March 31, 2026, vs. $89.289 million in 2025
R&D expenses
$81.148 million
First quarter 2026, vs. $72.743 million in Q1 2025
SG&A expenses
$48.304 million
First quarter 2026, vs. $41.527 million in Q1 2025
Cash and investments
$1.045 billion
Cash, cash equivalents and marketable securities as of March 31, 2026
AQVESME prescriptions
242 prescriptions
Written by REMS-certified U.S. physicians as of March 31, 2026
Net loss per share
$1.69
Basic and diluted, three months ended March 31, 2026
Key Terms
supplemental New Drug Application (sNDA), accelerated approval pathway, Risk Evaluation and Mitigation Strategy (REMS), thalassemia, +2 more
6 terms
supplemental New Drug Application (sNDA) regulatory
"Company plans to submit mitapivat sNDA for sickle cell disease in the second quarter of 2026"
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.
accelerated approval pathway regulatory
"plan to submit our mitapivat sNDA for sickle cell disease under the U.S. accelerated approval pathway"
The accelerated approval pathway is a process that allows new medicines to be approved more quickly based on early evidence that they may be effective, rather than waiting for full proof. This can help patients access promising treatments faster, but it also means ongoing studies are needed to confirm the benefits. For investors, it highlights potential faster market entry and earlier revenue opportunities, along with some uncertainty about long-term outcomes.
Risk Evaluation and Mitigation Strategy (REMS) regulatory
"prescriptions for thalassemia were written by Risk Evaluation and Mitigation Strategy (REMS)-certified U.S. physicians"
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.
thalassemia medical
"AQVESME in thalassemia, with 242 prescriptions written as of March 31, 2026"
A genetic blood disorder that reduces the body's ability to make hemoglobin, the protein in red blood cells that carries oxygen; think of it as a factory line that produces weaker or fewer delivery trucks, so tissues get less oxygen. It matters to investors because thalassemia creates ongoing demand for therapies, diagnostics, transfusions and related care, and progress in treatments or approvals can change the market value of companies working on those solutions.
sickle cell disease medical
"submit mitapivat sNDA for sickle cell disease in the second quarter of 2026"
Sickle cell disease is an inherited blood disorder where red blood cells become rigid and crescent-shaped, causing them to clump and block small blood vessels; this leads to recurrent pain, organ damage and higher risk of infection. For investors it matters because the condition drives ongoing healthcare costs, creates demand for new drugs, gene therapies and diagnostics, affects payer and hospital economics, and can influence workforce productivity and insurance liabilities—making progress or setbacks in treatments a market-moving factor.
lower-risk myelodysplastic syndromes (LR-MDS) medical
"Tebapivat – Lower-Risk Myelodysplastic Syndromes (LR-MDS) – Agios expects to report topline results"
Earnings Snapshot
Total revenue: $20,746 thousand
Q1 2026
Total revenue
$20,746 thousand
Net loss
$99,111 thousand
Net loss per share (basic and diluted)
$1.69
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): April 29, 2026
(Exact Name of Registrant as Specified in Charter)
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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):
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Securities registered pursuant to Section 12(b) of the Act:
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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 April 29, 2026, Agios Pharmaceuticals, Inc. issued a press release announcing its results for the quarter ended March 31, 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 April 29, 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: April 29, 2026 | By: | /s/ Brian Goff | ||||||
Brian Goff Chief Executive Officer | ||||||||
Agios Reports First Quarter 2026 Financial Results and Provides Business Update
•Mitapivat (PYRUKYND® and AQVESME™) generated worldwide net revenues of $20.7 million in the first quarter of 2026, compared to $8.7 million in the first quarter of 2025
•Strong initial U.S. commercial launch of AQVESME in thalassemia, with 242 prescriptions written as of March 31, 2026
•Company plans to submit mitapivat sNDA for sickle cell disease in the second quarter of 2026
•Pipeline advancing to multiple value-driving inflection points in 2026, including two Phase 2 readouts for next-generation PK activator tebapivat
•$1.0 billion in cash, cash equivalents, and marketable securities as of March 31, 2026
CAMBRIDGE, Mass., Apr. 29, 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 first quarter ended March 31, 2026.
“Our first-quarter performance reflects strong execution and significant progress against our 2026 strategic objectives,” said Brian Goff, Chief Executive Officer, Agios. “The solid early momentum of our U.S. commercial launch of AQVESME in thalassemia highlights both the medicine’s clinical value and strong community reception. Additionally, following collaborative engagement with the FDA, we now plan to submit our mitapivat sNDA for sickle cell disease under the U.S. accelerated approval pathway in the second quarter. 2026 marks an important growth inflection point for Agios as we continue to build a sustainable rare disease company that is rooted in hematology and focused on delivering differentiated medicines that create meaningful long-term value for patients and shareholders.”
First Quarter 2026 and Recent Corporate Highlights
Mitapivat (PYRUKYND® and AQVESME™) Commercial Performance and Update
•$18.8 million in U.S. net revenue and $1.9 million in ex-U.S. net revenue in the first quarter of 2026.
oU.S. net revenue was driven by the U.S. commercial launch of AQVESME™ (mitapivat) in thalassemia in late January 2026.
oEx-U.S. net revenue reflected demand for PYRUKYND® (mitapivat) in thalassemia in Saudi Arabia.
•242 AQVESME mitapivat prescriptions for thalassemia were written by Risk Evaluation and Mitigation Strategy (REMS)-certified U.S. physicians as of March 31, 2026, driven by motivated prescribers and highly engaged patients.
R&D Highlights
•Mitapivat
oThalassemia –
In March 2026, the Emirates Drug Establishment of the United Arab Emirates (UAE) approved PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. With this approval, PYRUKYND becomes the only medicine approved in the UAE for this broad patient population.
Mitapivat is now approved for adults with thalassemia in the U.S., Saudi Arabia, and the UAE. A marketing application for mitapivat in thalassemia is currently under review by the European Commission.
oSickle Cell Disease –
Agios confirmed plans to pursue U.S. accelerated approval for mitapivat in sickle cell disease, following completion of its pre-supplemental New Drug Application (sNDA) meeting with the U.S. Food and Drug Administration (FDA).
The FDA’s accelerated approval pathway expedites the availability of medicines that can fill a medical need for a serious condition, with the requirement of a confirmatory clinical trial to convert to a traditional approval.
The company now plans to submit an sNDA for mitapivat in sickle cell disease in the second quarter of 2026.
•Tebapivat
oLower-Risk Myelodysplastic Syndromes (LR-MDS) –
Agios expects to report topline results from its Phase 2b trial in the first half of 2026. Based on findings from the Phase 2a trial, the Phase 2b open-label trial is evaluating three higher daily dose levels of tebapivat (10 mg, 15 mg, and 20 mg) over a 24-week period. The primary endpoint is the proportion of participants achieving transfusion independence, defined as being transfusion-free for at least 8 consecutive weeks during the 24-week period.
oSickle Cell Disease –
Agios expects to report topline results from its Phase 2 trial in the second half of 2026. This double-blind, randomized, placebo-controlled trial is evaluating three daily dose levels of tebapivat (2.5 mg, 5 mg, and 7.5 mg) versus matched placebo over a 12-week period. The primary endpoint is hemoglobin response, defined as a ≥1.0 g/dL increase in average
hemoglobin concentration from Week 10 through Week 12 compared with baseline.
First Quarter 2026 Financial Results
For the quarter ended March 31, 2026, net loss was $99.1 million, compared to net loss of $89.3 million for the quarter ended March 31, 2025.
•Net product revenue from U.S. sales of mitapivat (PYRUKYND and AQVESME) for the first quarter of 2026 was $18.8 million, compared to $8.7 million for the first quarter of 2025.
•Net product revenue from ex-U.S. sales of mitapivat (PYRUKYND) for the first quarter of 2026 was $1.9 million.
•Cost of sales for the first quarter of 2026 was $1.3 million.
•Research and Development (R&D) Expenses were $81.1 million for the first quarter of 2026, compared to $72.7 million for the first quarter of 2025, due to workforce-related expenses supporting pipeline advancement efforts, as well as increased mitapivat process development expenses.
•Selling, General and Administrative (SG&A) Expenses were $48.3 million for the first quarter of 2026, compared to $41.5 million for the first quarter of 2025, due to an increase in activities to support the U.S. commercial launch of AQVESME in thalassemia, as well as an increase in stock compensation expense.
•Cash, cash equivalents and marketable securities were $1.0 billion as of March 31, 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, advance the company’s existing clinical programs, and opportunistically expand its pipeline through both internally- and externally-discovered assets.
First Quarter 2026 Conference Call Information
Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s first 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), tebapivat, AG-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including mitapivat, tebapivat, AG-236 and AG-181; Agios’ expectations for the review of marketing applications for mitapivat by regulatory agencies, including the FDA and European Commission; 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 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) | ||||||||||||||||||||
| March 31, 2026 | December 31, 2025 | |||||||||||||||||||
| Cash, cash equivalents, and marketable securities | $ | 1,045,492 | $ | 1,164,438 | ||||||||||||||||
| Accounts receivable, net | 16,132 | 10,577 | ||||||||||||||||||
| Inventory | 35,087 | 32,920 | ||||||||||||||||||
| Total assets | 1,184,990 | 1,297,225 | ||||||||||||||||||
| Stockholders' equity | 1,109,115 | 1,193,114 | ||||||||||||||||||
| Consolidated Statements of Operations Data | |||||||||||
| (in thousands, except share and per share data) | |||||||||||
| (Unaudited) | |||||||||||
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Revenues: | |||||||||||
| Product revenue, net | $ | 20,746 | $ | 8,726 | |||||||
| Total revenue | 20,746 | 8,726 | |||||||||
| Operating expenses: | |||||||||||
| Cost of sales | $ | 1,319 | $ | 1,085 | |||||||
| Research and development | 81,148 | 72,743 | |||||||||
| Selling, general and administrative | 48,304 | 41,527 | |||||||||
| Total operating expenses | 130,771 | 115,355 | |||||||||
| Loss from operations | (110,025) | (106,629) | |||||||||
| Interest income, net | 10,795 | 16,087 | |||||||||
| Other income, net | 119 | 1,253 | |||||||||
| Net loss | $ | (99,111) | $ | (89,289) | |||||||
| Net loss per share - basic and diluted | $ | (1.69) | $ | (1.55) | |||||||
| Weighted-average number of common shares used in computing net loss per share – basic and diluted | 58,782,241 | 57,459,195 | |||||||||
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
How did Agios (AGIO) perform financially in the first quarter of 2026?
Agios reported Q1 2026 total product revenue of $20.746 million, up from $8.726 million a year earlier. The company posted a net loss of $99.111 million, compared with a $89.289 million net loss in Q1 2025, reflecting higher R&D and SG&A spending.
What were Agios (AGIO) mitapivat sales in Q1 2026?
Mitapivat (PYRUKYND and AQVESME) generated $20.7 million in worldwide net revenue in Q1 2026, versus $8.7 million in Q1 2025. This included $18.8 million from U.S. sales and $1.9 million from ex-U.S. markets, demonstrating strong early commercial uptake.
What is Agios (AGIO) planning for mitapivat in sickle cell disease?
Agios plans to submit a supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease in the second quarter of 2026. The company intends to pursue the U.S. FDA’s accelerated approval pathway, which requires a confirmatory trial to convert to traditional approval.
How strong is Agios (AGIO) cash position after Q1 2026?
As of March 31, 2026, Agios held $1.045 billion in cash, cash equivalents and marketable securities, down from $1.164 billion at December 31, 2025. Management believes this balance, plus anticipated product revenue and interest income, can fund key commercial and pipeline activities.
What clinical milestones are upcoming for Agios (AGIO) tebapivat program?
Agios expects to report Phase 2b topline data in lower-risk myelodysplastic syndromes in the first half of 2026. It also plans to share Phase 2 topline data in sickle cell disease in the second half of 2026, both evaluating different tebapivat dose levels and disease-specific endpoints.
How many AQVESME prescriptions were written in Q1 2026 for Agios (AGIO)?
By March 31, 2026, REMS-certified U.S. physicians had written 242 AQVESME (mitapivat) prescriptions for thalassemia. This early prescription volume reflects initial demand following the late January 2026 U.S. commercial launch targeting adult patients with thalassemia.