Agios Pharmaceuticals Inc.
07/30/2026 | Press release | Distributed by Public on 07/30/2026 07:56
Quarterly Report for Quarter Ending June 30, 2026 (Form 10-Q)
Management's Discussion and Analysis of Financial Condition and Results of Operations
Forward-looking Information
The following discussion of our financial condition and results of operations should be read in conjunction with our unaudited condensed consolidated financial statements as of June 30, 2026 and for the three and six months ended June 30, 2026 and 2025, and related notes included in Part I, Item 1 of this Quarterly Report on Form 10-Q, as well as the audited consolidated financial statements and notes and Management's Discussion and Analysis of Financial Condition and Results of Operations, included in our Annual Report on Form 10-K for the year ended December 31, 2025 filed with the SEC on February 12, 2026. This Management's Discussion and Analysis of Financial Condition and Results of Operations contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on current expectations, estimates, forecasts and projections, and the beliefs and assumptions of our management, and include, without limitation, statements with respect to our expectations regarding our research, development and commercialization plans and prospects, results of operations, selling, general and administrative expenses, research and development expenses, and the sufficiency of our cash for future operations. Words such as "aim," "anticipate," "believe," "continue," "could," "estimate," "expect," "goal," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "strategy," "target," "vision," "will," "would" or the negatives of these words and similar expressions are intended to identify these forward-looking statements, although not all forward-looking statements contain these identifying words. Readers are cautioned that these forward-looking statements are predictions and are subject to risks, uncertainties and assumptions that are difficult to predict. Therefore, actual results may differ materially and adversely from those expressed in any forward-looking statements. Among the important factors that could cause actual results to differ materially from those indicated by our forward-looking statements are those discussed under the heading "Risk Factors" in Part II, Item 1A and elsewhere in this report, and in our Annual Report on Form 10-K for the year ended December 31, 2025. We undertake no obligation to revise the forward-looking statements contained herein to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events, except as required by law.
Overview
We are a commercial-stage biopharmaceutical company dedicated to redefining 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.
The lead product candidate in our portfolio, mitapivat, is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. Mitapivat is approved under the brand name AQVESME™ in the United States for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia. Mitapivat is approved under the brand name PYRUKYND®: in the United States for the treatment of hemolytic anemia in adults with PK deficiency; in the European Union, or EU, for the treatment of PK deficiency and for the treatment of non-transfusion dependent and transfusion dependent alpha- or beta-thalassemia in adult patients; in the Kingdom of Saudi Arabia for the treatment of adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia; in the United Arab Emirates for the treatment of non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in adult patients; and in Great Britain for the treatment of PK deficiency in adult patients.
We submitted a supplemental New Drug Application, or sNDA, for the U.S. accelerated approval of mitapivat in sickle cell disease, or SCD, in May 2026, which was accepted with priority review by the FDA and granted a Prescription Drug User Fee Act, or PDUFA, goal date of November 1, 2026. To support this accelerated approval pathway, we initiated REIGNITE, a phase 3 confirmatory trial designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with SCD aged 12 years or older. Additionally, in the second quarter of 2026 we filed for regulatory approval for SCD in the Kingdom of Saudi Arabia.
We are also developing (i) AG-181, our phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU; (ii) AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., or Alnylam, targeting the transmembrane serine protease 6, or TMPRSS6, gene for the potential treatment of polycythemia vera, or PV; and (iii) cevidoplenib, an oral spleen tyrosine kinase, or SYK, inhibitor in-licensed from Oscotec Inc., or Oscotec, for the potential treatment of immune thrombocytopenia, or ITP, and other autoimmune indications.
Oscotec License Agreement
On June 1, 2026, we entered into a license agreement with Oscotec, a South Korean biopharmaceutical company, under which Agios acquired the worldwide rights (subject to Oscotec's right to opt-in to develop and commercialize cevidoplenib in South Korea) to develop, manufacture, and commercialize cevidoplenib as a potential treatment for ITP and other autoimmune
indications. Because the acquired assets do not meet the definition of a business in accordance with ASC 805, Business Combinations, Agios will account for the agreement as an asset acquisition.
In accordance with the agreement, in the three months ended June 30, 2026, Agios made an up-front payment to Oscotec and recognized in-process research and development of $25.0 million, which was recorded in research and development expense within our Consolidated Statements of Operations and classified as investing activities within our Consolidated Statements of Cash Flows. In addition, Agios is responsible to pay up to $140.0 million in potential development and regulatory milestones in the U.S. and Europe, in addition to sales milestones as well as tiered royalties on future net sales, if any, of licensed products, which may be subject to specified reductions and offsets.
Sale of Oncology Business to Servier Pharmaceuticals, LLC (Servier) and Sale of Contingent Payments
In March 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier. The transaction included the sale of our entire oncology business, including our clinical-stage product candidate vorasidenib, and included a royalty of 15% of U.S. net sales of vorasidenib from the first commercial sale of vorasidenib through loss of exclusivity, or the Vorasidenib Royalty Rights. The Vorasidenib Royalty Rights are referred to as contingent payments and recognized as income when realizable.
In May 2024, we entered into a purchase and sale agreement to sell the Vorasidenib Royalty Rights to Royalty Pharma Investments 2019 ICAV, or Royalty Pharma. The sale was contingent upon FDA approval of vorasidenib and other customary closing conditions.
Upon consummation of the sale in August 2024, Royalty Pharma acquired 100% of the Vorasidenib Royalty Rights payments made by Servier on account of up to $1.0 billion in U.S. net sales for each calendar year. In addition, any such Vorasidenib Royalty Rights payments made by Servier on account of U.S. net sales in each calendar year in excess of $1.0 billion will be split, with Royalty Pharma having the rights to a 12% earn-out on those excess payments and Agios retaining the rights to a 3% earn-out on those excess payments, or the Retained Earn-Out Rights. Royalty income related to the Retained Earn-Out Rights, if any, will be recognized in the period when realizable.
Financial Operations Overview
General
Since inception, our operations have primarily focused on organizing and staffing our company, business planning, raising capital, assembling our core capabilities in cellular metabolism and classical hematology, identifying potential product candidates, undertaking preclinical studies, conducting clinical trials, establishing a commercial infrastructure, preparing for and executing on the commercial launches of PYRUKYND® and AQVESME™ and, prior to the sale of our oncology business to Servier in March 2021, marketing TIBSOVO® and IDHIFA®. Through March 2021, we financed our operations primarily through proceeds from the sale of our royalty rights, commercial sales of TIBSOVO®, funding received from our collaboration agreements, private placements of our preferred stock, our initial public offering of our common stock and concurrent private placement of common stock to an affiliate of Celgene, and our follow-on public offerings. Following the sale of our oncology business to Servier in March 2021, we have financed and expect to continue to finance our operations primarily through cash on hand, potential royalty payments with respect to the Retained Earn-Out Rights, the actual and potential future sales of PYRUKYND® and AQVESME™ and, potentially, collaborations, strategic alliances, licensing arrangements and other nondilutive strategic transactions. In addition, we may pursue opportunistic debt offerings, and equity or equity-linked offerings.
Additionally, since inception, we have historically incurred significant operating losses. Our net loss for the six months ended June 30, 2026 was $199.8 million and for the six months ended June 30, 2025 was $201.3 million. As of June 30, 2026, we had an accumulated deficit of $761.5 million. We expect to continue to incur significant expenses and net losses until such time we are able to report profitable results. We may never achieve or maintain profitability, and our net losses may fluctuate significantly from year to year. We expect that we will continue to incur significant expenses as we continue to advance and expand our commercialization activities for PYRUKYND® and AQVESME™; continue to advance clinical development and regulatory activities for mitapivat, AG-181, AG-236 and cevidoplenib; expand and protect our intellectual property portfolio, including by in-licensing or acquiring assets for pipeline growth; and hire additional commercial and development personnel.
Revenues
Our wholly owned product, PYRUKYND®, received approval from the FDA on February 17, 2022, for the treatment of hemolytic anemia in adults with PK deficiency in the United States. Our wholly owned product, AQVESME™, received approval from the FDA on December 23, 2025, for the treatment of anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in the United States. Upon FDA approval of PYRUKYND® and AQVESME™ in the United States, we began generating product revenue from sales of PYRUKYND® and AQVESME™, respectively. We sell PYRUKYND® and AQVESME™ in the United States to a limited number of specialty distributors and specialty pharmacy providers. These customers subsequently resell PYRUKYND® and AQVESME™ to pharmacies or dispense PYRUKYND® and AQVESME™ directly to patients. In addition to distribution agreements with these customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND® and AQVESME™.
In July 2024, we entered into a distribution agreement, or the NewBridge Agreement, with NewBridge Pharmaceuticals FZ-LLC, or NewBridge, pursuant to which we granted NewBridge the right to commercialize PYRUKYND® in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and the United Arab Emirates, also known as the Gulf Council Countries, or GCC. In June 2025, we entered into a distribution agreement, or the Avanzanite Agreement, with Avanzanite Bioscience B.V., or Avanzanite, pursuant to which we granted Avanzanite the right to commercialize PYRUKYND® in the European Economic Area, Switzerland, and the U.K. For further discussion of our revenue recognition policy, see Note 8, Product Revenue, to the condensed consolidated financial statements in this Quarterly Report on Form 10-Q.
In the future, we expect to continue to generate revenue from product sales of PYRUKYND® and AQVESME™. We may also generate revenue from milestone payments, upfront payments or royalties on product sales under collaborations or licensing agreements that we may enter into in the future.
Cost of Sales
Cost of sales consists primarily of manufacturing costs for sales of PYRUKYND® and AQVESME™. Based on our policy to expense costs associated with the manufacturing of our products prior to regulatory approval, certain of the manufacturing costs associated with product shipments of PYRUKYND® and AQVESME™ recorded during the three and six months ended June 30, 2026 and 2025 were expensed prior to applicable regulatory approval, and, therefore, are not included in costs of sales during the three and six months ended June 30, 2026 and 2025. The amounts excluded from cost of sales were not significant during the three and six months ended June 30, 2026 and 2025.
Inventories are reviewed periodically to identify excess or obsolete inventory based on projected sales activity as well as product shelf-life. Expired inventory is disposed of, and the related costs are recognized as cost of sales in our consolidated statements of operations, when, based on the expiry date, we do not believe we are able to sell the inventory. We have not reserved for excess or obsolete inventory during the three and six months ended June 30, 2026 and 2025.
Research and Development Expenses
Research and development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect research and development costs related to our portfolio to increase as our product candidate development programs progress. However, the successful development of our product candidates is highly uncertain. As such, at this time, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development of and to commercialize these product candidates. We are unable to predict the amount of net cash inflows from any of our products or product candidates. This is due to the numerous risks and uncertainties associated with developing medicines, including the uncertainty of:
•establishing an appropriate safety profile with an investigational new drug application, or IND, and/or NDA-enabling toxicology and clinical trials;
•successfully enrolling in, and completion of, clinical trials;
•receiving marketing approvals from applicable regulatory authorities;
•establishing compliant commercial manufacturing capabilities or making arrangements with third-party manufacturers;
•obtaining and maintaining patent and trade secret protection and regulatory exclusivity for our product candidates;
•launching commercial sales of the products, if and when approved, in the United States or in other jurisdictions,
whether alone or in collaboration with others, including pursuant to the NewBridge Agreement and Avanzanite Agreement;
•maintaining an acceptable safety profile of the products following approval; and
•complying with any approval or post-approval requirements.
A change in the outcome of any of these variables with respect to the development of any of our product candidates would significantly change the costs and timing associated with the development of that product candidate.
Research and development expenses consist primarily of costs incurred for our research activities, including our drug discovery efforts, and the development of our product candidates, which include:
•employee-related expenses, including salaries, benefits and stock-based compensation expense;
•expenses incurred under agreements with third parties, including contract research organizations, or CROs, that conduct research and development and both preclinical and clinical activities on our behalf, and the cost of consultants;
•the cost of lab supplies and acquiring, developing and manufacturing preclinical study and clinical trial materials; and
•facilities, depreciation, and other expenses, which include direct and allocated expenses for rent and the maintenance of facilities, insurance and other operating costs.
The following summarizes our products and most advanced programs:
PYRUKYND®/AQVESME™ (mitapivat): First-in-Class PK Activator
Mitapivat is an orally available small molecule and a potent activator of the wild-type and mutated PK enzymes. Mitapivat is approved under the brand name AQVESME™ in the United States for the treatment of anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia, and is the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. We commercially launched AQVESME™ in the United States in late January 2026 following our implementation of a Risk Evaluation and Mitigation Strategy, or REMS, to mitigate the risk of hepatocellular injury. Mitapivat will continue to be marketed as PYRUKYND® in the United States for the PK deficiency indication, which does not require a REMS. The AQVESME™ REMS requires liver tests prior to the first AQVESME™ dose, every four weeks thereafter for 24 weeks, and then as clinically indicated. It also includes education and certification requirements for patients, prescribing physicians, and pharmacists, which are common components of REMS.
Mitapivat is approved under the brand name PYRUKYND®: in the United States for the treatment of hemolytic anemia in adults with PK deficiency; in the EU for the treatment of PK deficiency and for the treatment of non-transfusion dependent and transfusion dependent alpha- or beta-thalassemia in adult patients; in the Kingdom of Saudi Arabia for the treatment of adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia; in the United Arab Emirates for the treatment of non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia in adult patients; and in Great Britain for the treatment of PK deficiency in adult patients.
We submitted an sNDA for the U.S. accelerated approval of mitapivat in SCD in May 2026, which was accepted with priority review by the FDA and granted a PDUFA goal date of November 1, 2026. To support this accelerated approval pathway, we initiated REIGNITE, a phase 3 confirmatory trial designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with SCD aged 12 years or older. Additionally, in the second quarter of 2026 we filed for regulatory approval for SCD in the Kingdom of Saudi Arabia.
Mitapivat has received orphan drug designation from the FDA for the treatment of thalassemia and SCD, and orphan medicinal product designation from the EMA for the treatment of SCD. Additionally, mitapivat was granted orphan drug designation for the treatment of PK deficiency by the FDA and EMA.
We have full ownership rights to PYRUKYND® and AQVESME™ and expect to fund the future development and commercialization costs related to PYRUKYND® and AQVESME™. We built our commercial infrastructure to support the commercialization of PYRUKYND® in adult PK deficiency in the United States, and have expanded this infrastructure to support the commercial launch of AQVESME™ in thalassemia in the United States and the potential commercial launch of mitapivat in SCD in the United States. In July 2024, we entered into the NewBridge Agreement, pursuant to which we granted NewBridge the right to commercialize PYRUKYND® in the GCC region. In June 2025, we entered into the Avanzanite Agreement, pursuant to which we granted Avanzanite the right to commercialize PYRUKYND® in the European Economic Area, Switzerland, and the United Kingdom. Under the NewBridge Agreement and the Avanzanite Agreement, we rely on NewBridge and Avanzanite, respectively, to assist with regulatory filings, prepare applications for pricing and reimbursement
approval, negotiate with payors, conduct medical affairs activities and import, distribute, promote and commercialize our products in accordance with applicable law in the applicable jurisdictions.
In connection with our regulatory approvals in the EU, Great Britain, Saudi Arabia and the United Arab Emirates, we provide access to PYRUKYND® to eligible patients through the Avanzanite Agreement and the NewBridge Agreement, as applicable, and on either a free of charge or for charge basis for eligible patients in those jurisdictions and other jurisdictions through a global managed access program. Revenues associated with the NewBridge Agreement, the Avanzanite Agreement and the for charge portion of our global managed access program are included in the rest of world product revenue line within Results of Operations. We continue to evaluate other options for the commercialization of PYRUKYND® outside of the United States, including through exploring potential partnership opportunities.
We are evaluating mitapivat in numerous clinical trials, including the following:
•REIGNITE, a confirmatory clinical trial required to be conducted under the U.S. accelerated approval pathway, to support our sNDA for mitapivat in SCD. REIGNITE is a global phase 3 trial designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with SCD aged 12 years or older. REIGNITE includes a 52-week, double-blind, randomized, placebo-controlled period, in which approximately 159 participants are randomized 2:1 to receive oral mitapivat (100 mg) twice daily or matched placebo. Upon completing this period, participants have the option to transition into an open-label extension period where all such participants will receive mitapivat. The primary endpoint of REIGNITE is the proportion of patients achieving transfusion-free status from Week 4 through Week 52, and the first key secondary endpoint is the number of red blood cell units transfused from Week 4 through Week 52. To further assess the anti-hemolytic benefits of mitapivat, the trial also includes key secondary endpoints measuring the average change from baseline in hemoglobin, indirect bilirubin, and lactate dehydrogenase. This trial is currently enrolling patients.
•An extension study evaluating the long-term safety, tolerability and efficacy of treatment with mitapivat in SCD patients who are 16 years of age or older from RISE UP, our completed double-blind phase 3 study evaluating the efficacy and safety of mitapivat as a potential treatment for SCD.
•Extension studies evaluating the long-term safety, tolerability and efficacy of treatment with mitapivat in pediatric patients from ACTIVATE-kids and ACTIVATE-kidsT, our completed double-blind phase 3 studies evaluating the efficacy and safety of mitapivat as a potential treatment for PK deficiency in not regularly transfused and regularly transfused patients between one and 18 years old, respectively.
•An extension study evaluating the long-term safety, tolerability and efficacy of treatment with mitapivat in patients from ACTIVATE and ACTIVATE-T, our completed pivotal trials of mitapivat in not regularly transfused and regularly transfused adult patients with PK deficiency.
•An extension study evaluating the long-term safety, tolerability and efficacy of treatment with mitapivat in patients from DRIVE PK, our completed global phase 2, first-in-patient, open-label safety and efficacy clinical trial of mitapivat in adult, not regularly transfused patients with PK deficiency.
•An extension study evaluating the long-term efficacy and safety of treatment with mitapivat in patients from ENERGIZE and ENERGIZE-T, our completed pivotal trials of mitapivat in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
During the double-blind periods of ENERGIZE and ENERGIZE-T, two patients on mitapivat experienced events of hepatocellular injury. In addition, during the open-label extension periods of both trials, a total of three patients experienced events of hepatocellular injury after switching from placebo to mitapivat. All of these events occurred within the first six months of exposure to mitapivat and liver tests improved following discontinuation of mitapivat. Based on the results of the ENERGIZE and ENERGIZE-T trials, we included in our regulatory applications hepatocellular injury as an important potential risk of mitapivat in patients with thalassemia and proposed monthly monitoring of liver tests for the first six months of treatment with mitapivat. We updated our mitapivat clinical trial protocols across all indications to incorporate monthly monitoring of liver tests for the first six months of treatment.
Tebapivat: Novel PK Activator
In May 2026, we announced that we will not advance tebapivat in lower-risk myelodysplastic syndromes, or LR-MDS, as results from the phase 2b trial of tebapivat in LR-MDS did not meet our predefined threshold to support further development in the indication.
In July 2026, we announced that we will not continue development of tebapivat in SCD, as results from the phase 2 trial of tebapivat in SCD did not establish sufficient differentiation from other PK activators that we believe necessary to support continued development.
Other Programs
We are developing AG-181, a PAH stabilizer for the potential treatment of PKU, for which we filed an IND in December 2023. We initiated a phase 1 clinical trial of AG-181 in healthy volunteers in the first quarter of 2024, initiated the multiple ascending dose portion of the trial in the second quarter of 2025, and completed the trial in December 2025. We initiated a phase 1b trial of AG-181 in patients with PKU in the first half of 2026 and expect to confirm proof of mechanism in the second half of 2026.
We entered into a license agreement with Alnylam for the development and commercialization of products containing or comprised of an siRNA preclinical development candidate discovered by Alnylam and targeting the TMPRSS6 gene, and we are developing a product candidate, AG-236, for the potential treatment of patients with PV. We filed an IND with the FDA for AG-236 for the treatment of PV, which cleared in June 2025, and initiated a phase 1 clinical trial evaluating AG-236 in healthy volunteers in July 2025. In June 2026, we announced topline results from the phase 1 clinical trial, which demonstrated dose-dependent hepcidin induction, sustained activity across doses, rapid and sustained biomarker changes and no dose-limiting toxicities. We expect to initiate the phase 2 portion of a phase 2/3 trial of AG-236 in patients with PV in the second half of 2026.
In June 2026, we entered into a license agreement with Oscotec for the development, manufacture and commercialization of cevidoplenib, a novel, highly selective, oral SYK inhibitor in-licensed from Oscotec for the potential treatment of ITP and other autoimmune indications. We expect to initiate a phase 3 clinical trial of cevidoplenib in patients with ITP in the first half of 2028, following completion of additional chemistry, manufacturing and controls development work.
Selling, General and Administrative Expenses
Selling, general and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, for personnel in executive, finance, business development, commercial, legal, information technology and human resources functions. Other significant costs include facility-related costs not otherwise included in research and development expenses, legal fees relating to patent and corporate matters, and fees for accounting and consulting services.
We anticipate that our selling, general and administrative expenses will increase in the future to support continued research and development activities, and ongoing and future commercialization activities related to our portfolio, including the ongoing commercialization of PYRUKYND®, AQVESME™ and any of our other product candidates, which may include the hiring of additional personnel.
Critical Accounting Estimates
Our critical accounting estimates are those which require the most significant judgments and estimates in the preparation of our condensed consolidated financial statements. We have determined that our most critical accounting estimates are those relating to revenue recognition, accrued research and development expenses and stock-based compensation. As of June 30, 2026, there have been no material changes to our existing critical accounting estimates discussed in Part II, Item 7 of our Annual Report on Form 10-K for the year ended December 31, 2025.
Results of Operations
Comparison of the three and six months ended June 30, 2026 and 2025
Revenues
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||||||||||||
| (In thousands) | 2026 | 2025 | 2026 | 2025 | ||||||||||||||||||||||
| Revenues: | ||||||||||||||||||||||||||
| Product revenue, net | ||||||||||||||||||||||||||
| United States | $ | 40,917 | $ | 12,151 | $ | 59,768 | $ | 20,877 | ||||||||||||||||||
| Rest of world | 3,828 | 304 | 5,723 | 304 | ||||||||||||||||||||||
| Total product revenue, net | 44,745 | 12,455 | 65,491 | 21,181 | ||||||||||||||||||||||
| Total revenue | $ | 44,745 | $ | 12,455 | $ | 65,491 | $ | 21,181 | ||||||||||||||||||
Total Revenue - Three Months Ended June 30, 2026 vs. Three Months Ended June 30, 2025 - The increase in total revenue of $32.3 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily due to an increase of $28.8 million in United States product revenue, which was primarily due to AQVESME™ sales, which we began recognizing product revenue for in the first quarter of 2026.
Total Revenue - Six Months Ended June 30, 2026 vs. Six Months Ended June 30, 2025 - The increase in total revenue of $44.3 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was due to an increase of $38.9 million in United States product revenue and an increase of $5.4 million in rest of world product revenue. The increase in United States product revenue was primarily due to AQVESME™ sales, which we began recognizing product revenue for in the first quarter of 2026, and increased volume associated with PYRUKYND®. The increase in rest of world product revenue was primarily due to increased volume associated with the NewBridge Agreement and sales associated with the Avanzanite Agreement discussed above in Overview.
Total Operating Expenses
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||||||||||
| (In thousands) | 2026 | 2025 | 2026 | 2025 | |||||||||||||||||||
| Operating expenses: | |||||||||||||||||||||||
| Cost of sales | $ | 2,996 | $ | 1,702 | $ | 4,315 | $ | 2,787 | |||||||||||||||
| Research and development | 100,754 | 91,940 | 181,902 | 164,683 | |||||||||||||||||||
| Selling, general and administrative | 51,547 | 45,869 | 99,851 | 87,396 | |||||||||||||||||||
| Total operating expenses | $ | 155,297 | $ | 139,511 | $ | 286,068 | $ | 254,866 | |||||||||||||||
Total Operating Expenses - Three Months Ended June 30, 2026 vs. Three Months Ended June 30, 2025 - The increase in total operating expenses of $15.8 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily due to an increase in research and development expenses of $8.8 million, which is described below under Research and Development Expenses, and an increase in selling, general and administrative expenses of $5.7 million, driven by an increase in commercial-related activities as we execute our launch of AQVESME™ in thalassemia.
Total Operating Expenses - Six Months Ended June 30, 2026 vs. Six Months Ended June 30, 2025 - The increase in total operating expenses of $31.2 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily due to an increase in research and development expenses of $17.2 million, which is described below under Research and Development Expenses, and an increase in selling, general and administrative expenses of $12.5 million, driven by an increase in commercial-related activities as we execute our launch of AQVESME™ in thalassemia, and an increase in stock compensation expense.
Research and Development Expenses
Our research and development expenses, by major program, are outlined in the table below:
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||||||||||
| (In thousands) | 2026 | 2025 | 2026 | 2025 | |||||||||||||||||||
| PK activator (PYRUKYND®/AQVESME™) | $ | 25,849 | $ | 29,809 | $ | 49,754 | $ | 51,130 | |||||||||||||||
| Novel PK activator (tebapivat) | 6,304 | 7,852 | 12,229 | 13,240 | |||||||||||||||||||
| PAH stabilizer (AG-181) | 338 | 1,664 | 1,306 | 2,046 | |||||||||||||||||||
| siRNA targeting TMPRSS6 (AG-236) | 2,839 | 2,426 | 4,547 | 3,922 | |||||||||||||||||||
| In-process research and development | 25,000 | 10,000 | 25,000 | 10,000 | |||||||||||||||||||
| Other research and platform programs | 694 | 1,304 | 2,049 | 2,870 | |||||||||||||||||||
| Total direct research and development expenses | 61,024 | 53,055 | 94,885 | 83,208 | |||||||||||||||||||
| Compensation and related expenses | 30,352 | 27,594 | 67,141 | 60,584 | |||||||||||||||||||
| Facilities and IT related expenses & other | 9,378 | 11,291 | 19,876 | 20,891 | |||||||||||||||||||
| Total indirect research and development expenses | 39,730 | 38,885 | 87,017 | 81,475 | |||||||||||||||||||
| Total research and development expense | $ | 100,754 | $ | 91,940 | $ | 181,902 | $ | 164,683 | |||||||||||||||
Total Research and Development Expenses - Three Months Ended June 30, 2026 vs. Three Months Ended June 30, 2025 - The increase in total research and development expenses of $8.8 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily due to an $8.0 million increase in our direct expenses. The increase in direct expenses was primarily due to an increase in in-process research and development of $15.0 million, driven by the $25.0 million up-front payment associated with the agreement with Oscotec discussed above under Overview, partially offset by a decrease in PYRUKYND®/AQVESME™ costs of $4.0 million primarily due to lower process development expenses.
Total Research and Development Expenses - Six Months Ended June 30, 2026 vs. Six Months Ended June 30, 2025 - The increase in total research and development expenses of $17.2 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was due to a $11.7 million increase in our direct expenses and a $5.5 million increase in our indirect expenses. The increase in direct expenses was primarily due to an increase in in-process research and development of $15.0 million, driven by the $25.0 million up-front payment associated with the agreement with Oscotec discussed above under Overview. The increase in our indirect expenses was primarily due to higher compensation and related expenses driven principally by workforce-related expenses and an increase in stock compensation expenses.
Other Income and Expense
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||||||||||
| (In thousands) | 2026 | 2025 | 2026 | 2025 | |||||||||||||||||||
| Interest income, net | $ | 9,730 | $ | 14,513 | $ | 20,525 | $ | 30,600 | |||||||||||||||
| Other income, net | 119 | 523 | 238 | 1,776 | |||||||||||||||||||
Other Income and Expense - Three Months Ended June 30, 2026 vs. Three Months Ended June 30, 2025 - The decrease in interest income, net was due to a lower investment balance in the three months ended June 30, 2026 compared to the three months ended June 30, 2025.
Other Income and Expense - Six Months Ended June 30, 2026 vs. Six Months Ended June 30, 2025 - The decrease in interest income, net was due to a lower investment balance in the six months ended June 30, 2026 compared to the six months ended June 30, 2025.
Net Loss
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||||||||||
| (In thousands) | 2026 | 2025 | 2026 | 2025 | |||||||||||||||||||
| Net loss | $ | (100,703) | $ | (112,020) | $ | (199,814) | $ | (201,309) | |||||||||||||||
Net Loss - Three Months Ended June 30, 2026 vs. Three Months Ended June 30, 2025 - The decrease in net loss for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily driven by the increase in product revenue discussed above under Revenues, partially offset by the increase in research and development expenses discussed above
under Research and Development Expenses, the increase in selling, general and administrative expenses discussed above under Total Operating Expenses and the decrease in interest income, net discussed above in Other Income and Expense.
Net Loss - Six Months Ended June 30, 2026 vs. Six Months Ended June 30, 2025 - The decrease in net loss for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily driven by the increase in product revenue discussed above under Revenues, partially offset by the increase in research and development expenses discussed above under Research and Development Expenses, the increase in selling, general and administrative expenses discussed above under Total Operating Expenses and the decrease in interest income, net discussed above in Other Income and Expense.
Liquidity and Capital Resources
Sources of Liquidity
Since our inception, and through March 2021, we financed our operations primarily through proceeds from the sale of our royalty rights, commercial sales of TIBSOVO®, funding received from our collaboration agreements, private placements of our preferred stock, our initial public offering of our common stock and concurrent private placement of common stock to an affiliate of Celgene, and our follow-on public offerings. Following the sale of our oncology business to Servier in March 2021, we have financed and expect to continue to finance our operations primarily through cash on hand, potential royalty payments with respect to the Retained Earn-Out Rights, the actual and potential future sales of PYRUKYND® and AQVESME™ and, potentially, collaborations, strategic alliances, licensing arrangements and other nondilutive strategic transactions. In addition, we may pursue opportunistic debt offerings, and equity or equity-linked offerings.
In March 2021, we completed the sale of our oncology business to Servier. The transaction included the sale of our entire oncology business, including our clinical-stage product candidate vorasidenib and the Vorasidenib Royalty Rights. The Vorasidenib Royalty Rights are referred to as contingent payments and recognized as income when realizable.
In May 2024, we entered into a purchase and sale agreement to sell the Vorasidenib Royalty Rights to Royalty Pharma. The sale was contingent upon FDA approval of vorasidenib and other customary closing conditions.
Upon consummation of the sale in August 2024, Royalty Pharma acquired 100% of the Vorasidenib Royalty Rights payments made by Servier on account of up to $1.0 billion in U.S. net sales for each calendar year. In addition, any such Vorasidenib Royalty Rights payments made by Servier on account of U.S. net sales in each calendar year in excess of $1.0 billion will be split, with Royalty Pharma having the rights to a 12% earn-out on those excess payments and Agios retaining the rights to a 3% earn-out on those excess payments, or the Retained Earn-Out Rights. Royalty income related to the Retained Earn-Out Rights, if any, will be recognized in the period when realizable.
Our cash, cash equivalents and marketable securities balance was $1.0 billion at June 30, 2026. The Retained Earn-Out Rights discussed above are our only committed potential external sources of funds. We cannot predict what success, if any, Servier may have in the United States with respect to the sale of vorasidenib, and consequently, we cannot estimate the amount of payments, if any, we may receive on account of the Retained Earn-Out Rights.
Cash Flows
The following table provides information regarding our cash flows for the six months ended June 30, 2026 and 2025:
| Six Months Ended June 30, | |||||||||||
| (In thousands) | 2026 | 2025 | |||||||||
| Net cash used in operating activities | $ | (176,232) | $ | (188,613) | |||||||
| Net cash provided by investing activities | 181,522 | 191,553 | |||||||||
| Net cash provided by financing activities | 5,227 | 1,684 | |||||||||
| Net change in cash and cash equivalents | $ | 10,517 | $ | 4,624 | |||||||
Net cash used in operating activities. Cash used in operating activities of $176.2 million during the six months ended June 30, 2026 was primarily due to operating expenses driven by research and development costs described above under Research and Development Expenses, partially offset by cash received from product revenues of $56.2 million and interest income of $22.1 million.
Cash used in operating activities of $188.6 million during the six months ended June 30, 2025 was primarily due to operating expenses driven by research and development costs described above under Research and Development Expenses, partially offset by cash received from interest income of $31.5 million and product revenues of $22.0 million.
Net cash provided by investing activities. Cash provided by investing activities of $181.5 million during the six months ended June 30, 2026 was primarily due to higher proceeds from maturities and sales of marketable securities than purchases of marketable securities.
Cash provided by investing activities of $191.6 million during the six months ended June 30, 2025 was primarily due to higher proceeds from maturities and sales of marketable securities than purchases of marketable securities.
Net cash provided by financing activities. Cash provided by financing activities of $5.2 million during the six months ended June 30, 2026 was due to net proceeds received from stock option exercises and purchases made pursuant to our Amended and Restated 2013 Employee Stock Purchase Plan, or 2013 ESPP.
Cash provided by financing activities of $1.7 million during the six months ended June 30, 2025, was due to net proceeds received from stock option exercises and purchases made pursuant to our 2013 ESPP.
Funding Requirements
We expect our expenses to increase as we continue the research, development and clinical trials of, seek marketing approvals for, and commercialize our product candidates in our portfolio, including as we continue to commercialize PYRUKYND® and AQVESME™ for their approved indications. If we obtain additional marketing approvals for mitapivat in SCD or in other indications, or for any of our other product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution.
We expect that our existing cash, cash equivalents and marketable securities as of June 30, 2026, 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 SCD, advance our existing clinical programs, and opportunistically expand our pipeline through both internally and externally discovered assets. Our expectations regarding our long-term funding requirements are based on assumptions that may prove to be wrong, and we may need additional capital resources to fund our operating plans and capital expenditure requirements.
Our future capital requirements will depend on many factors, including:
•the amount and timing of future revenue received from commercial sales of PYRUKYND®, AQVESME™ or any of our other product candidates for which we may receive marketing approval;
•the amount of payments, if any, we may receive on account of the Retained Earn-Out Rights;
•the costs and timing of our ongoing and future commercialization activities, including product manufacturing, sales, marketing and distribution for PYRUKYND® and AQVESME™ in the approved jurisdictions and indications and for any product candidate for which we may receive approval;
•the scope, progress, results and costs of preclinical development, laboratory testing and clinical trials for our product candidates;
•the costs associated with in-licensing or acquiring assets for pipeline growth, including the amount and timing of future milestone and royalty payments potentially payable to Alnylam and Oscotec pursuant to the respective license agreements;
•the costs, timing and outcome of regulatory review of our product candidates;
•the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending intellectual property-related claims;
•our ability to establish and maintain collaborations on favorable terms, if at all;
•our ability to successfully execute on our strategic plans;
•operational delays due to public health epidemics; and
•operational delays, disruptions and/or increased costs associated with global economic and political developments.
Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs primarily through cash on hand, potential royalty payments with respect to the Retained Earn-Out Rights, the actual and potential future sales of PYRUKYND® and AQVESME™ and, potentially, collaborations, strategic alliances, licensing arrangements and other nondilutive strategic transactions. In addition, we may pursue opportunistic debt offerings, and equity or equity-linked offerings. We do not have any committed external source of funds other than the Retained Earn-Out Rights. To the extent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing, if available, may involve agreements that include covenants limiting or restricting
our ability to take specific actions, such as incurring additional debt, selling or licensing our assets, making capital expenditures or declaring dividends.
If we raise funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates, or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed or on attractive terms, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts, or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Contractual Obligations
In June 2026, Agios entered into a license agreement with Oscotec as discussed above under Overview. Under the license agreement, Agios is responsible to pay up to $140.0 million in potential development and regulatory milestones, in addition to sales milestones as well as tiered royalties on annual net sales, if any, of licensed products, which may be subject to specified reductions and offsets.
There were no other material changes to our contractual obligations and commitments described under Management's Discussion and Analysis of Financial Condition and Results of Operations in our Annual Report on Form 10-K for the year ended December 31, 2025.
Agios Pharmaceuticals Inc. published this content on July 30, 2026, and is solely responsible for the information contained herein. Distributed via EDGAR on July 30, 2026 at 13:57 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]