Mitapivat

Mitapivat (PYRUKYND ® and AQVESME™) delivered worldwide net revenues of $44.7 million in the second quarter of 2026, compared to $12.5 million in the second quarter of 2025 Strong U.S. commercial launch of AQVESME in thalassemia, with 442 cumulative prescriptions written as of June 30, 2026 sNDA for mitapivat granted FDA Priority Review in sickle cell disease with PDUFA goal date of November 1, 2026 Diversified and expanded late-stage pipeline with licensing of cevidoplenib for immune thrombocytopenia and advancement of AG-236 into Phase 2/3 development for polycythemia vera $964.8 million in cash, cash equivalents, and marketable securities as of June 30, 2026; well capitalized to support commercial execution and pipeline advancement CAMBRIDGE, Mass., July 30, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced financial results and updates for the second quarter ended June 30, 2026. “Our second-quarter performance reflects continued execution across the key priorities that will drive sustainable growth for Agios: strong commercial momentum, pipeline diversification, and strategic portfolio discipline,” said Brian Goff, Chief Executive Officer, Agios. “We are encouraged by the ongoing U.S. commercial launch of AQVESME in thalassemia, which continues to see robust engagement from both physicians and patients. We also progressed mitapivat toward a potential new indication in sickle cell disease, highlighted by the FDA granting Priority Review for our sNDA. Beyond these milestones, we strengthened our hematology pipeline with the licensing of cevidoplenib and advancement of AG-236 into Phase 2/3 development, while maintaining disciplined capital allocation. Together, these achievements underscore our ability to deliver meaningful innovation for patients and long-term shareholder value.” Second Quarter 2026 and Recent Corporate Highlights Mitapivat (PYRUKYND ® and AQVESME™) Commercial Performance and Update – $40.9 million in U.S. net revenue and $3.8 million in ex-U.S. net revenue in the second quarter of 2026. U.S. net revenue was driven by the U.S. commercial launch of AQVESME (mitapivat) in thalassemia in late January 2026. Ex-U.S. net revenue reflected anticipated demand for PYRUKYND (mitapivat) in Europe following approval for thalassemia in May 2026, as well as continued, consistent early demand in Gulf Cooperation Council (GCC) countries. As of June 30, 2026, 442 cumulative AQVESME prescriptions for thalassemia have been written by Risk Evaluation and Mitigation Strategy (REMS)-certified U.S. physicians. Business Development – Agios announced an agreement with Oscotec to license the exclusive global rights to cevidoplenib, a highly-selective, next-generation, oral spleen tyrosine kinase (SYK) inhibitor for immune thrombocytopenia (ITP). The addition of cevidoplenib diversifies Agios’ rare hematology portfolio and represents an opportunity to unlock up to $1.0 billion in peak U.S. sales potential in this indication. Agios expects to advance cevidoplenib into Phase 3 development for ITP in the first half of 2028, following completion of additional chemistry, manufacturing, and controls (CMC) development work. Research and Development (R&D) Highlights ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Mitapivat (pyruvate kinase [PK] activator) Thalassemia – The European Commission (EC) granted marketing authorization for PYRUKYND in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, with an orphan medicinal product designation. With this decision, PYRUKYND is the only medicine approved in all European Union (EU) member states for this broad patient population. Mitapivat is now approved for adults with thalassemia in the U.S., Saudi Arabia, United Arab Emirates, and EU. Sickle Cell Disease – The U.S. Food and Drug Administration (FDA) accepted Agios’ supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease with a Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date for this sNDA, submitted under the FDA’s accelerated approval pathway, is November 1, 2026. Additionally, Agios dosed the first patient in the REIGNITE Phase 3 trial, the confirmatory clinical trial required to be conducted under the accelerated approval pathway. This global trial is designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in patients with sickle cell disease aged 12 years or older. Agios also filed for regulatory approval of mitapivat for sickle cell disease in Saudi Arabia. AG-236 (siRNA targeting TMPRSS6) Polycythemia Vera – Results from Agios’ Phase 1 trial of AG-236 in healthy volunteers demonstrated sustained hepcidin control and effects on iron regulation biomarkers without the need for titration, supporting iron pathway modulation that can potentially address excess red blood cell production in polycythemia vera. The data also indicate the potential for an up to every-six-month dosing schedule. Based on these results, Agios will advance AG-236 into a Phase 2/3 development program in polycythemia vera, with initiation of the Phase 2 portion expected in the second half of 2026. AG-181 (phenylalanine hydroxylase [PAH] stabilizer) Phenylketonuria (PKU) – Agios dosed the first patient in the Phase 1b trial evaluating the safety and tolerability of AG-181 in adults with PKU. Data from this trial are expected in the second half of 2026. Tebapivat (PK activator) Lower-Risk Myelodysplastic Syndromes (LR-MDS) – Agios announced that it will not advance tebapivat in LR-MDS following results from the company's Phase 2b trial. While tebapivat demonstrated evidence of biological activity, it did not demonstrate clinical benefit in a sufficient proportion of patients or any patient subgroup to meet the company's predefined threshold for advancement in LR-MDS. Sickle Cell Disease – Agios announced that it will not advance tebapivat in sickle cell disease following results from the company’s Phase 2 trial. The data further reinforced PK activation as a clinically validated mechanism in sickle cell disease; however, they did not demonstrate a sufficiently differentiated pro to other PK activators to justify continued development of tebapivat in this indication. Second Quarter 2026 Financial Results For the quarter ended June 30, 2026, net loss was $100.7 million, compared to net loss of $112.0 million for the quarter ended June 30, 2025. Net product revenue from U.S. sales of mitapivat (PYRUKYND and AQVESME) for the second quarter of 2026 was $40.9 million, compared to $12.2 million for the second quarter of 2025. Net product revenue from ex-U.S. sales of mitapivat (PYRUKYND) for the second quarter of 2026 was $3.8 million, compared to $0.3 million for the second quarter of 2025. Cost of sales for the second quarter of 2026 was $3.0 million. Research and Development (R&D) expenses were $100.8 million for the second quarter of 2026, compared to $91.9 million for the second quarter of 2025, driven primarily by the $25.0 million up-front payment associated with the agreement with Oscotec to license cevidoplenib. Selling, General and Administrative (SG&A) expenses were $51.5 million for the second quarter of 2026, compared to $45.9 million for the second quarter of 2025, due to an increase in activities related to the U.S. commercial launch of AQVESME in thalassemia. Cash, cash equivalents and marketable securities were $964.8 million as of June 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, advance the company’s existing clinical programs, and opportunistically expand its pipeline through both internally- and externally-discovered assets. Second Quarter 2026 Conference Call Information Agios will host a conference call and live webcast today, July 30, 2026, at 8:00 a.m. ET to discuss the company’s second quarter 2026 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website ( ) 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 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 ( ) 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, AG-181 and cevidoplenib; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including mitapivat, tebapivat, AG-236, AG-181 and cevidoplenib; 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 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) June 30, 2026 December 31, 2025 Cash, cash equivalents, and marketable securities $ 964,830 $ 1,164,438 Accounts receivable, net 20,112 10,577 Inventory* 35,752 32,920 Total assets 1,108,424 1,297,225 Stockholders' equity 1,026,021 1,193,114 *June 30, 2026 balance includes $5.6 million of long-term inventory included in other non-current assets within our condensed consolidated balance sheets. Consolidated Statements of Operations Data (in thousands, except share and per share data) (Unaudited) Three Months Ended June 30, Six Months Ended June 30, 2026 2025 2026 2025 Revenues: Product revenue, net $ 44,745 $ 12,455 $ 65,491 $ 21,181 Total revenue 44,745 12,455 65,491 21,181 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 Loss from operations (110,552 ) (127,056 ) (220,577 ) (233,685 ) Interest income, net 9,730 14,513 20,525 30,600 Other income, net 119 523 238 1,776 Net loss $ (100,703 ) $ (112,020 ) $ (199,814 ) $ (201,309 ) Net loss per share - basic and diluted $ (1.69 ) $ (1.93 ) $ (3.38 ) $ (3.49 ) Weighted-average number of common shares used in computing net loss per share – basic and diluted 59,488,871 57,932,576 59,137,508 57,697,193 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

iStock, ismagilov With the second quarter earnings period underway, BioSpace examines five biotechs with meaningful catalysts that analysts and investors are watching. Biotechs are heading into the second quarter earnings period ready to pressure their Big Pharma peers with new drug launches. More than ever, smaller biotechs are choosing to go at it alone to market their drugs, rather than partner up or sell. This has created a dynamic environment among these upstart companies, with plenty of eyes watching to see if they succeed. From Agios Pharmaceuticals to Ultragenyx, BioSpace takes a look at five biotechs with major catalysts to report on over the next few weeks. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); Earnings Q2 earnings arrive as pharma picks up steam, buoyed by dealmaking, policy tailwinds Biopharma is entering its second-quarter earnings season riding high on a wave of massive deals and venture capital flow, plus a clearing of regulatory and policy overhangs. What can industry watchers expect to hear on the upcoming investor calls? July 15, 2026 · 6 min read · Tristan Manalac Read more Agios Pharmaceuticals Q2 stock performance: 35% Earnings date: July 30 After reporting the failure of a Phase 2 study for tebapivat in sickle cell disease, Agios’ management will have to show confidence in the biotech’s remaining pipeline when reporting second quarter earnings. Last week, Agios announced that the next-generation PK activator would be discontinued after failing to differentiate from the company’s own mitapivat or Novo Nordisk’s etavopivat. Analysts hadn’t given tebapivat a lot of credit heading into the readout, but the candidate served as a “narrative enricher for longer-term franchise durability,” according to Truist Securities. The failure of the candidate puts more “concentrated risk” on mitapivat, just as the drug class gets more competition from Novo Nordisk. Mitapivat is already approved as Pyrukynd for pyruvate kinase (PK) deficiency and Aqvesme for alpha- or beta-thalassemia. In the second quarter presentation, Truist will be watching for details on the thalassemia launch and the drug’s potential in sickle cell disease with tebapivat out of the running. The drug is under FDA review via the accelerated pathway for the indication with a decision date set for November 1. Arrowhead Pharmaceuticals FY3Q stock performance: 567% Earnings date: August 4 Arrowhead Pharmaceuticals has already begun its fiscal fourth quarter of 2026 on strong footing, with the announcement last week that two Phase 3 studies of plozasiran in severe hypertriglyceridemia (sHTG) were successful . The results sent the company’s shares up 20% that day to $91.48, while rival Ionis Pharmaceuticals dipped. Analysts had been anticipating the readout in the leadup to fiscal third quarter earnings. Beyond the outstanding results of those sHTG trials, analysts are watching for a second half readout for Arrowhead’s obesity programs , ARO-INHBE and ARO-ALK7. ARO-INHBE has already shown a doubling of the weight loss achieved by Eli Lilly’s tirzepatide in an interim readout from a small group of patients in two Phase 1/2a trials. “ARWR is ahead of peers across both the INHBE+ALK7 programs, w/ multiple paths forward depending on data,” Jefferies wrote in an earnings preview. Arrowhead is also expecting data for the Alzheimer’s disease therapy ARO-MAPT in the third quarter, which could help validate the company’s blood-brain barrier delivery tech. On the commercial side, Arrowhead celebrated its first ever approval late last year, when plozasiran was approved to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). As of the company’s last earnings call, the drug was “trending better” than Ionis’ rival product, according to Jefferies. The firm is expecting sales of about $6 million for the quarter. C-suite Caught In Sarepta ‘Downdraft,’ Arrowhead Becomes RNAi Knight in Shining Armor Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. August 12, 2025 · 7 min read · Annalee Armstrong Read more BridgeBio Q2 stock performance: 125% Earnings date: TBD BridgeBio is on track to deliver a strong second quarter report, as Attruby continues to steal market share in the crowded transthyretin amyloid cardiomyopathy (ATTR-CM) space, according to Jefferies. The drug is expected to make sales of $215 million for the quarter thanks to increases in front-line uptake, the firm predicted in a July 19 note to clients. This growth is expected to continue quarter-over-quarter going forward, which will in turn build investor confidence in BridgeBio’s next three planned launches in rare disease, Jefferies said. The company is working toward FDA approvals for BBP-418 in limb girdle muscular dystrophy type 2, infigratinib in alternating hemiplegia of childhood and encaleret in autosomal dominant hypocalcemia type 1. Infigratinib was approved in 2021 for cholangiocarcinoma and is marketed as Truseltiq. Each of these could mean $1 billion opportunities for BridgeBio, Jefferies noted, while Attruby is on track to deliver $3–$4 billion in peak sales in ATTR-CM. The drug faces competition from Pfizer’s Vyndaqel and Vyndamax, Alnylam’s Amvuttra and Ionis/AstraZeneca’s eplotersen in the space, but fears that BridgeBio will lose significant market share are subsiding as the market becomes more mature, according to Jefferies. Ionis Pharmaceuticals Q2 performance: 190% Earnings date: July 29 Ionis is well ahead in the marketplace with Tryngolza, which now has two approved indications in familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) but is still facing immense pressure from Arrowhead. Analysts are expecting Ionis’ overall revenue to decline by more than half when the company reports earnings this week, as the price of the drug declines significantly to match the entry into a more common sHTG indication. Guggenheim analysts have revenue estimates above consensus at $209 million. This would mark a year-over-year decline of about 54% from last year, when revenue was $452 million . The slightly optimistic take as compared to consensus stems from higher than expected estimates for R&D collaboration revenue, since Recordati paid a $30 million upfront fee for a partnership with Ionis at the end of June. With the price reduction, Guggenheim has lowered its sales expectations to $2.9 billion as compared to $3.2 billion prior. But the analysts are optimistic that the dip for Tryngolza, which is now approved for FCS and sHTG, will be short lived as the sHTG label highlighted the therapy’s ability to reduce the risk of acute pancreatitis. Recent data also showed its ability to normalize hepatic fat. Moving forward, analysts will be focused on three upcoming Phase 3 readouts, including the Lp(a) HORIZON trial of pelacarsen, which is testing the drug for reducing lipoprotein(a). Rare diseases Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026. February 18, 2026 · 3 min read · Annalee Armstrong Read more Ultragenyx 2Q performance: -1.33% Earnings date: TBD Ultragenyx has been through the wringer this year, witha high pro for a bone disease drug and an FDA rejection for a rare disease gene therapy. Heading into the third quarter, analysts are keenly watching for any details on a Phase 3 readout for apazunersen in Angelman syndrome, which could help turn the ship around. Jefferies anticipates peak worldwide sales of about $1.87 billion for the drug in 2036 if ultimately approved. Ultragenyx executives have had to make some tough decisions following the events of 2025, including implementing an expense reduction plan and layoffs . They also sold off two priority review vouchers that were awarded with the recent approvals of rare disease therapies Mepsevii and Crysvita. The biotech is awaiting two key regulatory decisions. Pariglasgene brecaparvovec, an AAV8 gene therapy, is under review for glycogen storage disease type 1a with an FDA action date of August 23. And rebisufligene etisparvovec, an AAV9 gene therapy, was accepted for priority review by the FDA with a decision date of September 19. Jefferies believes that both launches could be successful because there is unmet need in both indications. As for sales, Jefferies predicts Crysvita to come in between $500 and $520 million while Dojolvi could take home $100–110 million.