On July 30, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported financial results and updates for the second quarter ended June 30, 2026.

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"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 –
o$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.
oAs 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 –
oAgios 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.
oAgios 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
•Mitapivat (pyruvate kinase [PK] activator)
oThalassemia –
▪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.

oSickle 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)
oPolycythemia 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)
oPhenylketonuria (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)
oLower-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.

oSickle 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 profile relative 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 (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.

(Press release, Agios Pharmaceuticals, JUL 30, 2026, View Source [SID1234669525])