On April 29, 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 first quarter ended March 31, 2026.

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"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.
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 demand for PYRUKYND (mitapivat) in thalassemia in Saudi Arabia.

242 AQVESME 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
Thalassemia –
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.
Sickle 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
Lower-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.

Sickle 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.

(Press release, Agios Pharmaceuticals, APR 29, 2026, View Source [SID1234664892])