On October 30, 2025 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 third quarter ended September 30, 2025.

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"As we approach year-end, we remain focused on our two key PYRUKYND milestones – the potential U.S. approval in thalassemia and the topline results from the RISE UP Phase 3 trial in sickle cell disease. Our recent engagements with these communities have underscored the urgent need for innovation and PYRUKYND’s potential to address critical gaps in care for these serious and life-threatening diseases," said Brian Goff, Chief Executive Officer, Agios. "We continued strong execution across our rare disease portfolio in the third quarter, including the completion of enrollment in our Phase 2b tebapivat trial for lower-risk MDS. We look forward to building on this strong momentum and remain steadfast in our commitment to delivering meaningful progress for the patients we serve."

Third Quarter 2025 and Recent Corporate Highlights
Commercial Performance – PYRUKYND (mitapivat)

Generated $12.9 million in net revenue for the third quarter of 2025, representing an increase of 44 percent from $9.0 million in the third quarter of 2024 and a 3 percent increase from $12.5 million in the second quarter of 2025.
262 unique patients completed prescription enrollment forms, representing an increase of 6 percent over the second quarter of 2025.
149 patients are on therapy in the U.S., inclusive of new starts and continued therapy, representing an increase of 5 percent over the second quarter of 2025.
R&D Highlights
PYRUKYND (mitapivat)

Thalassemia –
United States –
U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) of PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia by three months, to December 7, 2025.
Extension was triggered by FDA request for a Risk Evaluation and Mitigation Strategy (REMS) to address the potential risk of hepatocellular injury described in the original application.
Extension was not the result of new or additional efficacy or safety data requested by the FDA or submitted by Agios.
U.S. commercial launch preparations remain underway, and the application remains under active FDA review.
Europe –
Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of PYRUKYND in adults for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.
A final decision from the European Commission is expected by early 2026.
Gulf Cooperation Council (GCC) –
PYRUKYND received approval in Saudi Arabia for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
Commercial launch activities are underway in Saudi Arabia in partnership with NewBridge Pharmaceuticals.
In the United Arab Emirates, PYRUKYND thalassemia regulatory application remains under active review.
Sickle Cell Disease –
Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease are expected by year-end, potentially supporting a U.S. commercial launch in 2026.
Tebapivat

Lower-risk Myelodysplastic Syndromes (LR-MDS) –
Completed patient enrollment in the Phase 2b trial of tebapivat in LR-MDS. Following findings from the Phase 2a trial, the Phase 2b trial is evaluating three higher daily doses (10 mg, 15 mg, and 20 mg) versus placebo over 24 weeks. Topline results from this trial are expected in early 2026.
Third Quarter 2025 Financial Results
For the quarter ended September 30, 2025, net loss was $103.4 million dollars, compared to net income of $947.9 million dollars for the quarter ended September 30, 2024. The net income in the third quarter of 2024 was due to the milestone payment from Servier and the sale of royalty rights to Royalty Pharma, both of which were recorded in that quarter.

Net product revenue from sales of PYRUKYND for the third quarter of 2025 was $12.9 million, compared to $9.0 million for the third quarter of 2024.

Cost of sales for the third quarter of 2025 was $1.7 million.

Research and Development (R&D) Expenses were $86.8 million for the third quarter of 2025, an increase of $14.3 million compared to the third quarter of 2024. The year-over-year increase was primarily driven by increased clinical trial costs associated with the PK activation franchise.

Selling, General and Administrative (SG&A) Expenses were $41.3 million for the third quarter of 2025, representing an increase of $2.7 million compared to the third quarter of 2024, primarily driven by disciplined investment in preparation for the potential U.S. commercial launch of PYRUKYND in thalassemia.

Cash, cash equivalents and marketable securities as of September 30, 2025, were $1.3 billion compared to $1.5 billion as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND commercial launches in thalassemia and sickle cell disease, advance existing clinical programs, and opportunistically expand its pipeline through both internally and externally discovered assets.
Conference Call Information
Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s third quarter 2025 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, OCT 30, 2025, View Source [SID1234657125])