Agios Pharmaceuticals — Third Quarter 2026 Earnings Summary

Financial Performance:

• Net product revenue for the quarter ended September 30, 2026, was $55.4 million, compared to $12.9 million in the same period of 2025, representing a more than fourfold increase.
• U.S. net product revenue was $48.7 million in Q3 2026, up from $12.3 million in Q3 2025; ex-U.S. net revenue was $6.6 million, up from $0.6 million.
• U.S. net revenue increased 20% sequentially, while ex-U.S. net revenue increased 74% sequentially.
• Net loss for the quarter was $65.0 million, a reduction from $103.4 million in Q3 2025.
• Cost of sales was $2.5 million for the quarter.
• Research and development (R&D) expenses were $73.5 million, down from $86.8 million in Q3 2025.
• Selling, general and administrative (SG&A) expenses were $56.5 million, up from $41.3 million in Q3 2025.
• Cash, cash equivalents, and marketable securities totaled $907.0 million as of September 30, 2026, down from $1.2 billion as of December 31, 2025.
• Accounts receivable, net, increased to $25.3 million from $10.6 million at the end of 2025.

Guidance and Future Outlook:

• The company targets achieving non-GAAP profitability in 2028.
• The PDUFA goal date for the mitapivat sickle cell disease supplemental New Drug Application (sNDA) has been set for February 1, 2027.
• Agios expects cost savings from R&D headcount reductions to be realized in 2027.
• The company anticipates that its current cash position, combined with anticipated product revenue and interest income, will provide financial independence to execute commercial launches and progress the pipeline.

Business Segments and Product Lines:

• Mitapivat (marketed as PYRUKYND® and AQVESME®) is the primary revenue driver, with demand expanding in the U.S. for thalassemia and globally for sickle cell disease.
• AQVESME cumulative prescriptions with completed start forms from REMS-certified physicians reached 619 as of September 30, 2026, with an additional 48 prescriptions in process.
• Approximately 75% of patients remain on AQVESME after six months of treatment.
• Ex-U.S. revenue growth is driven by early access programs in Europe following the late May 2026 approval of PYRUKYND for thalassemia in the EU.
• The company is advancing capital-efficient partnerships with Avanzanite Bioscience B.V. in Europe and NewBridge Pharmaceuticals in the Gulf Cooperation Council (GCC) region.
• AG-236 Phase 1 trial data in healthy volunteers supports every-six-month dosing for polycythemia vera.
• Mitapivat RISE UP Phase 3 trial data shows durable hemoglobin improvements for up to 115 weeks in sickle cell disease.

Market and Competitive Landscape:

• Agios is positioning itself as a focused, rare hematology company with a commercial foundation built on the U.S. launch of AQVESME.
• The company highlighted the breadth and depth of its rare hematology portfolio with 14 abstracts accepted at ASH 2026.
• Mitapivat is being developed for sickle cell disease, thalassemia, and polycythemia vera, with the sickle cell disease indication representing the largest opportunity.

Risks and Challenges:

• The FDA requested additional information regarding the necessity of a REMS for mitapivat in sickle cell disease, resulting in a three-month extension of the sNDA review.
• The FDA classified Agios' response to the REMS inquiry as a major amendment to the sNDA.
• Forward-looking statements are subject to risks including clinical trial results, regulatory approval timing, commercial supply, and competitive factors.

Management Commentary and Tone:

• CEO Brian Goff described the third quarter as a record for product revenue, noting that the quarter's performance outperformed the entire 2025 total.
• Management expressed conviction in mitapivat for sickle cell disease based on robust efficacy and safety data.
• The company stated it is "launch-ready" ahead of the February 1, 2027, PDUFA goal date.
• Agios emphasized a disciplined operating model and a strong balance sheet as key drivers for sustained growth.

Other Key Points:

• Agios reviewed its operating model in 2026 and plans to reduce R&D headcount by over 100 roles by year-end, a decrease of more than 35% compared to 2025.
• The company received $2.97 million in royalty income from the gain on sale of its oncology business during the quarter.
• Agios is in labeling discussions with the FDA regarding the potential REMS for mitapivat in sickle cell disease.
• The company will host a conference call and live webcast on November 5, 2026, to discuss these results.