Agios Pharmaceuticals (AGIO) investor relations material

Executive summary

• Achieved $44.7 million in Q2 2026 net revenue, a 3.6x year-over-year increase, driven by the U.S. AQVESME launch in thalassemia and 442 cumulative prescriptions from REMS-certified physicians as of June 30, with expanding demand in the U.S. and internationally.

• Advanced pipeline with FDA acceptance of mitapivat sNDA for sickle cell disease (PDUFA date: Nov 1, 2026), in-licensed cevidoplenib for ITP, and progressed AG-236 into Phase 2/3 for polycythemia vera.

• Ended quarter with ~$1 billion in cash, cash equivalents, and marketable securities, supporting ongoing commercial and R&D activities.

• Net loss for Q2 2026 was $100.7 million, improved from $112 million year-over-year, reflecting higher revenue offset by increased R&D and SG&A expenses.

• Commercial launch of AQVESME in the U.S. for thalassemia began in January 2026, with regulatory filings for mitapivat in sickle cell disease underway.

Financial highlights

• Q2 2026 net product revenue: $44.7 million ($40.9M U.S., $3.8M ex-U.S.), up from $12.5 million in Q2 2025.

• Cost of sales: $3 million; R&D expense: $100.8 million (up from $91.9M YoY, including $25M cevidoplenib payment); SG&A expense: $51.5 million (up from $45.9M YoY, reflecting launch activities).

• Net loss: $100.7 million in Q2 2026, improved from $112 million in Q2 2025.

• Approximately $5 million in Q2 2026 revenue attributed to one-time stocking and gross-to-net benefit.

• U.S. product revenue for the six months was $59.8 million, with rest of world revenue at $5.7 million.

Outlook and guidance

• 2026 U.S. PK deficiency revenue expected at $45M-$50M.

• Full-year operating expenses to remain flat versus 2025, excluding $25M cevidoplenib payment.

• Priorities: drive AQVESME launch, prepare for sickle cell disease approval, advance pipeline, maintain financial discipline.

• Ex-U.S. revenues expected to remain modest as regions ramp up; sickle cell launch not expected to materially contribute to 2026 revenue.

• Cash and investments expected to fund operations for at least the next twelve months.