Agios Pharmaceuticals (AGIO) investor relations material

Executive summary

• Net revenues reached $20.7 million in Q1 2026, a 138% year-over-year increase from $8.7 million, driven by the U.S. launch of AQVESME in thalassemia and increased PYRUKYND sales.

• 242 AQVESME prescriptions were written by REMS-certified U.S. physicians as of March 31, 2026, reflecting strong initial demand.

• Net loss increased to $99.1 million from $89.3 million year-over-year, reflecting higher R&D and SG&A expenses.

• Mitapivat is approved for thalassemia in the U.S., Saudi Arabia, and UAE, with a European application under review and sNDA for sickle cell disease planned for Q2 2026.

• Robust pipeline progress includes upcoming Phase 2 data for tebapivat in MDS and sickle cell disease.

Financial highlights

• Q1 2026 net revenues were $20.7 million ($18.8M U.S., $1.9M ex-U.S.), with product revenue up $12.0 million year-over-year.

• R&D expenses rose to $81.1 million, up $8.4 million year-over-year, and SG&A expenses increased to $48.3 million, up $7 million.

• Cost of sales was $1.3 million, maintaining a high gross margin.

• Net loss per share was $(1.69), with weighted-average shares outstanding at 58.8 million.

• Cash, cash equivalents, and marketable securities totaled $1.0 billion at March 31, 2026, down from $1.2 billion at year-end 2025.

Outlook and guidance

• Operating expenses for 2026 are expected to remain approximately flat versus 2025.

• Focus remains on maximizing AQVESME launch, advancing mitapivat sNDA for sickle cell disease, and pipeline diversification.

• Two Phase 2 tebapivat readouts are expected in 2026, targeting lower-risk MDS in H1 and sickle cell disease in H2.

• Cash and anticipated revenues are expected to support ongoing launches and pipeline advancement.