Analyst Conference Summary

biotechnology

conference date: July 30, 2026, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2025 (Q2, second quarter 2026)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $ million, up % sequentially from $20.75 million, and up % from $ million year-earlier.

Net income was negative $ million, up sequentially from negative $99 million, and down from negative $ million year-earlier.

EPS (diluted GAAP) was negative $, up sequentially from negative $1.69, and down from $ year-earlier.

Guidance:

Conference Highlights:

Brian Goff, CEO of Agios said ""

Hopes to expand treatment into MDS (myelodysplastic syndrome) and sickle cell anemia. Also could diversity through acuiring external innovation.

Pyrukynd and Aqvesme (mitapivat) revenue was $ million, up % sequentially from $20.75 million, and up % from $ million year-earlier. U.S. revenue was $ million, driven by Aqvesme launch. Ex-US revenue was $ million, reflecting mainly demand from Saudi Arabia. There were 44 presciptions of Aqvesme for thalassemia in the U.S. by the end of January 2026. Sees the physician and patient reactioon to the launch as very strong. It typically takes 10 to 12 weeks to move from presciption to treatment; using a single specialty pharmacy.

The FDA approved Aqvesme (mitapivat for thalassemia) in December 2025. Q1 242 prescriptions written. Initial launch will target 4,000 U.S. patients with high frequency doctor visits. EU decision, following the positive CHMP recomendation in October 2025, likely in early 2026. In EU Agios is partnered with Avanzanite Bio. Was approved for sale in Saudi Arabia in August 2025. There are about 6,000 diagnosed adult thalassemia patients in the U.S.

The Phase 3 mitapivat sickle cell study topline results were reported November 2025. Sickle Cell sNDA to be submitted in Q2 2026. Possible sickle cell launch in 2026. Met primary endpoint and two secondary endpoints, but missed two other secondary endpoints. See Agios Announced Topline Results. EU gave orphan status.

Tebapivat (AG-946) for low-risk MDS Phase 2b completed enrollment in Q3 2025, topline data expected 1H 2026. In tebapivat for sickle cell the Phase 2 trial enrollment completed in Q4 2025, with data expected in 2H 2026.

Believes over $10 billion in potential global revenue by 2030 [with the big bump coming following sickle cell disease approval.]

The BCAT2 preclinical program targets acidemias.

AG-181 is a PAH stabilizer for phenylketonuria in an ongoing Phase 1 trial.

Cash (including equivalents & securities) ended at $ billion, down sequentially from $1.05 billion. No debt.

GAAP operating expenses were $ million, consisting of: Cost of goods $ million; $ million for R&D and $ million for SG&A. Loss from operations was $ million. Interest income was $ million. Other income $0 million. Income tax $0 million.

Q&A selective summary:

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