Analyst Conference Summary

biotechnology

conference date: November 6, 2026, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2026 (Q3, third quarter 2026)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $ million, up % sequentially from $45 million, and up % from $5 million year-earlier.

Net income was negative $ million, down sequentially from negative $101 million, and up from negative $ million year-earlier.

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

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said ""

Pyrukynd and Aqvesme (mitapivat) revenue was $ million, up % sequentially from $44.7 million, and up % from $ million year-earlier. U.S. revenue was $ million, driven by Aqvesme launch. Ex-US revenue was $ million. In Q2 approved in EU for anemia from transfusion-dependent and non-transfusion-dependent thalassemias. 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. Q3 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. There are about 6,000 diagnosed adult thalassemia patients in the U.S.

Mitapivat for Sickle Cell sNDA submitted in Q2 2026. PDUFA November 1, 2026. Possible sickle cell launch in 2026. The Phase 3 mitapivat sickle cell study topline results were reported November 2025. Met primary endpoint and two secondary endpoints, but missed two other secondary endpoints. See Agios Announced Topline Results. EU gave orphan status. In Q3 2026 Agios dosed the first patient in the Phase 3 confirmatory trial, required under the accelerated approval pathway.

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.

in Q2 2026 licensed cevidoplenib for immune thrombocytopenia, from Oscotec. Expects to begin Phase in 1H 2028.

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

In Q2 2026 advanced AG-236 into Phase 2/3 development for polycythemia vera. This follows positive Phase 1 results.

The BCAT2 preclinical program targets acidemias.

AG-181 is a PAH stabilizer for phenylketonuria in Phase 1 trial. Data expected by end of 2026.

In Q2 Agios it will not continue to develop tebapivat, in MDS of SCD, due to insufficiently differentiated results in Phase 2 trials.

Cash (including equivalents & securities) ended at $ million, down sequentially from $965 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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