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: Revenue continues to grow rapidly on a small base.

Basic data (GAAP):

Revenue was $45 million, up 117% sequentially from $20.75 million, and up 260% from $12.5 million year-earlier.

Net income was negative $101 million, down sequentially from negative $99 million, and up from negative $112 million year-earlier.

EPS (diluted GAAP) was negative $1.69, flat sequentially from negative $1.69, and up from $1.93 year-earlier.

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "Our second-quarter performance reflects continued execution across the key priorities that will drive sustainable growth for Agios: strong commercial momentum, pipeline diversification, and strategic portfolio discipline. We are encouraged by the ongoing U.S. commercial launch of Aqvesme in thalassemia, which continues to see robust engagement from both physicians and patients. We also progressed mitapivat toward a potential new indication in sickle cell disease, highlighted by the FDA granting Priority Review for our sNDA. Beyond these milestones, we strengthened our hematology pipeline with the licensing of cevidoplenib and advancement of AG-236 into Phase 2/3 development, while maintaining disciplined capital allocation. Together, these achievements underscore our ability to deliver meaningful innovation for patients and long-term shareholder value."

Pyrukynd and Aqvesme (mitapivat) revenue was $44.7 million, up 117% sequentially from $20.75 million, and up 260% from $12.5 million year-earlier. U.S. revenue was $40.9 million, driven by Aqvesme launch. Ex-US revenue was $3.8 million, reflecting mainly demand from Saudi Arabia. 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. Q2 442 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.

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 $965 million, down sequentially from $1.05 billion. No debt.

GAAP operating expenses were $155 million, consisting of: Cost of goods $3 million; $101 million for R&D and $52 million for SG&A. Loss from operations was $111 million. Interest income was $10 million. Other income $0 million. Income tax $0 million.

Q&A selective summary:

Time on treatment? We had faster than anticipated transitions. We won't know the continuation rate until the second half of the year. Positive feedback has been strong, so we believe the continuation rates will be similar to the study rates. We are starting to see repeat prescriptions.

Expanded access program, continuations? Open label data showed high continuation for those who completed the clinical trials. Good maintenance of response. For Energize trial we had over 90% continuation.

Sickle Cell label, pricing? We have not decided which brand name will be used. May not warrant the REMS (Risk Evaluation and Mitigation Strategies). Won't know pricing and label until the decision and launch. High unmet need.

Insurer coverage? Prescription growth and revenue growth will not be strictly correlated in 2H. Will see about REMS and reimbursement.

Gross to net? Expect range to continue at 10% to 20%.

6 month evaluations? There is some variation in how physicians define benefits. Look at transfusion reductions. Mostly we hear they decide on a patient by patient basis. How patients feel is also important. For non-transfusion they will look at blood data. We are very encouraged so far.

Sickle Cell launch timeline? We don't think that revenue will be material in 2026.

LOE for mitapivat? LOE 2035, but with potential for extensions.

Medicaid for sickle cell? We will provide pricing at time of approval, driven by label and competition. Population has a higher medicaid proportion, so gross-to-net will go to a higher level.

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