Analyst Conference Summary

biotechnology

conference date: April 29, 2026, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2025 (Q1, first quarter 2026)

Forward-looking statements

Overview: Increasing Pyrukynd sales, but far from break-even.

Basic data (GAAP):

Revenue was $20.75 million, up 4% sequentially from $20.0 million, and up 139% from $8.7 million year-earlier.

Net income was negative $99 million, up sequentially from negative $108 million, and down from negative $89 million year-earlier.

EPS (diluted GAAP) was negative $1.69, up sequentially from negative $1.86, and down from $1.55 year-earlier.

Guidance:

Conference Highlights:

Brian Goff, CEO of Agios said "Our first-quarter performance reflects strong execution and significant progress against our 2026 strategic objectives. The solid early momentum of our U.S. commercial launch of Aqvesme in thalassemia highlights both the medicine’s clinical value and strong community reception. Additionally, following collaborative engagement with the FDA, we now plan to submit our mitapivat sNDA for sickle cell disease under the U.S. accelerated approval pathway in the second quarter. 2026 marks an important growth inflection point for Agios as we continue to build a sustainable rare disease company that is rooted in hematology and focused on delivering differentiated medicines that create meaningful long-term value for patients and shareholders."

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 $20.75 million, up 4% sequentially from $20.0 million, and up 139% from $8.7 million year-earlier. U.S. revenue was $18.8 million, driven by Aqvesme launch. Ex-US revenue was $1.9 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.]

Believes there could be 4,000 on-label Pyrukynd patients in the U.S., which could lead to annual revenue of $200 to $225 million. Thalassemia and sickle cell potential patient numbers are much larger [18,000 and 120,000].

The BCAT2 preclinical program targets acidemias.

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

In Q3 2025 Phase 1 of AG-236 for polycemia vera was launched. It is an siRNA targetting TMPRSS6.

Cash (including equivalents & securities) ended at $1.05 billion, down sequentially from $1.16 billion. No debt.

GAAP operating expenses were $131 million, consisting of: Cost of goods $1 million; $81 million for R&D and $48 million for SG&A. Loss from operations was $110 million. Interest income was $11 million. Other income $0 million. Income tax $0 million.

Q&A selective summary:

Aqvesme demand in Q2? No comment on Q2. Excited with early launch, driven by highly motivated patients. Q1 not likely to be the run rate for future quarters.

Tebapivat in sickle cell market opportunity? It is more potent. The franchise goal in sickle cell is very substantial. The disease can support multiple treatment options. Community is looking for improved quality of life. Hoping Tebapivat will be best in class, looking forward to data.

In Thalassemia, were many of the initial scripts patients from the clinical trial? Open label extension patients are not in the scripts. There is a lag with the prescriptions before the patients get on therapy. The demand started being generated in January, but the prescriptions were mostly written starting in February.

Types of physicians prescribing? Healthy from academic, but majority are from community setting.

Implication of Novo's recent results in sickle cell disease? We have not seen their full data yet. Our Riseup data is very strong, including on hospitalizations and quality of life.

Aqvesme patient therapy duration? Too early in launch for that. We expect strong performance in thalassemia in the real world.

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