Analyst Conference Summary

biotechnology

conference date: October 30, 2025, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2025 (Q3, third quarter 2025)

Forward-looking statements

Overview: Received EU CHMP positive opinion on October 17 for mitapivat for transfusion-dependent thalassemia after getting a CLR from FDA in September.

Basic data (GAAP):

Revenue was $12.9 million, up 3% sequentially from $12.5 million, and up 44% from $9.0 million year-earlier.

Net income was negative $103 million, up sequentially from negative $112 million, and down from $948 million year-earlier.

EPS (diluted GAAP) was negative $1.78, up sequentially from negative $1.93, and down from $16.22 year-earlier.

Guidance:

Expects continued robust Pyrukynd growth.

Conference Highlights:

Brian Goff, CEO of Agios said "As we approach year-end, we remain focused on our two key Pyrukynd milestones: the potential U.S. approval in thalassemia and the topline results from the RISE UP Phase 3 trial in sickle cell disease. Our recent engagements with these communities have underscored the urgent need for innovation and Pyrukynd’s potential to address critical gaps in care for these serious and life-threatening diseases." Cash will be used for product launches and to extend rare disease franchise.

Pyrukynd (mitapivat) revenue was $12.9 million, up 3% sequentially from $12.5 million, and up 44% from $9.0 million year-earlier. 149 patients were on Pyrukynd. 262 completed conscription enrollment forms since launch. Slow adoptation is because it is an ultra-rare disease with long times to confirm diagnosis.

Completed enrollment of Phase 3 sickle cell study in Q3 2024, with data expected late 2025, possible sickle cell launch in 2026. EU gave orphan status.

Reported positive mitapivat full Phase 3 transfusion-dependent thalassemia results in December 2024. FDA PDUFA now December 7, 2025. Initial conversations with payers have been positive. 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. There are about 6,000 diagnosed adult thalassemia patients in the U.S.

Working on tailored commercial launch strategies for the various upcoming PK treatable indications. In Q2 2024 Agios entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of Pyrukynd for Thalassemia in the Gulf Cooperation Council region. NewBridge, a leading will commercialize Pyrukynd in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates. There are an estimated 70,000 Thalassemia patients in the GCC area.

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].

Tebapivat (AG-946) for low-risk MDS Phase 2b completed enrollment in Q3 2025. Tebapivat granted Orphan Drug Designation in Q3 2024. In Q2 2025 dosed first patient with tebapivat for sickle cell in the Phase 3 trial.

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 $1.26 billion, down sequentially from $1.34 billion. No debt.

GAAP operating expenses were $130 million, consisting of: Cost of goods $2 million; $87 million for R&D and $41 million for SG&A. Loss from operations was $117 million. Interest income was $13 million. Other income $0 million. Income tax $0 million.

Q&A selective summary:

Thalassemia review process, Rems program? Rems was included because of chance of hepatocellular injury. Rems programs vary. Cannot comment on while review is in process.

External asset search? We are open minded. We are looking at a number of indications and opportunities, but likely in rare diseases. We like early derisking opportunities, prefering best in class.

Liver monitoring requirements in Saudi Arabia? Once a month monitoring for the first six months. That is the likely EU label as well.

We do not anticipate the rems requirement in the U.S. will be a barrier to prescriptions.

Tebapivat for MDS v luspatercept? We will take luspatercept into account in our plans, but there is still a huge unmet medical need in MDS.

Have there been any additional liver events as you follow people? For thalassemia nothing has changed that would change our understanding of the issue.

Thalessemia is much more common in Saudi Arabia (rate of) than in the U.S.

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