Analyst Conference Summary

biotechnology

conference date: July 31, 2025, @ 5:00 AM Pacific Time
for quarter ending: June 30, 2025 (Q2, second quarter 2025)

Forward-looking statements

Overview: Slow product ramp, but PDUFA set for September 7, 2025 for much larger indication.

Basic data (GAAP):

Revenue was $12.5 million, up 44% sequentially from $8.7 million, and up 45% from $8.6 million year-earlier.

Net income was negative $112 million, down sequentially from negative $89.3 million, and down from negative $96 million year-earlier.

EPS (diluted GAAP) was negative $1.93, down sequentially from $1.55, and down from negative $1.69 year-earlier.

Guidance:

Agios expects that its cash and equivalents, together with anticipated product revenue and interest income, will be sufficient to prepare for potential Pyrukynd launches in thalassemia and sickle cell disease, advance existing programs, and opportunistically expand its pipeline.

Conference Highlights:

Brian Goff, CEO of Agios said "With fewer than 40 days to our PDUFA goal date, our commercial team is prepared for the potential U.S. approval of Pyrunynd for thalassemia. In the second quarter, we made progress advancing our early- and mid-stage pipeline and remain on track to deliver topline results of the RISE UP Phase 3 trial for Pyrunynd in sickle cell disease by the end of the year. Collectively, our progress reflects our continued focus on delivering innovative medicines with the potential to transform the lives of those affected by rare diseases and deliver long-term shareholder value." Has cash to add external assets to the pipeline.

Pyrukynd (mitapivat) revenue was $12.5 million, up sequentially from $8.7 million, and up from $8.6 million year-earlier. 142 patients were on Pyrukynd. 248 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. Applied to the FDA and EU in Q4 2024, with a PDUFA of September 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 likely in early 2026.

In Q2 2025 Agios entered into a distribution agreement with Avanzanite Bioscience B.V., a rapidly growing European specialty pharmaceutical company focused on rare diseases, to distribute and commercialize Pyrukynd across the European Economic Area, the United Kingdom and Switzerland.

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 was initiated in Q3 2024, double-blind, with three dosing levels. MDS Phase 2b enrollment should complete in 2025. Tebapivat granted Orphan Drug Designation in Q3 2024. In Q2 2025 dosed first patient with tebapivat for sickle cell as well 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 Q2 2025 Agios filed an NDA and received an IND for AG-236 for polycemia vera. It is an siRNA targetting TMPRSS6.

Cash (including equivalents & securities) ended at $1.34 billion, down sequentially from $1.42 million. No debt.

GAAP operating expenses were $139.5 million, consisting of: Cost of goods $1.7 million; $92 million for R&D and $46 million for SG&A. Loss from operations was $127 million. Interest income was $14.5 million. Other income $0.5 million. Income tax $0 million.

Q&A selective summary:

Any change to safety profile for Pyrukynd? No.

No update yet on non-U.S. filing review timelines.

Safety label for thalassemia? We had some hepatocellular injury. So PKD label could be updated. But process is ongoing.

Cost of launch? We do expect to see a little bit more growth on sales expenses, upon launch. The team is ready for the new launch.

8,000 diagnosed U.S. thal patients, 6,000 are adults, 2,000 children. Will run trial in pediatric population.

We have not had disruptions in our FDA engagements.

We are exploring lower doses in the sickle cell Phase 2 trials.

OLE data timeline? Nothing definate yet, but we will share that when appropriate.

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