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 $ million, down % sequentially from $8.7 million, and up % from $ million year-earlier.

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

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

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said "" Has cash to add external assets to the pipeline.

Pyrukynd (mitapivat) revenue was $ million, down % sequentially from $8.7 million, and up % from $ million year-earlier. ? patients were on Pyrukynd. ? completed conscription enrollment forms. 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.

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.

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 MDD Phase 2b was initiated in Q3 2024, double-blind, with three dosing levels. Tebapivat granted Orphan Drug Designation in Q3 2024. Looking at tebapivat in sickle cell as well.

The BCAT2 preclinical program targets acidemias.

In mid-2025 Agios plans to file an NDA for AG-236 for polycemia vera. It is an siRNA targetting TMPRSS6.

Cash (including equivalents & securities) ended at $ billion, down sequentially from $1.42 million. 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 $ billion. Income tax $ million.

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