Analyst Conference Summary

biotechnology

conference date: May 1, 2025, @ 5:00 AM Pacific Time
for quarter ending: March 31, 2025 (Q1, first 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, up % sequentially from $10.7 million, and up % from $ million year-earlier.

Net income was $ million, down sequentially from negative $96.5 million, and down from negative $ million year-earlier.

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

Guidance:

none

Conference Highlights:

Brian Goff, CEO of Agios said ""

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.

Pyrukynd (mitapivat) revenue was $ million, up % sequentially from $10.7 million, and up % from $ million year-earlier. 130 patients were on Pyrukynd. 223 completed conscription enrollment forms. Slow adoptation is because it is an ultra-rare disease with long times to confirm diagnosis.

The Phase 3 trial for pediatric PK deficiency, regularly transfused, completed enrollment in Q2 2024. Topline results for regularly transfused children did not reach pre-specified statistical significance, but were clinically meaningful. The not regularly transfused study topline results are expected in 2025.

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.

Vorasidenib (now Voranigo) was approved by the FDA in August 2024. In Q2 2024 Agios announced a $905 million agreement with Royalty Pharma, selling its vorasidenib royalty rights. $905 million was paid upon FDA approval of the drug. Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion, and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios retains a 3% royalty on annual U.S. net sales greater than $1 billion.

Cash (including equivalents & securities) ended at $ billion, down sequentially from $1.5 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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