Agios Pharmaceuticals

AGIO

Market cap $1.7B

Agios slides to a 52-week low after mixed phase III RISE UP data show a hemoglobin boost but no significant cut in pain crises.

Agios Pharmaceuticals, Inc. ( AGIO ) Discusses Topline Results from RISE UP Phase 3 Trial of Mitapivat in Sickle Cell Disease November 19, 2025 8:00 AM EST Company Participants Morgan Sanford Brian Goff - CEO & Director Sarah Gheuens - Chief Medical Officer and Head of Research & Development Tsveta Milanova - Chief Commercial Officer Conference Call Participants Biree Andemariam Eric Schmidt - Cantor Fitzgerald & Co., Research Division Salveen Richter - Goldman Sachs Group, Inc., Research Division Ellen Horste - TD Cowen, Research Division Alec Stranahan - BofA Securities, Research Division Emily Bodnar - H.C. Wainwright & Co, LLC, Research Division Tessa Romero - JPMorgan Chase & Co, Research Division Luca Issi - RBC Capital Markets, Research Division Presentation Operator Good morning, and welcome to the Agios Pharmaceuticals Investor Conference Call and Webcast.

Despite missing a key endpoint, there's still likely to be value in mitapavit. Management plans for a marketing application early in 2026.

Agios Pharmaceuticals stock shed about half its value Wednesday after the company's sickle cell disease treatment lagged expectations.

Agios Pharmaceuticals Inc. (NASDAQ: AGIO) stock plunged on Wednesday after the company released topline results from RISE UP Phase 3 data for mitapivat in sickle cell disease.

Agios Pharmaceuticals said on Wednesday its sickle cell drug met one of its two main goals in a late-stage study of patients aged 16 and older, but failed to show a statistically significant reduction in pain crises.

Trial met primary endpoint of hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect bilirubin Trial showed trend favoring mitapivat but did not meet statistical significance in primary endpoint of annualized rate of SCPCs (pain crises), and the key secondary endpoint of change from baseline in PROMIS Fatigue was not met Patients in the mitapivat arm who achieved hemoglobin response had clinically meaningful benefits in SCPC-related endpoints and PROMIS Fatigue Favorable safety profile observed in RISE UP Phase 3 trial was consistent with that observed in prior mitapivat sickle cell disease trials Company will share the comprehensive results from the RISE UP clinical program with the FDA and intends to submit for potential U.S. regulatory approval Company will host investor conference call and webcast today at 8:00 a.m. ET CAMBRIDGE, Mass.

Agios Pharmaceuticals delivered strong Q3'25 results, with revenue up 44% year-over-year and continued momentum in its rare-disease pipeline. AGIO's key catalysts include the FDA decision on Pyrukynd's thalassemia sNDA in December 2025 and topline data from the 'RISE UP' sickle cell trial. Despite premium valuation, the company maintains a robust balance sheet and is positioned for significant growth if regulatory approvals and market expansion materialize.

CAMBRIDGE, Mass., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 67th American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2025) in Orlando, Florida, December 6-9, 2025.

Agios posts a narrower-than-expected third-quarter loss as Pyrukynd drives a 44% revenue rise, with potential label expansions on the horizon.