Agios Awaits Pyrukynd Decision in Thalassemia as FDA Misses Due Date

AGIO faces uncertainty as the FDA misses its Dec. 7 deadline related to the sNDA for Pyrukynd in thalassemia and offers no new timeline.

Zacks Investment Research • Dec 9, 2025

Agios Provides Update on U.S. sNDA for Mitapivat in Thalassemia

Agios today announced that the U.S. Food and Drug Administration (FDA) has not yet issued a regulatory decision on the sNDA for mitapivat in thalassemia

GlobeNewsWire • Dec 8, 2025

AGIO Stock Hits 52-Week Low on Mixed Sickle Cell Disease Study Results

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.

Zacks Investment Research • Nov 20, 2025

Agios Pharma reports mixed results in late-stage study for sickle cell disease drug

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.

Reuters • Nov 19, 2025

Why Shares in Agios Pharmaceuticals Got Crushed Today

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

The Motley Fool • Nov 19, 2025

Why Agios Plummeted, Losing Half Its Value, While Fulcrum Surged

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

Investors Business Daily • Nov 19, 2025

Why Is Agios Pharmaceuticals Stock Sinking Today?

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.

Benzinga • Nov 19, 2025

Agios Pharmaceuticals, Inc. (AGIO) Discusses Topline Results from RISE UP Phase 3 Trial of Mitapivat in Sickle Cell Disease Transcript

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.

Seeking Alpha • Nov 19, 2025

Agios Announces Topline Results from RISE UP Phase 3 Trial of Mitapivat in Sickle Cell Disease

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.

GlobeNewsWire • Nov 19, 2025

Q3 2025 Highlights - Agios Posts Strong Pyrukynd Revenue And Eyes FDA Milestones

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.

Seeking Alpha • Nov 4, 2025