Agios Pharmaceuticals

AGIO

Market cap $1.62B

AQVESME™ (mitapivat) U.S. commercial launch in thalassemia underway following December 2025 FDA approval Pre-sNDA meeting with FDA for mitapivat in sickle cell disease anticipated in first quarter of 2026, with planned U.S. regulatory submission to follow Company progressing early- and mid-stage pipeline in multiple high-value indications Clear path to profitability through the company's existing commercial presence in thalassemia and PK deficiency, with potential to achieve over $1 billion in peak global sales CAMBRIDGE, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced its 2026 strategic priorities and key milestones anticipated during the year.

Agios today announced that its management team is scheduled to present at the 44th Annual J.P. Morgan Healthcare Conference on Wednesday, January 14, 2026

Agios Pharmaceuticals (AGIO) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings estimate revisions for the stock doesn't suggest further strength down the road.

Agios Pharmaceuticals, Inc. (AGIO) Discusses FDA Approval of AQVESME for Anemia in Alpha and Beta Thalassemia Transcript

Agios Pharmaceuticals upgraded to buy (market perform) after FDA approval of Aqvesme for Alpha- or Beta-Thalassemia, despite boxed warning for liver injury. AGIO's commercial upside from thalassemia is capped, with peak sales estimates around $500M, insufficient alone for profitability; SCD approval remains a major potential catalyst. AGIO maintains a strong cash position of $1.3B versus $101M in liabilities, providing ample runway for commercialization and further development.

Agios Pharmaceuticals wins FDA approval for Aqvesme in alpha- and beta-thalassemia, making mitapivat the only approved option, despite a new REMS.

The new indication is relatively rare in the U.S., but pricing is substantial, and the need for the treatment is significant. The positive update turns the company's fortunes around after a disappointing update earlier in the year.

Shares of Agios Pharmaceuticals surged nearly 15% in premarket trading on Wednesday after the US Food and Drug Administration approved an expanded use of its drug mitapivat, strengthening investor optimism around the company's growth prospects.

Shares of Agios Pharmaceuticals jumped nearly 12% premarket on Wednesday after the U.S. Food and Drug Administration approved the expanded use of its drug for the treatment of a type of blood disorder.

AQVESME is the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia Marketed under AQVESME brand name in the U.S. for thalassemia indication; PYRUKYND® (mitapivat) remains the U.S. brand name for PK deficiency indication  AQVESME expected to be available in late January 2026, following AQVESME REMS program implementation Company will host investor conference call and webcast tomorrow, December 24 at 8:00 a.m. ET CAMBRIDGE, Mass.