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Agios Pharmaceuticals, Inc. is a biopharmaceutical firm dedicated to the research and advancement of new treatments, specifically targeting cellular metabolism and related biological fields. The company's offerings include PYRUKYND (mitapivat), a medication designed to activate both wild-type and various mutated pyruvate kinase (PK) enzymes, used in the management of hemolytic anemias. Additionally, Agios is developing AG-946, which is currently undergoing Phase I clinical studies for treating hemolytic anemias and other diseases. Established in 2007, the company's main office is located in Cambridge, Massachusetts.

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) stock is trading lower after the update on the sickle cell disease program.

Agios Pharmaceuticals said on Tuesday it will stop development of its sickle cell disease treatment, sending its shares down 14% in premarket trading.

Hemoglobin response rates for tebapivat, a PK activator, were consistent with the class of medicine, but Phase 2 results did not establish a differentiated profile to support continued development in sickle cell disease Safety and tolerability of tebapivat were consistent with prior sickle cell disease trials sNDA for mitapivat, company's foundational PK activator, is under FDA Priority Review in sickle cell disease; PDUFA goal date is November 1, 2026 CAMBRIDGE, Mass., July 21, 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 topline results from the Phase 2 trial of tebapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years or older with sickle cell disease.

Agios stock surged after FDA granted priority review for mitapivat in sickle cell disease. Mitapivat showed mixed Phase 3 results, and competition is coming from Novo Nordisk.

Agios stock jumps after the FDA accepts and grants priority review to its sNDA for mitapivat in sickle cell disease, with a decision due on Nov. 1, 2026.