Tebapivat (AG-946)

July 21, 2026: Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) reported topline results from a Phase 2 trial of tebapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease patients ≥16 years. In the 12-week, double-blind, placebo-controlled study (n=59; randomized 2:2:2:1 to tebapivat 2.5 mg, 5.0 mg, 7.5 mg once daily, or placebo), hemoglobin and hemolysis improvements were observed across dose levels, consistent with PK activation.

The primary endpoint (hemoglobin response defined as ≥1.0 g/dL increase in average hemoglobin at Weeks 10–12 vs baseline) was achieved by 43.8% (7/16), 47.1% (8/17), and 29.4% (5/17) in the 2.5 mg, 5.0 mg, and 7.5 mg arms, respectively, and 33.3% (3/9) with placebo. Safety/tolerability was consistent with prior sickle cell disease trials. Agios concluded the results did not show sufficient differentiation to support continued development and will not advance tebapivat in sickle cell disease.

Separately, Agios noted its supplemental NDA for accelerated approval of mitapivat in sickle cell disease is under FDA Priority Review, with a PDUFA goal date of November 1, 2026.