- 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.
The Phase 2 trial was designed to characterize the dose-response relationship of tebapivat in sickle cell disease and evaluate whether its profile was meaningfully differentiated relative to other PK activators. The trial included a 12-week, double-blind, placebo-controlled period in which 59 participants were randomized 2:2:2:1 to receive one of three once-daily (QD) tebapivat doses (2.5 mg, 5.0 mg, or 7.5 mg) or placebo.
Over the 12-week treatment period, improvements in hemoglobin levels and hemolysis were observed across all tebapivat dose levels, consistent with the established PK activation mechanism. The trial’s primary endpoint of hemoglobin response (≥1.0 g/dL increase in average hemoglobin concentration from Weeks 10 through 12 compared with baseline) was achieved by 43.8% (n=7/16), 47.1% (n=8/17), and 29.4% (n=5/17) in the 2.5 mg QD, 5.0 mg QD, and 7.5 mg QD tebapivat arms, respectively, and 33.3% (n=3/9) in the placebo arm. The safety and tolerability of tebapivat in the Phase 2 trial were consistent with that observed in prior sickle cell disease trials. The trial did not demonstrate the level of differentiation required to support continued development; therefore, Agios has decided not to advance tebapivat in sickle cell disease.