Agios Pharmaceuticals has discontinued development of its sickle cell disease (SCD) treatment tebapivat after a mid‑stage trial failed to show sufficient benefit or differentiation compared with other pyruvate kinase (PK) activators.
A phase 2 trial evaluating tebapivat in SCD showed hemoglobin improvements but did not demonstrate a clear dose–response relationship or meaningful differentiation from existing therapies, leading the company to stop the program.
This setback marks Agios’s second SCD failure in two months, after an earlier phase 2 trial in lower‑risk myelodysplastic syndromes also fell short, and it narrows the company’s prospects in sickle cell disease to its mitapivat (Pyrukynd) program, which faces a U.S. FDA decision before November 2026.
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