Agios halts sickle-cell development of tebapivat after Phase 2 fails to show differentiation

Agios Pharmaceuticals announced on 21 July 2026 that it will not advance its PK activator tebapivat in sickle cell disease. In the Phase 2 trial, haemoglobin response rates were consistent with the class of medicine but the results did not establish a differentiated profile to support continued development in the indication. Safety and tolerability were consistent with prior sickle cell disease trials.

The setback does not affect Agios's foundational PK activator, mitapivat: a supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease is under FDA Priority Review. Agios said it will continue to evaluate tebapivat in other potential indications.