Agios halts sickle cell drug program after disappointing phase 2 results

Agios announced it is stopping development of its next‑generation pyruvate kinase activator, tebapivat, for sickle cell disease after a phase 2 trial failed to meet expectations.

The study enrolled 59 patients who received one of three daily doses of tebapivat or placebo. Across dose groups, only 29.4% to 47.1% of participants achieved the primary endpoint, which the company deemed insufficient to justify further work.

This marks the second indication Agios has dropped in two months, following the recent termination of tebapivat’s program in lower‑risk myelodysplastic syndromes. The decision underscores the challenges of developing PK activators for sickle cell disease, even as competitors such as Novo Nordisk continue to advance similar agents.