Agios drops next-gen sickle cell drug after Phase 2 data show no advantage over Novo’s candidate

Agios Pharmaceuticals said it is halting development of its next‑generation pyruvate kinase activator, tebapivat, for sickle cell disease after a mid‑stage trial failed to demonstrate a benefit over competing assets.

The Phase 2 study enrolled 59 patients aged 16 and older and tested daily doses of 2.5 mg, 5 mg and 7.5 mg. Hemoglobin response rates at 12 weeks were 43.8%, 47.1% and 29.4% respectively, compared with 33.3% in the placebo group, showing no clear dose‑response and missing Agios’ predefined criteria for continuation.

Novo Nordisk’s investigational PK activator etavopivat achieved a 48.7% hemoglobin response in its Phase 3 HIBISCUS trial after 24 weeks, and Agios’ own mitapivat remains under FDA review. Tebapivat is not being pursued for any other indications.