Clinical TrialMay 29, 2026, 07:01 AM
Agios Halts Tebapivat Development for LR-MDS After Phase 2b Trial
AI Summary
Agios Pharmaceuticals announced it will not advance tebapivat, an oral pyruvate kinase activator, in lower-risk myelodysplastic syndromes (LR-MDS). This decision follows Phase 2b trial results that did not meet the company's predefined threshold for further development in this indication. While the drug showed biological activity and was well tolerated with no new safety signals, clinical benefit was not observed in a sufficient proportion of patients. Agios will continue to evaluate tebapivat in a Phase 2 trial for sickle cell disease, with topline data expected in the second half of 2026.
Key Highlights
- Agios will not advance tebapivat in lower-risk myelodysplastic syndromes (LR-MDS).
- Phase 2b trial results did not meet predefined threshold for further development.
- Tebapivat was well tolerated across all dose levels with no new safety signals.
- The open-label, multicenter Phase 2b trial included 65 patients with LR-MDS and anemia.
- Primary endpoint was 8 consecutive weeks of transfusion independence.
- Tebapivat continues to be evaluated in a Phase 2 trial for sickle cell disease.
- Topline data from the sickle cell disease Phase 2 trial is anticipated in H2 2026.
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