Agios Pharmaceuticals announced on May 29, 2026 that it will discontinue development of tebapivat for lower-risk myelodysplastic syndromes after a 65-patient mid-stage trial failed to meet predefined efficacy thresholds. The study did not show sufficient benefit for patients with the blood cancer. Shares fell 6.2% in premarket trading following the announcement.
Tebapivat trial failure
- ▪Agios Pharmaceuticals announced on May 29, 2026 that it will stop developing tebapivat for lower-risk myelodysplastic syndromes after a mid-stage trial failed to show enough benefit
- ▪The 65-patient study of tebapivat for lower-risk myelodysplastic syndromes did not meet the predefined threshold for developing the drug
- ▪Tebapivat was generally well-tolerated with no new safety concerns in the lower-risk myelodysplastic syndromes trial
Myelodysplastic syndromes treatment
- ▪Agios Pharmaceuticals is based in Cambridge, Massachusetts
- ▪Lower-risk myelodysplastic syndromes are a group of cancers in which the bone marrow does not make enough healthy blood cells
- ▪Myelodysplastic syndromes can sometimes progress to leukemia
- ▪Agios Pharmaceuticals is a rare disease-focused drugmaker
Trial methodology
- ▪The tebapivat trial tested three once-daily oral doses over 24 weeks in patients with lower-risk myelodysplastic syndromes and anemia
- ▪Many patients in the tebapivat trial had already received other treatments for lower-risk myelodysplastic syndromes
- ▪The main goal of the tebapivat trial was to find whether patients could go at least eight weeks without needing a blood transfusion
Pyruvate kinase activators
- ▪Tebapivat belongs to a class of medicines called pyruvate kinase activators
- ▪Pyruvate kinase activators boost an enzyme in red blood cells to help them function more effectively
Sickle cell disease program
- ▪Agios Pharmaceuticals will continue testing tebapivat for sickle cell disease
- ▪Agios Pharmaceuticals shares fell 6.2% in premarket trading on May 29, 2026 following the announcement of the tebapivat trial failure
- ▪Early results from the tebapivat sickle cell disease trial are expected in the second half of 2026
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