Agios Pharmaceuticals Discontinues Blood Cancer Drug Program After Mid-Stage Trial Failure
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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