May 29 (Reuters) – Agios Pharmaceuticals said on Friday it will stop developing its experimental drug for a form of blood cancer after a mid-stage trial failed to show enough benefit, marking a setback for the rare disease-focused drugmaker.
Its shares fell 6.2% in premarket trading.
The company said the 65-patient study did not meet the predefined threshold for developing the drug, named tebapivat, to treat lower-risk myelodysplastic syndromes (LR-MDS), a group of cancers in which the bone marrow does not make enough healthy blood cells. The diseases can sometimes progress to leukemia.
The trial tested three once-daily oral doses of tebapivat over 24 weeks in patients with LR-MDS and anemia, many of whom had already received other treatments. The main goal was to find whether patients could go at least eight weeks without needing a blood transfusion.
The drugmaker said tebapivat was generally well-tolerated, with no new safety concerns.
Tebapivat belongs to a class of medicines called pyruvate kinase (PK) activators, which boost an enzyme in red blood cells to help them function more effectively.
The Cambridge, Massachusetts-based company said it will continue testing the drug for sickle cell disease, with early results expected in the second half of 2026.
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Sahal Muhammed)
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