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biotech/news//Reuters
The company said the 65-patient study did not meet the predefined threshold.
The tebapivat LR-MDS trial failed to meet the predefined threshold for advancing development.
KEY POINTS
Tebapivat was generally well-tolerated in the LR-MDS trial, with no new safety concerns identified.
Early clinical results for tebapivat in sickle cell disease are expected in the second half of 2026.
Aqvesme, an already approved drug, is now being considered as a potential sickle cell disease treatment.
Its shares fell 1.7% in morning trading.
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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.
J.P. Morgan analyst Tessa Romero said the outcome wasn’t surprising, as the trial was risky from the beginning due to the variability of the diseases and the heavily pre-treated patient group.
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.
Aqvesme, the company's drug to treat some inherited blood disorders, is being rolled out after it was approved last year. It could also have the potential to treat sickle cell disease, Romero said.
Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Sahal Muhammed