# The company said the 65-patient study did not meet the predefined threshold.

*biotech · news · 2026-05-29 · Reuters*

## Key points

- The tebapivat LR-MDS trial failed to meet the predefined threshold for advancing development.
- 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. Sign up here. 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

**Companies:** Company
**Countries:** United States, India

[Read the full story on Reuters](https://www.reuters.com/business/healthcare-pharmaceuticals/agios-scraps-blood-cancer-drug-program-after-study-miss-2026-05-29/)

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