Shares drop following disappointing mid-stage trial outcome
Agios Pharmaceuticals (NASDAQ:AGIO) came under pressure on Friday, with shares falling 13% after the company announced it would halt development of tebapivat for lower-risk myelodysplastic syndromes (LR-MDS).
The decision follows results from a Phase 2b study that failed to achieve the internal criteria required to support further development of the oral pyruvate kinase activator in this indication, representing a setback for the biotechnology group.
Trial failed to demonstrate sufficient clinical benefit
The open-label, multicentre study evaluated tebapivat over a 24-week treatment period in 65 patients with lower-risk myelodysplastic syndromes and anaemia.
Participants received once-daily doses of 10mg, 15mg or 20mg, with the primary endpoint focused on transfusion independence. The company defined success as patients remaining free from blood transfusions for at least eight consecutive weeks during the treatment period.
Although the trial produced signs of biological activity, the treatment did not generate meaningful clinical benefit in enough patients to justify advancing the programme into later-stage development.
Safety profile remained favourable
Despite the disappointing efficacy outcome, Agios reported that tebapivat was generally well tolerated across all dose groups.
The company said no new safety concerns emerged during the study, and the overall safety profile remained consistent with previous observations.
Management highlights complexity of the disease
Commenting on the results, Agios executives noted the challenges involved in developing effective therapies for lower-risk myelodysplastic syndromes.
“The results from the Phase 2b trial underscore the biological complexity of lower-risk myelodysplastic syndromes and the challenges of identifying patients most likely to benefit,” said Sarah Gheuens, Chief Medical Officer and Head of R&D at Agios.
The outcome reinforces the difficulty of treating LR-MDS, a group of bone marrow disorders that can cause chronic anaemia and often require ongoing blood transfusions.
Focus shifts to sickle cell disease programme
While development in LR-MDS has been discontinued, Agios remains committed to exploring tebapivat’s potential in other indications.
The company said it continues to see promise for the drug as a treatment for sickle cell disease and is progressing clinical development in that area.
Agios expects to report topline results from an ongoing Phase 2 study evaluating tebapivat in sickle cell disease during the second half of 2026.
Investors will now be looking to that programme as the next major catalyst for the asset following the setback in myelodysplastic syndromes.