Shares of Agios Pharmaceuticals (NASDAQ:AGIO) tumbled 13% on Friday after the Cambridge, Massachusetts-based biopharmaceutical company announced it will not advance tebapivat for lower-risk myelodysplastic syndromes (LR-MDS). The decision follows a Phase 2b trial whose results did not meet the company's predefined threshold to justify further development in that patient population.
The open-label, multicenter trial ran for 24 weeks and enrolled 65 patients with LR-MDS and anemia. Participants received once-daily oral tebapivat at one of three dose levels - 10 mg, 15 mg, or 20 mg - during the study window. The trial's primary endpoint was transfusion independence, specified as eight consecutive weeks without requiring a transfusion within the 24-week treatment period.
According to Agios, tebapivat produced evidence of biological activity in the study, but the proportion of patients who achieved clinical benefit was insufficient to clear the company's advancement threshold for LR-MDS. The company noted the drug was generally well tolerated across all tested doses and that no new safety signals emerged during the trial.
"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.
Despite the setback in LR-MDS, Agios said it continues to view tebapivat as a potential therapy for sickle cell disease. The company expects to release topline data from a Phase 2 sickle cell disease trial in the second half of 2026.
Context and market impact
The public reaction to the LR-MDS outcome was immediate, with the company's shares declining in session on the announcement. Agios' decision to stop advancing tebapivat in LR-MDS reflects the company meeting its internal criteria for progression, and underscores the difficulty of translating biological activity into a clinically meaningful response in this heterogeneous disease.
Next steps stated by the company
- Agios will not pursue further development of tebapivat for LR-MDS based on the Phase 2b results.
- The company will continue to evaluate tebapivat in sickle cell disease and plans to report Phase 2 topline data in H2 2026.
Investors and market participants will likely watch the upcoming sickle cell trial readout for indications of tebapivat's clinical utility in that separate indication.