Agios Shares Fall 13% After Phase 2b Tebapivat Trial Fails in LR-MDS
AGIO•Agios shares tumbled 13% after a Phase 2b study in 65 lower-risk myelodysplastic syndrome patients failed to meet the eight-week transfusion-independence endpoint. The company will discontinue its LR-MDS tebapivat program while advancing ongoing sickle cell disease trials with topline data due in H2 2026.
1. Phase 2b Trial Outcome
Agios halted its LR-MDS program after a 24-week Phase 2b study in 65 patients failed to achieve the predefined eight-week transfusion-independence endpoint. Participants received daily doses of 10 mg, 15 mg or 20 mg of tebapivat, but the treatment did not deliver sufficient clinical benefit to justify further development.
2. Market Reaction
Following the efficacy setback announcement, Agios shares plunged 13%, reflecting investor concerns over the lost oncology indication and the broader implications for the company’s growth trajectory.
3. Safety Findings
Despite the trial’s failure to meet its primary endpoint, tebapivat maintained a favorable safety profile with no new adverse events reported across all dose cohorts, consistent with earlier studies.
4. Pipeline Refocus on Sickle Cell
Agios will discontinue the LR-MDS indication and reallocate resources to its sickle cell disease program, expecting topline results from a Phase 2 study in the second half of 2026.
