Agios Halts LR-MDS Tebapivat Program After Phase 2b Misses Transfusion Independence Target
AGIO•Agios’ Phase 2b trial of tebapivat in 65 lower-risk MDS patients over 24 weeks failed to meet the predefined eight-week transfusion independence threshold. Tebapivat was well tolerated across 10 mg, 15 mg and 20 mg doses with no new safety signals identified.
1. Trial Design and Outcome
Agios conducted an open-label, multicenter, 24-week Phase 2b dose-finding trial of tebapivat in 65 lower-risk myelodysplastic syndrome patients with anemia. Patients received once-daily oral doses of 10 mg, 15 mg or 20 mg and were assessed for transfusion independence, defined as eight consecutive weeks without red blood cell transfusion.
2. Safety and Tolerability
Tebapivat showed evidence of biological activity but did not achieve sufficient clinical benefit in a subgroup or the overall population to meet the company’s predefined advancement threshold. Across all dose levels, the therapy was well tolerated and presented no new safety signals.
3. Next Steps and Pipeline Impact
Agios will discontinue tebapivat development in LR-MDS but continues to advance its next-generation PK activator in sickle cell disease, with topline Phase 2 data expected in the second half of 2026. The company will refocus resources on indications showing stronger efficacy signals.
