Small molecule · Activator of pyruvate kinase R (PKLR) · developed by Agios Pharmaceuticals
Tebapivat is an oral small-molecule activator of pyruvate kinase R (PK-R), the red cell isoform of pyruvate kinase. It enhances glycolytic pathway activity in red blood cells, increasing ATP levels and reducing 2,3-DPG levels to improve oxygen affinity and RBC deformability. It is studied in pyruvate kinase deficiency, sickle cell anemia, and myelodysplastic syndromes.
Summary written with AI from the sources listed at the end of this page.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT05490446 | Phase 2 | Myelodysplastic Syndromes | Active not recruiting | AGIO |
| NCT06745271 | Phase 1 | Healthy Participants | Completed | AGIO |
| NCT04536792 | Phase 1 | Healthy Volunteers | Completed | AGIO |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Myelodysplastic SyndromesAnemia, Sickle CellHealthy ParticipantsHealthy Volunteers
Updated Oct 3, 2026. Something wrong or missing? Report it on the AGIO page.
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