Small molecule · Activator of pyruvate kinase, red cell isoform (PK-R) (PKLR) · developed by Agios Pharms
Mitapivat is a small-molecule allosteric activator of pyruvate kinase (PK-R) that binds to the PK tetramer and increases its activity. It enhances glycolytic pathway activity, improves ATP levels, and reduces 2,3-DPG levels in red blood cells.
Summary written with AI from the sources listed at the end of this page.
From the indications section of the FDA label (version of Jun 30, 2026). Read the full label for the exact wording, limitations and safety information.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT05777993 | Phase 4 | Pyruvate Kinase Deficiency | Enrolling by invitation | AGIO |
| NCT03548220 | Phase 3 | Pyruvate Kinase Deficiency | Completed | AGIO |
| NCT04696393 | Phase 1 | Healthy Volunteers | Completed | AGIO |
| NCT04565678 | Phase 1 | Healthy Volunteers | Completed | AGIO |
| NCT04472832 | Phase 1 | Healthy Volunteers | Completed | AGIO |
| NCT03991312 | Phase 1 | Healthy Volunteers | Completed | AGIO |
| NCT03703505 | Phase 1 | Healthy Volunteers | Completed | AGIO |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Sickle Cell DiseaseThalassemiaAnemia, HemolyticHealthy VolunteersPyruvate Kinase Deficiency
Updated Oct 3, 2026. Something wrong or missing? Report it on the AGIO page.
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