Agios Pharmaceuticals Provides Update on Phase 2 Study of Mitapivant in Non-Transfusion-Dependent Thalassemia

June 12, 2020 – Agios Pharmaceuticals has provided an update on the preliminary findings from their Phase 2 study of mitapivat in non-transfusion-dependent thalassemia at the European Hematology Association (EHA) virtual medical congress. Mitapivat activates the pyruvate kinase-R enzyme, which improves red blood cell energy metabolism. Mitapivat was shown to improve ineffective erythropoiesis and anemia in a mouse model of thalassemia.

Mitapivat is currently undergoing proof-of-concept testing in patients with non-transfusion-dependent α– and β-thalassemia. Preliminary findings from the Phase 2 study show that oral mitapivat treatment resulted in a ≥1.0 g/dL increase in Hemoglobin (Hgb) concentration in all 4 (100%) α-thalassemia patients and 8 of 9 (88.9%) patients with β-thalassemia who completed 12 weeks of treatment. Seven of 8 patients with β-thalassemia who completed 24 weeks of mitapivat treatment met the criteria for sustained response. The mean change in Hgb levels for α-thalassemia patients was 1.17 g/dL over weeks 4-12, and 1.43 g/dL for β-thalassemia patients over weeks 4-24. Preliminary results for markers of hemolysis and erythropoiesis demonstrated improvements that were consistent with the Hgb increase. Mitapivat was well tolerated with no serious adverse events and no adverse events leading to treatment discontinuation.

Mitapivat has been granted Orphan Drug Designation for the treatment of thalassemia by the U.S. Food and Drug Administration (FDA). Orphan drug designation provides certain benefits, including market exclusivity if/when the drug receives regulatory approval.

The Phase 2 study of mitapivat in non-transfusion-dependent alpha- and beta-thalassemia has been fully enrolled. Further information about the preliminary Phase 2 findings can be found at:

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