science
Mitapivat Cuts Transfusions but Misses Main Pain-Crisis Goal in Sickle-Cell Trial
Agios Pharmaceuticals presented new Phase 3 data showing mitapivat reduced transfusion burden and raised hemoglobin in sickle cell disease patients. The results were unveiled at the European Hematology Association Congress in Stockholm.
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Agios Pharmaceuticals presented detailed results from the 52-week double-blind period of its global RISE UP Phase 3 trial of mitapivat in patients aged 16 years and older with sickle cell disease on June 13, 2026. The data were delivered during the Plenary Abstracts Session at the 31st European Hematology Association Congress in Stockholm, Sweden. 0001.
9 percent on placebo. 6 g/dL. 9 percent relative drop in average red blood cell units transfused per patient.
The transfusion reductions held whether or not patients were taking hydroxyurea. Hemoglobin responders experienced a 26 percent lower annualized rate of sickle cell pain crises and 34 percent fewer related hospitalizations than non-responders. They also showed a 53 percent reduction in overall healthcare utilization.
The trial met two key secondary endpoints for rapid and durable gains in hemoglobin and indirect bilirubin. The primary endpoint for annualized sickle cell pain crises did not reach statistical significance, and no overall difference appeared on patient-reported fatigue between the mitapivat and placebo groups.
Mitapivat was well tolerated, with a safety profile matching earlier studies in sickle cell disease.
M. ET on June 13. Topline results from RISE UP were first announced in November 2025. The new transfusion burden and hemoglobin responder analyses had not been previously disclosed.
