Home > Haematology > EHA 2020 > Benign Haematology > Mitapivat, a pyruvate kinase-R activator, in SCD is safe with early efficacy results

Mitapivat, a pyruvate kinase-R activator, in SCD is safe with early efficacy results

Presented By
Dr Kevin HM Kuo, Princess Margaret Cancer Centre, Canada
Conference
EHA 2020
Trial
Phase 1
Early phase 1 trial data show that mitapivat safely increased the amount of haemoglobin and decreased biomarkers of erythrocyte damage in adults with sickle cell disease (SCD). In this phase 1 proof-of-concept study, 25 patients will ultimately be enrolled, presented Dr Kevin HM Kuo (Princess Margaret Cancer Centre, Canada) [1]. The rationale of the study is based on the fact that mitapivat is an oral, small molecule activator of pyruvate kinase-R (PKR), an enzyme which consumes 2,3-diphosphoglycerate (2,3-DPG) in glycolysis. Accumulation of 2,3-DPG in red blood cells is thought to promote haemoglobin polymerisation, and thus the hypothesis is that mitapivat will mitigate that process. Patients receive increasing doses of mitapivat in this study — 5, 20, 50, and 100 mg twice daily — each lasting 2 weeks, followed by a treatment tapering period. To date, 9 pa...


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