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This study, published in the Journal of Pharmacology and Experimental Therapeutics, evaluates the response of both non-human primates and healthy human subjects to etavopivat. Etavopivat is a small molecule activator of the glycolytic enzyme erythrocyte pyruvate kinase. It is being developed to treat sickle cell disease (SCD) and is hypothesized to work via multiple mechanisms that increase hemoglobin-oxygen affinity and preserve red blood cell membrane integrity and deformability. 

A single dose of etavopivat was shown to decrease 2,3-diphosphoglycerate (2,3-DPG) in both primates and healthy human subjects and to significantly increase hemoglobin-oxygen affinity in the human subjects after 24 hours. Moreover, daily dosing for five days resulted in an increase in hemoglobin-oxygen affinity and a decrease in red blood cell sickling among ex vivo red blood cells of patients with SCD (HbSS or HbSC genotype).

The researchers concluded that etavopivat shows promise as a treatment option for SCD and could reduce vaso-occlusion while improving anemia [1].


[1] Schroeder, P., Fulzele, K., Forsyth, S., Ribadeneira, M. D., Guichard, S., Wilker, E., Marshall, C. G., Drake, A., Fessler, R., Konstantinidis, D. G., Seu, K. G., & Kalfa, T. A. (2022). Etavopivat, a pyruvate kinase activator in red blood cells, for the treatment of sickle cell disease. Journal of Pharmacology and Experimental Therapeutics, JPET-AR. https://doi.org/10.1124/jpet.121.000743

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