Santosh L Saraf, Robert Ward Hagar, Modupe Idowu, Ifeyinwa Osunkwo, Kimberly Cruz, Frans A Kuypers, R Clark Brown, James Geib, Maria D Ribadeneira, Patricia Schroeder, Eric Wu, Sanjeev Forsyth, Patrick F Kelly, Theodosia A Kalfa, Marilyn J Telen
Etavopivat is an investigational, once-daily, oral, selective erythrocyte pyruvate kinase (PKR) activator. A multicenter, randomized, placebo-controlled, double-blind, 3-part, phase 1 study (https://clinicaltrials.gov/study/NCT03815695) was conducted to characterize the safety and clinical activity of etavopivat. Thirty-six patients with sickle cell disease (SCD) were enrolled into 4 cohorts: one single-dose; two multiple ascending doses; one open-label [OL]. In the OL cohort, 15 patients (median age 33.0 [range, 17‒55] years received 400-mg etavopivat once daily for 12 weeks; 14 completed treatment...
April 19, 2024: Blood Advances