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Agios announces phase III ACTIVATE-T trial of mitapivat achieved primary endpoint for pyruvate kinase deficiency patients who are regularly transfused.

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Published:28th Jan 2021
Agios Pharmaceuticals, Inc. announced that the global, open-label Phase III ACTIVATE-T trial of mitapivat in regularly transfused adults with PK deficiency demonstrated a statistically significant and clinically meaningful reduction in transfusion burden . All 27 patients enrolled in the study were treated with mitapivat. In the 24-week fixed dose period, 37 percent (n = 10) achieved a greater than 33% reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks (1-sided p=0.0002). In addition, 22 percent (n = 6) were transfusion-free during the 24-week fixed dose period. Mitapivat is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes. Agios recently reported that its global, randomized, double-blind, placebo-controlled Phase III ACTIVATE trial of mitapivat in adults with PK deficiency who do not receive regular transfusions met its primary endpoint , with 40 percent of patients randomized to mitapivat achieving a hemoglobin response, defined as a greater than 1.5 g/dL sustained increase in hemoglobin concentration from baseline, compared to 0 patients randomized to placebo (2-sided p<0.0001). agios anticipates filing for regulatory approval based on data from activate and activate-t in the u.s. in q2 2021 and in the eu in mid-2021, with a potential 2022 commercial launch in both geographies. results from the activate-t trial were as follows : i. 37 percent of patients dosed with mitapivat (n="10" of 27) achieved a greater than 33% reduction in transfusion burden in the 24-week fixed dose period compared with individual historical transfusion burden standardized to 24 weeks (1-sided p="0.0002)" .ii. 22 percent of patients dosed with mitapivat (n="6" of 27) were transfusion-free during the 24-week fixed dose period. iii.treatment with mitapivat showed a reduction in the annualized total number of red blood cell units transfused during the study compared with the historical transfusion burden. iv.the safety profile observed in the study was consistent with previously reported. agios is conducting a full analysis of the activate-t results and expects to submit the complete results of the trial for presentation at the european hematology association (eha) virtual congress, which is being held june 9-17, 2021.>
Condition: Pyruvate Kinase Deficiency
Type: drug

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