Agios’ oral drug secures PhIII success in transfusion-independent thalassemia patients

Agios Phar­ma­ceu­ti­cals’ oral drug can­di­date is a step clos­er to mar­ket af­ter it suc­ceed­ed in a late-stage study in tha­lassemia pa­tients who don’t need reg­u­lar trans­fu­sions. The com­pa­ny is now gear­ing up for reg­u­la­to­ry ac­tiv­i­ties by year’s end.

The Phase III EN­ER­GIZE tri­al in­ves­ti­gat­ed the pyru­vate ki­nase ac­ti­va­tor mi­tapi­vat ver­sus place­bo in 194 sub­jects with non-trans­fu­sion-de­pen­dent al­pha- or be­ta-tha­lassemia.

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