Agios re­turns pos­i­tive PhI­II da­ta in a rare blood dis­or­der as it seeks to move past re­cent can­cer set­backs

About a month and a half af­ter Eu­ro­pean reg­u­la­tors spurned its AML drug Tib­so­vo, Agios is look­ing to turn the cor­ner with topline Phase III da­ta in a rare blood dis­or­der.

The biotech an­nounced Tues­day morn­ing that its ex­per­i­men­tal drug mi­tapi­vat hit the pri­ma­ry end­point in adults with pyru­vate ki­nase de­fi­cien­cy who do not re­ceive reg­u­lar blood trans­fu­sions. Mi­tapi­vat demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant and sus­tained in­crease in he­mo­glo­bin com­pared to place­bo, putting Agios on track to ap­ply for reg­u­la­to­ry ap­proval in both the US and Eu­rope some­time next year.

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