Can­cer drug­mak­er Agios of­fers promis­ing da­ta from two stud­ies test­ing blood dis­or­der drug

With two can­cer drugs on the mar­ket, the spot­light is on Agios’ rare meta­bol­ic dis­ease fran­chise led by mi­tapi­vat, its ex­per­i­men­tal drug un­der de­vel­op­ment for a range of con­di­tions in­clud­ing pyru­vate ki­nase de­fi­cien­cy, tha­lassemia, and sick­le cell dis­ease.

At the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion An­nu­al Con­gress (EHA), the com­pa­ny is­sued two up­dates on the ef­fect of mi­tapi­vat — a drug de­signed to tar­get pyru­vate ki­nase-R (PKR), an en­zyme in­volved in the con­ver­sion of sug­ar, or glu­cose, in­to en­er­gy that is crit­i­cal for the sur­vival of red blood cells.

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