As­traZeneca, Io­n­is un­veil full PhI­II da­ta for 'mega brand' AT­TR drug

As­traZeneca and Io­n­is fleshed out a Phase III win for eplon­ter­sen Mon­day morn­ing at this year’s Amer­i­can Acad­e­my of Neu­rol­o­gy meet­ing, il­lus­trat­ing why the pair is so con­fi­dent in its po­ten­tial suc­cess.

Eplon­ter­sen all but halt­ed pa­tients’ dis­ease pro­gres­sion af­ter 66 weeks in a fa­tal neu­rode­gen­er­a­tive dis­ease known as hered­i­tary transthyretin-me­di­at­ed amy­loid polyneu­ropa­thy (AT­TRv-PN). In a com­pos­ite score of polyneu­ropa­thy clin­i­cal mea­sure­ments like mus­cle strength and blood pres­sure, pa­tients tak­ing eplon­ter­sen record­ed a 0.28-point av­er­age in­crease from base­line com­pared to a 25.06-point av­er­age in­crease in the place­bo group.

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