Rac­ing against Al­ny­lam, Io­n­is posts pos­i­tive PhI­II AT­TR ef­fi­ca­cy num­bers, but ques­tions linger

One of the big ri­val­ries in biotech right now is the race be­tween Io­n­is $IONS and Al­ny­lam $AL­NY to field a new RNA ther­a­py for hered­i­tary TTR amy­loi­do­sis (hAT­TR) with polyneu­ropa­thy. And af­ter Al­ny­lam drew cheers for its top-line win, Io­n­is is rolling out a big peek at the bot­tom line.

Re­searchers post­ed a mean 19.73-point ben­e­fit for the neu­ropa­thy im­pair­ment score, its mNIS+7 co-pri­ma­ry end­point af­ter 15 months of treat­ment, say­ing that they could start track­ing a sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit at 8 months.

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