Their lead drug failed both main goals in key study, but Or­p­hazyme plans to pitch sub­group analy­ses to reg­u­la­tors

Or­p­hazyme at­tempt­ed on Wednes­day to en­thuse in­vestors about the re­sults of a Phase II/III study test­ing their lead drug, ari­mo­clo­mol, in pa­tients with a rare ge­net­ic dis­or­der, un­der­scor­ing some pos­i­tive sub­group analy­ses — even though the ex­per­i­men­tal treat­ment missed its pri­ma­ry end­points. The com­pa­ny is now prepar­ing mar­ket­ing fil­ings and plan­ning to en­gage with reg­u­la­tors to get ari­mo­clo­mol on the mar­ket at the ear­li­est.

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