Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been sev­er­al months since Ver­tex culled one of its small mol­e­cules for al­pha-1 an­tit­rypsin de­fi­cien­cy (AATD), tak­ing a big hit af­ter ev­i­dence of liv­er dam­age sur­faced in a key Phase II tri­al. Now we learned that the com­pa­ny has whiffed on its sec­ond shot, and there’s noth­ing left in the clin­ic to treat the rare ge­net­ic dis­ease — but that won’t stop it from try­ing.

De­spite avoid­ing the safe­ty is­sues that plagued the last can­di­date, Ver­tex $VRTX is tak­ing the axe to VX-864 af­ter Phase II re­sults re­vealed the mag­ni­tude of the drug’s re­sponse is “un­like­ly to trans­late in­to sub­stan­tial clin­i­cal ben­e­fit.” As a re­sult of the news, the com­pa­ny’s stock fell 12.5% af­ter hours.

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