BioCryst shares are blast­ed as a PhI­II HAE 'suc­cess' un­der­whelms an­a­lysts

It was de­signed to be the oral fix for hered­i­tary an­gioede­ma (HAE) — a rare, po­ten­tial­ly life-threat­en­ing ge­net­ic dis­or­der — but BioCryst Phar­ma­ceu­ti­cal’s ex­per­i­men­tal cap­sule has un­der­whelmed in a piv­otal late-stage study, de­spite meet­ing the pri­ma­ry end­point.

Two dos­es of the oral drug, BCX7353, (110 mg and 150 mg) were in­ves­ti­gat­ed in a 121-pa­tient, place­bo-con­trolled tri­al called APeX-2 for the pre­ven­tion of an­gioede­ma at­tacks — re­cur­rent episodes of se­vere swelling com­mon­ly in the limbs, face, in­testi­nal tract, and air­way.

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