CMO Carmen Bozic (Vertex)

Ver­tex shares take a hit as liv­er tox forces the re­search team to scrap a PhII drug for AAT pa­tients. Will we see new deals now?

Ver­tex is tak­ing the ax to one of their rare dis­ease drugs af­ter ev­i­dence of liv­er dam­age emerged in a key Phase II study that blocks any us­es of the high­er dos­es that would be need­ed to treat ge­net­ic cas­es of al­pha-1 an­tit­rypsin de­fi­cien­cy. And with that pro­gram down and out, one top an­a­lyst says that the big biotech has no choice but to start lin­ing up deals. Now.

The big biotech had high­light­ed a sim­i­lar ap­proach to mid-stage work that got them through ma­jor R&D work in cys­tic fi­bro­sis, which cre­at­ed the foun­da­tion the com­pa­ny is built on. But in the tri­al of some 50 pa­tients, 4 tak­ing dif­fer­ent dos­es of the drug demon­strat­ed el­e­vat­ed liv­er en­zymes “greater than 8 times the up­per lim­it of nor­mal.”

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