Ver­tex dis­con­tin­ues a PhII rare dis­ease drug, sets ear­ly 2024 time­line for piv­otal non-opi­oid pain pill read­out

Side ef­fects have led Ver­tex Phar­ma­ceu­ti­cals to stop work on an­oth­er Phase II drug can­di­date to treat a rare ge­net­ic dis­ease called al­pha-1 an­tit­rypsin de­fien­cy, or AATD, the biotech dis­closed Mon­day in its third-quar­ter earn­ings re­port.

Ver­tex is dis­con­tin­u­ing re­search on the small mol­e­cule called VX-864 be­cause of “non-se­ri­ous rash events in some pa­tients,” ac­cord­ing to a press re­lease. About three years ago, Ver­tex aban­doned an­oth­er AATD pro­gram, called VX-814, that made it to Phase II af­ter see­ing signs of liv­er dam­age. Ver­tex’s stock price moved down about 2% to $379 af­ter mar­kets closed Mon­day.

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