An ear­ly-stage fail high­lights on­go­ing ALS drug de­vel­op­ment chal­lenges, de­spite re­cent Bio­gen OK

A Take­da part­ner re­port­ed an ear­ly-stage ALS tri­al fail­ure Tues­day morn­ing and opt­ed to dis­con­tin­ue the pro­gram en­tire­ly, serv­ing as a re­minder that de­spite two re­cent FDA ap­provals, chal­lenges re­main in tack­ling the uni­ver­sal­ly fa­tal dis­ease.

Wave Life Sci­ences said its pro­gram, dubbed WVE-004, did not show any clin­i­cal ben­e­fit when com­pared to place­bo in a sub­set of ALS pa­tients with spe­cif­ic ge­net­ic mu­ta­tions. The com­pound ad­di­tion­al­ly failed to show a ben­e­fit for pa­tients with fron­totem­po­ral de­men­tia in a sep­a­rate ben­e­fit analy­sis.

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