Apel­lis re­ports PhII ALS fail af­ter stop­ping open-la­bel ex­ten­sion in April

Apel­lis and So­bi will dis­con­tin­ue de­vel­op­ment in ALS af­ter their ap­proved rare blood and eye dis­ease drug failed a Phase II study.

The pair was try­ing to see if pegc­eta­coplan, green­light­ed in two dif­fer­ent in­di­ca­tions since 2021, could al­so help pa­tients with the fa­tal neu­rode­gen­er­a­tive dis­ease. But pegc­eta­coplan missed its pri­ma­ry end­point of the “Com­bined As­sess­ment of Func­tion and Sur­vival” rank score af­ter a year, and al­so missed sev­er­al sec­ondary goals.

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