Apellis’ C3 inhibitor shows biomarker impact in small PhII rare kidney disease trial

Apel­lis Phar­ma­ceu­ti­cal’s pegc­eta­coplan has shown hints of ef­fi­ca­cy in a hand­ful of pa­tients with rare kid­ney dis­eases in a Phase II tri­al, five months af­ter the com­pound faced a clin­i­cal set­back in ALS.

Pegc­eta­coplan won US ap­proval un­der the brand name Em­paveli in 2021 for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria. The ac­tive in­gre­di­ent al­so forms the ba­sis of Apel­lis’ Syfovre, which was au­tho­rized for ge­o­graph­ic at­ro­phy in Feb­ru­ary this year.

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