Apel­lis turns up mixed re­sults in close­ly watched eye dis­ease tests. Are the da­ta strong enough for ap­proval?

Look­ing to scuf­fle with rare dis­ease gi­ant and now As­traZeneca sub­sidiary Alex­ion, lit­tle Apel­lis scored a break­through win ear­li­er this sum­mer for its own com­ple­ment fac­tor in­hibitor drug. Apel­lis, though, al­ways had its eyes on big­ger fish, but a first glimpse at piv­otal da­ta for a ma­jor in­di­ca­tion has mud­died the wa­ters.

In two Phase III stud­ies test­ing pegc­eta­coplan in pa­tients with ge­o­graph­ic at­ro­phy, Apel­lis’ drug nailed the pri­ma­ry end­points in one test but flopped the same check­points in a “car­bon copy” study, ac­cord­ing to topline da­ta re­leased Thurs­day.

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