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Ann: Retina Target Engagement Confirmed in Large Eye, page-84

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    And yet another blow for an Ionis CNS antisense drug candidate….

    Following the announcement last October of Phase 3 failure of tofersen in adults with ALS associated with a SOD1 mutation, Biogen and Ionis have suffered another disappointment in the ALS pipeline after BIIB078 (IONIS-C9Rx) failed to demonstrate clinical benefit in a 106 patient Phase 1 trial. BIIB078 is an investigational antisense therapy for the treatment of C9orf72-associated ALS.

    No consistent difference was seen between the 60mg cohort and placebo and the 90mg cohort showed a greater decline on secondary endpoints than placebo.
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