Ionis announces positive donidalorsen late-stage clinical progress in HAE

Two-year treatment with donidalorsen in the Phase 2 open label study resulted in consistent, sustained protection from HAE attacks Completed enrollment in the Phase 3 OASIS-HAE study Topline Phase 3 results expected in H1 2024 CARLSBAD, Calif., June 1, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive clinical progress with donidalorsen, its late-stage investigational prophylactic therapy for hereditary angioedema (HAE). Topline two-year open-label extension (OLE) results continue to demonstrate consistent efficacy and safety, with an overall sustained mean reduction in HAE attack rates of 96% from baseline through two years across dosing groups. The company also announced that it has completed enrollment in the Phase 3 OASIS-HAE study, which is evaluating the safety and efficacy of donidalorsen in preventing angioedema attacks. Topline data from the study are expected in the first half of 2024. HAE is a rare and potentially fatal genetic disease characterized by severe and potentially fatal swelling of the arms, legs, face and throat1,2.

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