Positive donidalorsen late-stage clinical progress in HAE announced by Ionis

1 June 2023
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Positive clinical progress with donidalorsen, a late-stage investigational prophylactic therapy for hereditary angioedema (HAE), were announced this morning by US RNA-targeted drug developer Ionis Pharmaceuticals (Nasdaq: IONS). The news sent Ionis’ shares up a modest 1.4% to $41.47 in early trading.

Top-line 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.

Ionis also announced that it has completed enrollment in the Phase III OASIS-HAE study, which is evaluating the safety and efficacy of donidalorsen in preventing angioedema attacks for which top-line data are expected in the first half of 2024.

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