Ionis presents positive two-year results from the Phase 2 open label extension study of donidalorsen in patients with hereditary angioedema

Continued treatment over two years with donidalorsen demonstrated sustained reduction in hereditary angioedema attacks and improved quality of life Donidalorsen recently received orphan drug designation in the U.S. Topline Phase 3 results expected in H1 2024 CARLSBAD, Calif., Nov. 9, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive results from an ongoing Phase 2 open-label extension (OLE) study evaluating the safety and efficacy of its investigational prophylactic medicine, donidalorsen, in patients with hereditary angioedema (HAE), a rare and life-threatening genetic disease. Over the two years, patients treated with donidalorsen via subcutaneous injection showed an overall sustained mean reduction in HAE attack rates of 96% from baseline, from 2.70 to 0.06 attacks per month, across all dosing groups. Furthermore, all patients treated with donidalorsen reported a clinically meaningful improvement in quality of life as measured by the Angioedema Quality of Life Questionnaire (AE-QoL) over two years. Treatment with donidalorsen was well tolerated in the studies, and there were no serious adverse events.

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