Ionis reports ‘positive’ two-year results from Phase 2 OLE study of donidalorsen

Ionis Pharmaceuticals announced “positive” results from an ongoing Phase 2 open-label extension, or OLE, study evaluating the safety and efficacy of its investigational prophylactic medicine, donidalorsen, in patients with hereditary angioedema, or HAE. 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 over two years. Treatment with donidalorsen was well tolerated in the studies, and there were no serious adverse events, the company reported.