Business
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 attacksCompleted enrollment in the
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"Two-year treatment with donidalorsen in the Phase 2 open label study resulted in consistent, sustained protection from HAE attacksCompleted enrollment in the Phase 3 OASIS-HAE studyTopline Phase 3 results expected in H1 2024CARLSBAD, 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.\n\n \n \n \n \n \n \n\n \n\"By completing enrollment in the Phase 3 study, we are one step closer to bringing a potentially transformative and differentiated prophylactic treatment to HAE patients,\" said Richard S. Geary, Ph.D., executive vice president and chief development officer at Ionis. \"We are also encouraged by the long-term safety and durable efficacy results seen in patients treated for two years in our ongoing open-label extension study. We look forward to the Phase 3 data readout in the first half of 2024 and are advancing our go-to-market preparations to commercialize donidalorsen.\"\nThe two-year Phase 2 OLE results will be presented at an upcoming medical congress.\nIn the Phase 2 study, through week 17, donidalorsen 80 mg monthly demonstrated a 90% reduction in angioedema attacks compared with placebo after the first dose, and a 97% reduction in angioedema attacks starting with the second dose. The Phase 2 results also showed a significant improvement in quality of life as assessed by the Angioedema Quality of Life Questionnaire (AE-QoL), in the patients treated with donidalorsen. Donidalorsen continues to demonstrate a favorable safety and tolerability profile with added two-year OLE data.\nAbout OASIS-HAE\nOASIS-HAE is a multi-center, doub...