Ionis Pharmaceuticals (NASDAQ:IONS), 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.
"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
The two-year Phase 2 OLE results will be presented at an upcoming medical congress.
In 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.