Ionis Pharmaceuticals (NASDAQ:IONS) reported 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 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 throat.
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.
