Ionis Pharmaceuticals’ donidalorsen meets primary endpoint in Phase 3 HAE trial

Ionis Pharmaceuticals announced positive topline results for the Phase 3 OASIS-HAE study of donidalorsen in people with hereditary angioedema, or HAE. The trial met its primary endpoint of reduction in rate of angioedema attacks in patients treated with donidalorsen via subcutaneous injection dosed every 4 or 8 weeks vs. placebo. In addition, donidalorsen achieved statistical significance on all secondary endpoints in the Q4W group and key secondary endpoints in the Q8W group. Donidalorsen demonstrated a favorable safety and tolerability profile in the study, and there were no serious adverse events in the patients treated with donidalorsen. HAE is a rare and life-threatening genetic disease that causes unpredictable and frequent severe swelling of the skin, gastrointestinal tract, upper respiratory system, face and throat. Based on these data, Ionis is preparing to submit a New Drug Application with the FDA. Otsuka, which has exclusive rights to commercialize donidalorsen in Europe, is preparing to submit a Marketing Authorization Application to the European Medicines Agency. Donidalorsen received Orphan Drug Designation in the U.S., and the Orphan Drug Designation procedure in the EU is ongoing. Ionis plans to present the Phase 3 OASIS-HAE results at an upcoming medical congress by mid-year. Ionis also plans to share results from the Phase 3 OASIS-Plus study by mid-year.