Ionis announces positive topline results from study of investigational donidalorsen, and prepares submissions to FDA and EMA

Ionis announces positive topline results from Phase 3 OASIS-HAE study of investigational donidalorsen, and prepares submissions to US FDA and European Medicines Agency

Ionis Pharmaceuticals Inc announced positive topline results for the Phase 3 OASIS-HAE study of donidalorsen in people with hereditary angioedema (HAE). The trial met its primary endpoint of reduction in rate of angioedema attacks in patients treated with donidalorsen (80mg) via subcutaneous injection dosed every 4 weeks (Q4W) (p<0.001) or every 8 weeks (Q8W) (p=0.004), compared to placebo. In addition, the trial showed 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.

Based on these data, Ionis is preparing to submit a New Drug Application with the US Food and Drug Administration. 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 US, and the Orphan Drug Designation procedure in the EU is ongoing.

Kenneth Newman, MD, Senior Vice President, Head of Clinical Development at Ionis, said: “We are very pleased with the positive topline results from the Phase 3 OASIS-HAE study of donidalorsen. Based on these results and the durable efficacy and favorable safety data seen in the ongoing Phase 2 open-label extension study, we believe donidalorsen, if approved, could be an attractive new treatment option for patients with HAE, many of whom continue to experience unpredictable, painful and severe breakthrough attacks despite currently available prophylactic treatments. We are grateful to the patients, caregivers, investigators and study teams who participated in the OASIS-HAE study.”

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, which includes both the open-label extension of the Phase 3 trial and a separate cohort of patients who have transitioned to donidalorsen from another prophylactic HAE medication (switch cohort).

(Source: Ionis Pharmaceuticals)