IONS - Rare Disease Focused Ionis Pharmaceuticals Gears Up for FDA Submission After Donidalorsen Outperforms Placebo In Swollen Vessels Study | Benzinga
Monday, Ionis Pharmaceuticals Inc (NASDAQ:IONS) announced 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 the 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.
Ionis is preparing to submit a New Drug Application to the FDA. Otsuka, ...