Ionis Pharmaceuticals (NASDAQ:IONS) on Monday announced positive topline Phase 3 trial results for its donidalorsen drug candidate for hereditary angioedema (HAE).
The Phase 3 OASIS-HAE study found donidalorsen met the primary endpoint of statistically significant reduction in HAE attacks in patients.
The drug candidate also achieved statistical significance on multiple secondary endpoints and demonstrated a favorable safety and tolerability profile in the study. Donidalorsen is an RNA-targeted prophylactic medicine designed to interupt the pathway that leads to HAE attacks.
Based on these data, Ionis is preparing to submit a new drug application with the U.S. FDA. Otsuka, which has exclusive rights to commercialize donidalorsen in Europe, is preparing to submit a marketing authorization application as well.
Donidalorsen holds orphan drug designation in the U.S., and the orphan drug designation procedure in the EU is ongoing.
Ionis (IONS) is up around 2% ahead of market open.