Ionis Pharmaceuticals (NASDAQ:IONS) on Monday announced positive results from the Phase 3 trial of its drug candidate donidalorsen for hereditary angioedema (HAE).
The phase 3 OASIS-HAE study found that donidalorsen met the primary endpoint of statistically significant reduction in HAE attacks in patients.
The drug candidate also achieved statistical significance across multiple secondary endpoints and demonstrated a favorable safety and tolerability profile in the study. Donidalorsen is an RNA-targeted prophylactic medication designed to disrupt the pathway that leads to HAE attacks.
Based on this data, Ionis is preparing to submit a new drug application to the US FDA. Otsuka, which has exclusive rights to market donidalorsen in Europe, is also preparing to submit a marketing authorization application.
Donidalorsen has orphan drug designation in the US and the orphan drug designation procedure in the EU is ongoing.
Ionis (IONS) is on the rise 2% before the market opens.
