Clinical Trial ResultsINZ-701 demonstrated improved survival with 80% of infants treated surviving beyond one year of age, compared to an approximately 50% historical rate.
Market OpportunityThere is encouragement by the market opportunity for INZ-701, given no approved therapies and prior data supporting its potential clinical benefit.
Regulatory DesignationsINZ-701 has orphan drug designation from the EMA and the FDA for the treatment of ENPP1 Deficiency, as well as FDA Fast Track designation and rare pediatric disease designation.