Ionis Pharmaceuticals To Expedite Development Of Rare Neurological Disease Candidate With FDA Breakthrough Status

Core Insights - The U.S. FDA granted Breakthrough Therapy designation to Ionis Pharmaceuticals' IONS ION582 for Angelman syndrome, indicating significant clinical improvements in communication, cognition, and motor function from the Phase 1/2 HALOS study [1][2] - The Breakthrough Therapy designation is intended to expedite the review process for serious conditions, with ION582 showing potential for substantial improvement over existing therapies [2] - Ionis has initiated the global Phase 3 REVEAL study, which aims to enroll children and adults with Angelman syndrome by 2026 [2] Company Performance - William Blair noted the Breakthrough Therapy designation as a significant achievement for Ionis's neurology division, with positive HALOS data impressing regulators [3] - ION582 is now in competition with Ultragenyx Pharmaceutical's apazunersen (GTX-102), which has completed recruitment for the Aspire study [3] - Following the news, IONS stock increased by 4.68%, reaching $63.86 [3]