From Yahoo Finance: 2025-06-23 01:37:00

In a pivotal Phase 3 REVEAL trial, Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) doses first participant with ION582 for Angelman syndrome, a severe neurodevelopmental disease. Trial aims to improve expressive communication in 200 individuals with UBE3A gene deletions or mutations, with quarterly dosages evaluated over 52 weeks.

ION582, an antisense RNA treatment by Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), inhibits UBE3A-ATS to promote UBE3A protein. FDA designates it as Rare Pediatric Disease, Fast Track, and Orphan Drug. Angelman syndrome affects 1 in 21,000 individuals globally, leading to severe physical and cognitive impairments.

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), known for SPINRAZA and QALSODY, expands neurology portfolio with eight treatments in development for rare and common disorders, including Alzheimer’s and Parkinson’s disease.

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Read more: First Patient Dosed In Phase 3 REVEAL Study Of ION582 For Angelman Syndrome