Ionis Pharmaceuticals Inc. has announced the initiation of the pivotal Phase 3 REVEAL clinical study for ION582, targeting Angelman syndrome, a rare neurodevelopmental disorder. The first patient has been dosed, marking a significant step in evaluating the efficacy and safety of ION582. The study will involve approximately 200 participants worldwide, including both children and adults with a maternal UBE3A gene deletion or mutation. Participants will be randomized to receive either ION582 or a placebo over a 52-week period, with an extension phase offering the active treatment for up to two years. The primary endpoint focuses on improvements in expressive communication, with secondary endpoints assessing overall disease severity and other functional domains. Enrollment completion is anticipated in 2026. Results from the study have not yet been presented.
Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Ionis Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20250611150485) on June 11, 2025, and is solely responsible for the information contained therein.
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