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# Ionis Upbeat on Angelman Drug Study
- URL: https://www.fdaweb.com/ionis-upbeat-on-angelman-drug-study/
- Published: 2024-07-22T12:00:00.000Z
- Updated: 2026-09-14T14:36:03.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5157400

Ionis Pharmaceuticals says data from its Phase 1/2 open-label study of ION582 in people with Angelman syndrome demonstrated consistent and encouraging clinical improvement on measures assessing all “functional domains” including communication, cognition and motor function. “Overall, 97% of people in the medium and high dose groups saw an improvement in overall AS symptoms as measured by the Symptoms of Angelman Syndrome–Clinician Global Impression-Change,” it says. The therapy also showed favorable safety and tolerability at all study dose levels. Based on the data, the company is planning to begin a Phase 3 study in the first half of next year.

Angelman syndrome is described as a serious, rare neurodevelopmental disorder that is caused by a loss of function in the maternal UBE3A gene. It affects about one in 21,000 people worldwide and presents in early childhood as severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. ION582 is an investigational antisense medicine designed to “unsilence” the normal paternal UBE3A gene to increase UBE3A protein production in the brain, the company says.