ION582 showed robust and consistent benefit in communication, cognition and motor function in a broad patient population evaluated with a comprehensive set of assessment tools that collect input from parents and clinicians 97% of patients in the medium and high dose groups saw improvement in overall Angelman syndrome symptoms as measured by the SAS-CGI-C Improvements on the Bayley-4 in cognition, communication and motor function exceeded those observed in natural history studies Ionis plans to initiate Phase 3 development in H1 2025 Ionis to host webcast on Monday, July 22 at 8:00am ET CARLSBAD, Calif. , July 22, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS ) today announced positive results from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS) demonstrating 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 (SAS-CGI-C). ION582 showed favorable safety and tolerability at all dose levels in the study. Detailed results will be presented in a company webcast today and at the 2024 Angelman Syndrome Foundation (ASF) Family Conference in Sandusky, Ohio on July 24, 2024 .
"Ionis looks forward to collabor.