Ionis shares positive clinical update from ongoing trial of ION582 for Angelman syndrome

Part 1 of HALOS trial evaluating ION582 has completed enrollment with results expected in mid-2024 ION582 has been generally well tolerated at all dose levels, and participants are continuing to Part 2, the long-term extension portion of the study Encouraging EEG and clinical observations support continued development MIAMI, Nov. 11, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the completion of enrollment and positive preliminary findings from Part 1 of the ongoing HALOS Phase 1/2a study of ION582 (BIIB121) in Angelman syndrome. ION582 was generally well tolerated in the study and showed encouraging electroencephalogram (EEG) activity trends and early signals of positive clinical improvement, which will need to be confirmed upon analysis of the full data set in mid-2024. The findings were presented today at the FAST (Foundation for Angelman Syndrome Therapeutics) Summit; the presentation is available here. Part 1 of the HALOS trial is a three-month, open-label, multiple-ascending dose study. Part 2 is a long-term extension study, which will evaluate ION582 for an additional 12 months.

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