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

Ionis Pharmaceuticals announced the completion of enrollment and preliminary findings from Part 1 of the ongoing HALOS Phase 1/2a study of ION582 in Angelman syndrome. ION582 was generally well tolerated in the study and showed encouraging electroencephalogram 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 at the FAST, Foundation for Angelman Syndrome Therapeutics, Summit. 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.