Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome

ION582 demonstrated consistent improvements across multiple functional domains in Angelman syndrome patients ION582 was safe and well tolerated at all dose levels Ionis plans to move ION582 into pivotal trial Detailed ION582 data to be presented at upcoming medical meeting CARLSBAD, Calif., May 16, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline data from the HALOS Phase 1/2a open-label study of ION582 in Angelman syndrome. ION582 was safe and well tolerated in the study and showed encouraging and consistent benefits in individuals living with Angelman syndrome, with the most robust improvements observed in key areas of functioning including cognition, communication and motor function.

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