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

May 16, 2024 at 7:00 AM EDT

• 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

Excerpt from the Press Release:

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.

Ionis also announced that it will independently advance ION582 as part of Ionis’ leading portfolio of potentially transformational medicines for serious neurological diseases. Ionis plans to review the ION582 Phase 1/2a results with regulatory authorities to align on the design of the pivotal program. Biogen has elected not to exercise its option to license ION582.

“There are currently no approved treatments for Angelman syndrome, which causes developmental delays, cognitive impairment and severe communications challenges, with most individuals unable to speak or live independently,” said Lynne Bird, M.D., professor of clinical pediatrics at UC San Diego and HALOS study investigator. “We are encouraged by the positive and consistent improvements seen in the HALOS study across multiple measures, as these functional improvements could have a transformative impact in the lives of people living with Angelman syndrome and their caregivers.

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