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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-2024ION582 has been generally well tolerated at all dose levels,
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"Part 1 of HALOS trial evaluating ION582 has completed enrollment with results expected in mid-2024ION582 has been generally well tolerated at all dose levels, and participants are continuing to Part 2, the long-term extension portion of the studyEncouraging EEG and clinical observations support continued developmentMIAMI, 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.\n\n \n \n \n \n \n \n\n \nAngelman syndrome is caused by a loss of function in the UBE3A gene. ION582 is designed to unsilence the paternal UBE3A allele in order to increase production of the UBE3A protein in the brain. Angelman syndrome affects an estimated one in 12,000 to 20,000 people globally1. It presents profound and severe developmental delays in motor, language and cognitive functioning, seizures and ataxia. It is a serious neuro-developmental disorder that presents in early childhood, resulting in complete dependence on a caregiver. There are no treatments specifically approved for Angelman syndrome.\n\"We are encouraged by the completion of enrollment in the HALOS trial in Angelman syndrome as well as the positive preliminary clinical findings,\" said Frank Bennett, Ph.D., executive vice president and chief scientific officer of Ionis. \"We look forward to reviewing results from the study mid-year next year, which will help us define the next stage of development for ION582. This community has an urgent need for new treatment advances given the significant neuro-developmental delays and challenges faced by people with Angelman syndrome and their caregivers. We look forward to working with the community, i...