Ionis Pharmaceuticals’ ION582 shows improvements in Angelman syndrome

Ionis Pharmaceuticals 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. At six months, approximately 65% of patients achieved an improvement in cognition on the Bayley-4 and showed improvements in Bayley-4 measures of fine and gross motor skills. ION582 was safe and well tolerated at all dose levels. Ionis will independently advance ION582 as part of Ionis’ 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.