Ionis receives U.S. FDA Breakthrough Therapy designation for ION582 in Angelman syndrome

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[{"type":"text","content":"\n- Pivotal Phase 3 REVEAL study enrollment on track to complete in 2026 -\n\n\n CARLSBAD, Calif.--(BUSINESS WIRE)--\nIonis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ION582 for the treatment of Angelman syndrome (AS), a rare neurological disease that often presents in infancy and is characterized by profound intellectual disability, impaired communication, motor impairment and debilitating seizures.\n\n\n“With no approved disease-modifying treatments available for people living with Angelman syndrome, receiving this Breakthrough Therapy designation for ION582 underscores both the severity of this disease and the significant unmet need for treatment,” said Holly Kordasiewicz, Ph.D., senior vice president of neurology, Ionis. “This recognition also highlights the potential of ION582 to deliver meaningful benefits for individuals with Angelman syndrome. We are deeply grateful to the Angelman syndrome community and investigators who have made this progress possible, and we remain committed to advancing ION582 as quickly as possible to bring this potential treatment to those in need.”\n\n\nThis Breakthrough Therapy designation is based on results from the Phase 1/2 HALOS study of ION582 that showed consistent and encouraging clinical improvement on all AS functional domains including communication, cognition and motor function, as well as favorable safety and tolerability. Breakthrough Therapy designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown preliminary clinical evidence indicating the potential for substantial improvement over available therapies.\n\n\nIonis initiated the global Phase 3 REVEAL study (NCT06914609) of ION582 earlier this year, which is anticipated to enroll children and adults with AS that have a maternal UBE3A gene deletion or mutation.\n\n\nAbout ION582\n\n\nION582 is an investigational RNA-targeted antisense medicine designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of UBE3A protein, for the potential treatment of Angelman syndrome (AS). The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) granted Orphan Drug designation to ION582. Additionally, the FDA...

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