Treatment for Angelman syndrome using ION582 brings significant relief: Study results reveal
In a significant breakthrough for the treatment of Angelman syndrome, Ionis Pharmaceuticals' investigational drug, ION582, has demonstrated encouraging results in a recent clinical trial.
The open-label HALOS clinical trial, which enrolled 51 patients aged 2 to 50, tested three doses of ION582 - low, medium, and high - for three months, injected directly into the spinal canal. The trial's main goal was to assess the safety and tolerability of multiple ascending doses of ION582.
The results, announced by Ionis Pharmaceuticals' CEO, Brett Monia, position ION582 as a potential cornerstone of Ionis' next wave of transformational, wholly owned medicines for neurological conditions.
Overall and clinically meaningful improvements were observed in 97% of patients given medium and high doses of ION582, as assessed by the Symptoms of Angelman Syndrome-Clinician Global Impression of Change (SAS-CGI-C) scale. When measured using the same scale, cognition improved in 85% of patients, and sleep improved in 61%.
Fine motor skills improved in 72% of the patients, cognition in 67%, and expressive communication in 69%. Socialization skills were improved in 63% to 87% of the patients, and daily living skills in 74% to 82%. Receptive communication skills in the Vineland-3 improved in 89% of participants, and expressive communication in 84%.
Gross motor skills improved in 53%, and behaviour improved in 56%. Improvements in communication, cognition, and motor skills were also reported for patients receiving medium and high doses of ION582.
Angelman syndrome is caused by mutations in the maternal copy of the UBE3A gene. ION582 was designed to turn on the paternal copy of the UBE3A gene by blocking a molecule, Ubiquitin Protein Ligase E3A-Antisense Transcript, that silences it.
Exploratory efficacy parameters include changes in patients' communication, cognition, motor function, sleep, seizures, and daily living skills. The Phase 1/2 study (NCT05127226) has now concluded, and eligible patients will now transition to the second part of the study, which will evaluate the effects of the medium and high doses of ION582 for an additional 12 months.
Ionis Pharmaceuticals expects to initiate Phase 3 studies for their investigational Angelman syndrome treatment ION582 in the first half of 2025. The company was granted orphan drug and rare pediatric disease status by the U.S. Food and Drug Administration (FDA) for ION582.
The promising results from the HALOS trial offer hope for families affected by Angelman syndrome, a rare genetic disorder that causes severe intellectual disability, speech impairment, and seizures. With ION582, Ionis Pharmaceuticals is one step closer to providing a much-needed treatment for this debilitating condition.
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