Ionis Pharmaceuticals has announced the design for a pivotal Phase III trial of ION582, an investigational treatment for AS, following discussions with the US Food and Drug Administration (FDA).
The Phase III randomised, global, placebo-controlled REVEAL study will enrol roughly 200 adults and children with AS, specifically those with a maternal ubiquitin-protein ligase E3A (UBE3A) gene mutation or deletion.
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The trial’s primary analysis is set to occur after one year of treatment and participants will then be moved to an open-label, long-term extension phase.
Patients in the trial will be randomised into a 2:1 ratio to receive either the active therapy or a placebo. ION582 will be assessed at two different dose levels every quarter without the need for a loading regimen.
The primary endpoint of the study is to measure improvements in expressive communication using the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), a clinical assessment tool.
The trial will also assess several secondary endpoints such as sleep, overall disease severity, communication, motor functioning and everyday living skills, along with other exploratory endpoints.
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By GlobalDataIonis Pharmaceuticals CEO Brett Monia said: “We will enrol a broad group of individuals living with AS in the global pivotal Phase III trial, planned to begin in the first half of 2025.
“We look forward to working with the community to advance a potential new treatment targeting the underlying cause of disease in this debilitating neurological condition that has no approved medicines.”
The data from the Phase II HALOS study supported the Phase II meeting. In the study’s multiple ascending dose (MAD) part, ION582 demonstrated clinically meaningful improvements across various functional domains.
Notably, 97% of participants in the medium and high dose groups exhibited improvements in overall AS symptoms as per the Angelman Syndrome Clinical Global Improvement Change (SAS-CGI-C) scale.
The safety and tolerability of ION582 were also favourable across all doses tested.
In 2022, the therapy received orphan drug designation (ODD) and rare paediatric disease designation from the FDA.
The REVEAL trial aims to address the issues of the patients living with this genetic disorder.
