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Getting the latest healthcare news for you
Getting the latest healthcare news for you
A promising drug for Angelman syndrome just hit a major wall. Ultragenyx's apazunersen failed to show meaningful improvement in cognitive and developmental outcomes in a late-stage trial, sending shares tumbling 45% after hours. The rare genetic disorder, which affects roughly 1 in 15,000 children, still has no approved disease-modifying treatment.
A promising drug for Angelman syndrome just hit a major wall. Ultragenyx Pharmaceutical's experimental drug apazunersen failed its primary endpoint in a late-stage clinical trial, showing no meaningful improvement in cognitive and nonverbal reasoning abilities or across broader developmental measures in young children with Angelman syndrome. The company said there were no differences between treated and control groups to support efficacy, and it will now evaluate the future of the apazunersen program.
The setback also casts a shadow over Ultragenyx's related Aurora study, which is testing apazunersen in Angelman syndrome patients with different genetic subtypes. Analysts at William Blair noted that even if Aurora hits its primary endpoint, commercialization would be an uphill battle given the small patient population.
By the Numbers:
Why it matters: Angelman syndrome currently has no approved disease-modifying treatments, leaving families with only supportive care options. This trial failure is a significant blow to one of the most advanced therapeutic programs targeting the condition, underscoring just how difficult it remains to develop effective treatments for rare neurodevelopmental disorders.
