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GlobeNewswire·

Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome

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  • Ultragenyx announced that the Phase 3 Aspire study for apazunersen (GTX-102) failed to achieve its primary endpoint of change from Baseline in Bayley-4 cognitive raw scores.
  • The study also missed the key secondary endpoint of net response in the Multidomain Responder Index (MDRI).
  • Clinical results showed no statistically significant differences between treated & control groups regarding Bayley Cognition raw scores or MDRI metrics.
  • The safety profile observed in Aspire remained consistent with data from Phase 1/2 trials.
  • Ultragenyx stated it will evaluate the apazunersen program to determine its future disposition following the negative results.
  • The company plans to assess operations to implement significant expense reductions while supporting its commercial business.
  • Ultragenyx CEO Emil Kakkis expressed disappointment regarding the outcome for the global patient community.
  • The company maintains focus on its commercial portfolio, including recent GENGLYCOS approval, potential UX111 approval, & product expansions.
  • Ultragenyx targets profitability in 2027 to support its pipeline & financial stability.
  • Apazunersen is an investigational antisense oligonucleotide therapy designed to reactivate the deficient UBE3A protein in Angelman syndrome.
  • Angelman syndrome affects approximately 60,000 people in commercially accessible geographies & currently has no approved therapies.