Ultragenyx's Angelman Syndrome Therapy Fails to Show Benefit in Phase 3 Trial
Ultragenyx announced that its experimental drug GTX-102 did not outperform a sham control in a large Phase 3 study for Angelman syndrome.
The biotech firm reported that the Phase 3 trial of GTX-102, aimed at treating Angelman syndrome, yielded no measurable advantage over placebo. Earlier-stage studies had generated optimism among families and advocates hoping for cognitive improvements. The setback undermines expectations that the therapy could become a breakthrough for intellectual-disability disorders. Ultragenyx, which already markets several ultra-rare disease treatments, now faces a blow to its profitability outlook.
Why it matters
The trial failure curtails hopes for a new treatment for Angelman syndrome and impacts Ultragenyx's growth prospects.
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