STAT+: Ultragenyx drug to treat Angelman syndrome, a rare disease, fails late-stage trial
Ultragenyx said its experimental therapy for Angelman syndrome, a rare disease, showed no benefit compared to a sham treatment in a Phase 3 trial.
Ultragenyx, a biotech firm, announced on Wednesday that its experimental treatment for Angelman syndrome, a rare disease causing severe intellectual disabilities and developmental delays, failed to demonstrate any advantage over a placebo in a large Phase 3 trial. The drug, called GTX-102, had previously shown promising results in earlier trials, sparking hope among families affected by the condition.
Many advocates for patients with other neurological disorders also anticipated that GTX-102 could be the first of several medicines to enhance cognition, communication, and other aspects of life for those with intellectual disabilities. The news represents a substantial setback for Ultragenyx's business prospects, as the company primarily focuses on ultra-rare diseases for which it already holds multiple approved medications.
Investors had placed significant hopes on the Angelman syndrome drug as Ultragenyx's route to financial success.
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