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Ultragenyx Drug Misses Key Goal for Rare Disorder
3 Sep
Summary
- Experimental apazunersen failed a late-stage trial for Angelman syndrome.
- The drug did not show meaningful improvement in cognitive abilities.
- Ultragenyx shares dropped 40% after the trial results were announced.
Ultragenyx Pharmaceutical announced on Wednesday that its experimental treatment, apazunersen, did not achieve its main objective in a late-stage clinical trial. The drug was being investigated as a therapy for Angelman syndrome, a rare genetic neurodevelopmental disorder.
Angelman syndrome impacts the nervous system and hinders typical brain development, affecting approximately 1 in 15,000 live births. The condition is commonly linked to a loss of function in the UBE3A gene, specifically on the copy inherited from the mother.
The late-stage trial results indicated that apazunersen did not exhibit significant improvement on key assessments. These included measures of cognitive and nonverbal reasoning abilities in young children, as well as an overall response across multiple developmental areas.
In the aftermath of this news, Ultragenyx Pharmaceutical's stock price saw a substantial drop, falling 40% in aftermarket trading. This market reaction underscores the significant impact of the trial's outcome on the company's valuation and future prospects.
