Ultragenyx Angelman drug fails late-stage trial, shares plunge

Ultragenyx Pharmaceutical announced on Wednesday that its experimental drug for Angelman syndrome failed to meet its main goals in a pivotal late-stage trial, a major setback for the company and the patient community awaiting the first potential treatment for the rare neurodevelopmental disorder. The drug, apazunersen, did not show statistically significant improvement compared to a placebo in the Phase 3 Aspire study, prompting the company to announce plans for significant cost reductions and a review of the program's future. The trial evaluated apazunersen's ability to improve cognitive function and overall developmental progress in pediatric patients with Angelman syndrome. According to the company, the drug failed to achieve its primary measure of improvement in cognitive scores and also missed crucial secondary measures of patient response. Ultragenyx stated there were no meaningful differences between participants receiving the treatment and those in the placebo group. The company said it will now evaluate the apazunersen program and make a decision on its disposition.