Ultragenyx Pharmaceuticals shares drop 43% after experimental Angelman syndrome treatment fails in Phase 3 trial
Ultragenyx Pharmaceuticals shares fell 43% after the company reported that its experimental drug, apazunersen, failed to meet primary and secondary endpoints in a Phase 3 trial for Angelman syndrome. The trial assessed the drug's ability to improve cognition in patients with the rare neurogenetic disorder, which causes developmental delays and speech impairment. Patients treated with apazunersen did not show significant improvements in cognitive raw scores or in clinical function as measured by the Multidomain Responder Index. Emil Kakkis, M.D., Ph.D., the chief executive officer and president of Ultragenyx Pharmaceuticals, expressed disappointment with the results, noting that the company is now evaluating the program's disposition. Following the trial failure, the company announced plans to assess operations to implement significant expense reductions. This move follows a previous restructuring and workforce reduction in February. While Ultragenyx Pharmaceuticals has other products, such as the recently approved Genglycos and the upcoming UX111 therapy, the failure of the apazunersen program represents a significant setback for the company's commercial goals.
Sources
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Ultragenyx fails phase 3 Angelman test, plots ‘significant expense reductions’
fiercebiotech.com
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'Fall From Grace' — Biotech Crashes 44% To A Record Low
Investor's Business Daily
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Ultragenyx to weigh ‘significant’ cost cuts as Angelman drug fails key study
BioPharma Dive