Ultragenyx to consider major cost cuts after Angelman drug fails trial

Ultragenyx announced that its experimental therapy GTX-102 (apazunersen) for Angelman syndrome failed to meet primary and secondary endpoints in a Phase 3 study.

The company said there were no differences between treated and control groups, prompting a review of the program and a decision on its disposition.

In response, Ultragenyx indicated it will seek significant expense reductions, shifting focus to its approved products and targeting profitability by 2027.

Analysts noted the disappointment, with little optimism for sub‑group benefits, and the stock fell nearly 50% after the news.