# Ultragenyx Phase 3 Trial for Angelman Syndrome Fails; Company Plans Cost Cuts

Ultragenyx announced that its phase 3 clinical trial for an Angelman syndrome treatment did not meet its primary endpoints. The company now plans to implement significant expense reductions. The failure follows the recent FDA approval of Ultragenyx’s drug Genglycos, which had generated optimism about the company’s pipeline. The phase 3 flop is a major setback for Ultragenyx and for patients awaiting a therapy for Angelman syndrome.

Angelman syndrome is a rare neurogenetic disorder caused by loss of function of the maternal copy of the UBE3A gene. It leads to severe developmental delays, seizures, and motor and speech impairments. No approved therapies currently address the underlying genetic cause. The failure of a late-stage trial in this indication highlights the difficulty of developing treatments for complex monogenic disorders. For the broader biotech industry, the result underscores the high risk inherent in phase 3 trials, even for companies that have recently achieved regulatory success.

Source: [original report](https://www.fiercebiotech.com/biotech/ultragenyx-fails-phase-3-angelman-test-plots-significant-expenses-reduction)