Ultragenyx Pharmaceutical Development Pipeline

Ultragenyx Pharmaceutical provided updates on its development pipeline, including investigational GTX-102 for Angelman syndrome (AS) at Analyst Day held Monday, October 16, 2023. 

For GTX-102 the antisense oligonucleotide for Angelman syndrome, UGX reported data from the extension cohorts in the Phase 1/2 study which they believed showed clinically meaningful improvements in multiple domains and some changes in EEG parameters. They also reported no new serious adverse events related to use of the drug had been recorded since late 2022.

“The data we are presenting today show that these investigational therapies are having meaningful clinical effects on difficult diseases with limited or no approved treatments and are potentially transformative for people living with these diseases if proven safe and effective in Phase 3 studies,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. 

Ultragenyx will be presenting updates on the GTX-102 program for Angelman syndrome at the FAST Global Science Summit on Saturday, November 11th at 10:30am ET.  Register in-person or virtually here. 

You can also review an archived version of the remarks from Analysis Day through the Ultragenyx website.

To read the full press release, please click here.