Ultragenyx’s GTX-102 Shows Promising Results in Mid-Stage Angelman Syndrome Study, Pivotal Study on the Horizon

1. Ultragenyx, a biopharmaceutical company, has achieved positive interim results from a Phase I/II study of its antisense oligonucleotide, GTX-102, in patients with Angelman Syndrome.
2. The study demonstrated significant improvements in cognitive, behavioral, sleep, and gross motor functions.
3. Following this mid-stage win, Ultragenyx is now planning a pivotal study to further evaluate the efficacy and safety of GTX-102 for Angelman Syndrome.
4. The positive data was announced by Ultragenyx approximately two days ago, on April 14, 2024.
5. The Angelman Syndrome Foundation has also shared the news about the positive interim Phase 1/2 data from GTX-102.