Ultragenyx has found early success with its experimental treatment for the rare disease Angelman syndrome, supporting the company’s decision to acquire its development partner two years ago.
Ultragenyx is one of the few companies developing a treatment for Angelman syndrome, a genetic condition that causes delayed development, problems with speech and balance, mental disability, and sometimes seizures.
The biotech reported Monday that its drug, the antisense oligonucleotide treatment GTX-102, was generally safe and led to rapid and clinically meaningful improvements in cognition, communication, and sleep. The next step is a meeting with the Food and Drug Administration to discuss the findings and plans for a Phase 3 pivotal study.