Ultragenyx Releases Long-Term Data on UX111

Ultragenyx Pharmaceutical announced new long-term data from clinical studies evaluating UX111, an investigational AAV9 gene therapy for Sanfilippo syndrome Type A, a fatal neurodegenerative lysosomal storage disorder. The results demonstrate durable biomarker improvements and functional benefits compared with natural history, with consistent and highly statistically significant results across age and disease severity. "These data continue to demonstrate a remarkable and unprecedented separation from the natural history of Sanfilippo syndrome through more than eight years of follow-up, with children in their teens retaining skills at an age when many of their untreated peers have sadly lost their most basic abilities and succumbed to this disease," Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "Our studies consistently show that reductions in heparan sulfate are associated with meaningful clinical benefits across multiple domains, underscoring the urgency to bring forward a treatment for families who currently have no options to stop or delay the heartbreaking and inevitable progression and loss of function associated with this disease."