Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

The latest data from the pivotal Transpher A and long-term follow-up studies will be presented at WORLDSymposium™ 2024

NOVATO, Calif., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebrospinal fluid (CSF) in patients with Sanfilippo syndrome type A (MPS IIIA), and that sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development. These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17). The results of this study along with the additional data from the long-term follow-up study for these subjects will be presented at the WORLDSymposium™ 2024 20th annual research meeting taking place February 4-9 in San Diego.

“It's impressive to see how our study patients treated with UX111 have maintained their communication skills despite being the age in which regression begins to occur,” stated Mireia del Toro, M.D., coordinator of the Metabolic Unit, Pediatric Neurology Department, Hospital Universitari Vall d´Hebron, Barcelona. “Sustaining the ability to communicate also has a very relevant impact on improving behavioral problems and thus family daily life.”

Following treatment with UX111 (3x1013 vg/kg), levels of CSF-HS decreased within the first month post treatment in all patients. 8 of 17 patients in the mITT group who reached 24 months post-treatment achieved an overall mean percent reduction from baseline of 51% (p