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Long-Term Data Presented at the 22nd Annual WORLDSymposium™ Highlights Tralesinidase Alfa Enzyme Replacement Therapy’s Potential as the First Disease-Modifying Treatment Option for Sanfilippo Syndrome Type B (MPS IIIB)
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commerci
About this update from Spruce Biosciences, Inc.
[{"type":"text","content":" SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, announced that data from two different analyses presented today at the 22nd Annual WORLDSymposium™ demonstrate that the long-term administration of tralesinidase alfa enzyme replacement therapy (TA-ERT) resulted in rapid and durable reduction of heparan sulfate and preserved cognitive and non-cognitive outcomes in patients with Sanfilippo Syndrome Type B (MPS IIIB) relative to natural history patients. “This long-term data supports tralesinidase alfa enzyme replacement therapy as potentially the first disease-modifying treatment option for individuals living with Sanfilippo Syndrome Type B, a fatal condition for which no approved therapies currently exist,” said Nicole Muschol, M.D., International Center for Lysosomal Disorders (ICLD) at the University Medical Center Hamburg-Eppendorf in Germany and Principal Investigator. “The stabilization and preservation of cognitive function, receptive and expressive communication, and fine and gross motor skills observed over six years of TA-ERT treatment are meaningful and provide hope for the families and patients affected by this devastating disorder.” In an analysis of 22 patients who enrolled in interventional studies of TA-ERT and followed up to six years, TA-ERT treatment: Rapidly and durably normalized levels of cerebral spinal fluid heparan sulfate non-reducing end (CSF HS-NRE), a surrogate endpoint reasonably likely to predict clinical benefit in patients with MPS IIIB; Stabilized cognitive function as assessed by Bayley-III Cognitive Raw Score (BSID-C), the cognitive subscale of the validated Bayley Scales of Infant and Toddler Development - Third Edition (Bayley-III) scale relative to declines seen in untreated natural history patients; Stabilized cortical gray matter volume, which declined in untreated natural history patients, and normalized liver and spleen volume; and Was generally consistent with the safety profile of intracerebroventricular (ICV) administration; over the six-year study, approximately 6,000 doses were administered to 22 patients. New analysis using the validated Vineland Adaptive Behavior Scales – Second Additio...