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Spruce Biosciences Announces Integrated Long-Term Clinical Data of Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) Demonstrating Profound and Durable Efficacy and Safety in Patients with Sanfilippo Syndrome Type B (MPS IIIB)
TA-ERT Profoundly and Durably Lowered Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE), the Factor Responsible for Neurodegeneration in
About this update from Spruce Biosciences, Inc.
[{"type":"text","content":"\nTA-ERT Profoundly and Durably Lowered Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE), the Factor Responsible for Neurodegeneration in MPS IIIB\n\n\nU.S. FDA Confirmed that CSF HS-NRE is a Surrogate Biomarker Reasonably Likely to Predict Clinical Benefit and Could Serve as Basis for Accelerated Approval\n\n\nCognition and Cortical Grey Matter Volume Were Stabilized in Patients Treated with TA-ERT Relative to Declines Observed in Untreated Patients\n\n\n SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--\nSpruce Biosciences, Inc. (OTCQB: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced results from a long-term data integration of tralesinidase alfa enzyme replacement therapy (TA-ERT) clinical program in patients with Sanfilippo Syndrome Type B (MPS IIIB).\n\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250814678929/en/Figure 1. TA-ERT Therapy Leads to Profound and Durable Reduction in CSF HS-NRE Over a 5-Year Period. Dashed line represents upper limit of normal (10 ng/mL) for CSF HS-NRE; LS = least squares; LS mean ±SE based on mixed-model repeated measures (MMRM) analysis with study visit as covariate. Note: CSF HS-NRE levels were averaged over each 0.5-year interval.\nSpruce integrated and evaluated group-level efficacy data for cerebral spinal fluid heparan-sulfate non-reducing end (CSF HS-NRE), cortical grey matter volume (CGMV), and Bayley-III Cognitive Raw Score (BSID-C), the cognitive subscale of the Bayley Scales of Infant and Toddler Development - Third Edition (Bayley-III), as well as safety data over a five-year period from clinical studies 201, 202, and 401. Data from treated patients (n=22) in the studies 201, 202, and 401 was compared with data from untreated patients with MPS IIIB in natural history studies 901 and 902.\n\n\n“These data demonstrate a rapid, profound, and durable effect of ICV-administered TA-ERT on normalizing CSF HS-NRE, the pathogenic factor leading to neurodegeneration, and in stabilizing CGMV and cognitive function, relative to the declines observed in untreated children with Sanfilippo Syndrome Type B (MPS IIIB) in the natural history studies,” said Paul Harmatz, M.D., Principal I...