Spruce Biosciences, Inc. presented data at the 18th International MPS & Related Lysosomal Diseases Symposium demonstrating that long-term treatment with tralesinidase alfa enzyme replacement therapy (TA-ERT) preserved cognitive and non-cognitive outcomes in patients with Sanfilippo Syndrome Type B (MPS IIIB). The findings, presented on June 4-7, 2026 in Florence, Italy, indicate that TA-ERT could become the first disease-modifying treatment for the fatal condition, which currently has no approved therapies. The analysis showed rapid and durable normalization of cerebral spinal fluid heparan sulfate non-reducing end (CSF HS-NRE), a surrogate endpoint reasonably likely to predict clinical benefit.
The presentation included data from 22 patients enrolled in interventional studies with follow-up of up to six years. Nicole Muschol, M.D., Principal Investigator for the TA-ERT clinical development program, delivered the findings. Dr. Muschol noted that in a progressive neurodegenerative disease like MPS IIIB, stability itself is a clinically meaningful outcome.
The data analysis revealed several key outcomes from TA-ERT treatment over the six-year period. The therapy stabilized cognitive function as assessed by the Bayley-III Cognitive Raw Score (BSID-C) relative to declines seen in untreated natural history patients. Additionally, TA-ERT stabilized receptive and expressive communication, as well as fine and gross motor skills, compared with declines in untreated patients as assessed by the Vineland Adaptive Behavior Scales – Second Edition (VABS-II).
Key Clinical Outcomes
| Outcome Area |
Result with TA-ERT |
Comparison to Natural History |
| CSF HS-NRE Levels |
Rapid and durable normalization |
Reduced levels in untreated patients |
| Cognitive Function |
Stabilized (BSID-C) |
Decline observed |
| Communication & Motor Skills |
Stabilized (VABS-II) |
Decline observed |
| Cortical Gray Matter Volume |
Stabilized |
Decline observed |
| Liver and Spleen Volume |
Normalized |
Not applicable |
The treatment also stabilized cortical gray matter volume, which declined in untreated natural history patients, and normalized liver and spleen volume. Safety data showed the profile was generally consistent with intracerebroventricular (ICV) administration. Approximately 6,000 doses were administered to 22 patients over the six-year study duration.
Sanfilippo Syndrome Type B (MPS IIIB) is an ultra-rare, serious, and fatal genetic disease characterized by a deficiency in the N-Acetyl-Alpha-Glycosaminidase (NAGLU) enzyme. It affects fewer than one in 200,000 people in the United States. The accumulation of toxic levels of cerebral spinal fluid heparan sulfate in the brain drives the underlying pathophysiology, leading to progressive neurodegeneration, cognitive impairment, and motor skill deficits. The estimated life expectancy for individuals with MPS IIIB ranges from 15 to 19 years of age.