Spruce Biosciences' TA-ERT Shows Promise in MPS IIIB Clinical Trials
Spruce Biosciences' TA-ERT, a fusion protein for MPS IIIB, has shown promising results in clinical trials. The therapy, designed as an enzyme replacement, has received U.S. FDA Breakthrough Therapy Designation for treating Sanfilippo Syndrome Type B. It aims to normalize a key biomarker and stabilize cognitive function in patients lacking the rhNAGLU enzyme.
TA-ERT's potential lies in its ability to efficiently deliver to lysosomes, thanks to its fusion with an insulin-like growth factor 2 peptide. Integrated long-term data from three clinical studies involving 22 MPS IIIB participants demonstrates its rapid, profound, and durable effect in normalizing cerebrospinal fluid (CSF) hexosaminidase A and B subunit N-acetylglucosamine (HS-NRE). This stabilization of cognitive function is particularly notable as MPS IIIB is a progressive neurodegenerative disease.
The therapy's safety profile has been deemed adequate based on five years of safety data. Spruce Biosciences plans to submit a Biologics License Application in the first quarter of 2026, with the potential for accelerated approval. The FDA has confirmed that CSF HS-NRE is a surrogate biomarker reasonably likely to predict clinical benefit for TA-ERT in MPS IIIB. This is significant as MPS IIIB is an ultra-rare, serious, and fatal genetic disease with no current disease-modifying therapies.
TA-ERT's BTD is a testament to its potentially transformative clinical impact. With no FDA-approved therapies currently available for MPS IIIB, and management limited to palliative care, TA-ERT offers hope for patients and their families. Spruce Biosciences' upcoming Biologics License Application submission could pave the way for this innovative therapy to reach those in need.
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