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Spruce Biosciences Secures Breakthrough Therapy Designation for Rare Disease Treatment
6 Oct
Summary
- FDA grants Breakthrough Therapy Designation to Spruce Biosciences' enzyme replacement therapy for Sanfilippo Syndrome Type B
- Therapy shows potential to normalize cerebral spinal fluid heparan sulfate, a key factor in neurodegeneration
- Company maintains strong financial position with more cash than debt
In a significant development, Spruce Biosciences, a small-cap biotech company, has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B (MPS IIIB). This rare genetic disease is characterized by a deficiency in the enzyme required for the breakdown of heparan sulfate, leading to progressive neurodegeneration, cognitive impairment, and early death.
The Breakthrough Therapy Designation was supported by clinical data showing that TA-ERT was able to normalize cerebral spinal fluid heparan sulfate non-reducing end (CSF HS-NRE), a key pathogenic factor in the disease. The FDA has confirmed that this biomarker is reasonably likely to predict clinical benefit, potentially serving as a basis for accelerated approval.
Despite the company's stock experiencing significant volatility, with a beta of 2.48, InvestingPro analysis suggests that Spruce Biosciences' stock is currently trading below its Fair Value. The company maintains a strong financial position, with more cash than debt on its balance sheet and a healthy current ratio of 2.6, indicating its ability to meet short-term obligations.
In other recent news, Spruce Biosciences has announced that it will resume trading on the Nasdaq Capital Market, marking a significant step for the company as it re-engages with the public trading community.
