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SOUTH SAN FRANCISCO - Spruce Biosciences, Inc. (NASDAQ:SPRB), a small-cap biotech with a market capitalization of approximately $5 million, announced Monday that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B (MPS IIIB). According to InvestingPro data, the company maintains a strong financial position with more cash than debt on its balance sheet.
The designation was supported by clinical data showing normalization in cerebral spinal fluid heparan sulfate non-reducing end (CSF HS-NRE), which the FDA confirmed could serve as a surrogate biomarker reasonably likely to predict clinical benefit and potentially serve as a basis for accelerated approval. While the company’s stock has experienced significant volatility, with a beta of 2.48, InvestingPro analysis suggests the stock is currently trading below its Fair Value. Discover 10+ additional exclusive insights and real-time metrics with InvestingPro.
Breakthrough Therapy Designation is designed to expedite development and regulatory review of promising therapies for serious conditions where preliminary clinical evidence suggests potential substantial improvement over existing treatments. The designation provides more intensive FDA guidance and eligibility for rolling submission and priority review.
"The integrated group-level clinical data demonstrates a rapid, profound, and durable effect of TA-ERT in normalizing CSF HS-NRE, the pathogenic factor leading to neurodegeneration, and stabilizing cortical grey matter volume and cognitive function in children with MPS IIIB," said Javier Szwarcberg, Chief Executive Officer of Spruce Biosciences, in a press release statement.
MPS IIIB is an ultra-rare genetic disease characterized by deficiency in N-Acetyl-Alpha-Glycosaminidase (NAGLU), an enzyme required for the breakdown of heparan sulfate in lysosomes. The condition affects fewer than 1:200,000 people in the United States and typically leads to progressive neurodegeneration, cognitive impairment, and early death, with life expectancy ranging from 15 to 19 years.
TA-ERT is a fusion protein designed to restore enzyme activity in the central nervous system following intracerebroventricular injection. The therapy has been evaluated in three clinical studies involving 22 participants with MPS IIIB.
The company stated that its Biologics License Application submission for TA-ERT remains on track for the first quarter of 2026. Despite current negative profit margins and a challenging year that saw the stock decline over 70%, the company maintains a healthy current ratio of 2.6, indicating strong ability to meet short-term obligations. Analysts following undervalued biotech stocks can access comprehensive financial health scores and detailed metrics through InvestingPro’s advanced analytics platform.
In other recent news, Spruce Biosciences announced that it will resume trading on the Nasdaq Capital Market. The company has confirmed that trading will begin today under the ticker symbol SPRB. This development follows a period during which Spruce Biosciences was not actively trading on the market. The biopharmaceutical company shared this information in a press release, emphasizing its return to the Nasdaq platform. Investors and market participants will now have the opportunity to trade shares of Spruce Biosciences once again. The company, which focuses on developing therapies for neurological disorders, provided the CUSIP number 85209E 208 for reference. This marks a significant step for Spruce Biosciences as it re-engages with the public trading community.
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