Spruce Biosciences Reports Positive FDA Meetings as It Prepares TA-ERT Filing for Rare Pediatric Disorder

Spruce Biosciences, Inc. (NASDAQ:SPRB) said it has completed two Type B meetings with the U.S. Food and Drug Administration (FDA), clearing key regulatory discussions ahead of its planned biologics license application (BLA) submission for tralesinidase alfa enzyme replacement therapy (TA-ERT), its experimental treatment for Sanfilippo Syndrome Type B (MPS IIIB).

The company met with the FDA in December 2025 to review clinical data and regulatory strategy, followed by a January 2026 meeting focused on chemistry, manufacturing and controls (CMC).

During the December meeting, the FDA indicated that integrated data from TA-ERT interventional studies, together with available natural history data, could potentially qualify as an adequate and well-controlled dataset to evaluate the therapy’s effect on cerebrospinal fluid heparan sulfate non-reducing end (CSF HS-NRE). The agency said CSF HS-NRE could serve as a reasonably likely surrogate endpoint (RLSE) to support accelerated approval.

The FDA also provided recommendations to strengthen the use of CSF HS-NRE as a surrogate marker, which Spruce said it is incorporating into its planned BLA submission. The company and the agency additionally aligned on the timing and design of a required confirmatory study, agreeing that the study could begin during the BLA review period.

At the January CMC meeting, the FDA reviewed Spruce’s approach to drug product process performance qualification (DP PPQ) batch requirements. According to official meeting minutes received on February 12, 2026, the agency requires one DP PPQ batch at the time of BLA filing and data from a second batch before the mid-cycle review.

As a result, Spruce now expects to submit its BLA for TA-ERT in the fourth quarter of 2026.

“The company appreciates the productive and constructive engagement with FDA as we gain clarity on specific clinical and CMC requirements expected of us by the Agency for our planned upcoming BLA submission of TA-ERT,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive Officer of Spruce Biosciences. “While the timing of our submission is now expected in the fourth quarter of this year, we believe in the strength of our long-term data for TA-ERT which demonstrates that reductions in CSF HS-NRE are associated with meaningful clinical benefits across cognition, communication, and motor skill acquisition. TA-ERT has the potential to provide a meaningful option to change the course of this fatal disease, and we are motivated every day to bring forward a treatment for families and patients with MPS IIIB who currently have no treatment options.”

Dr. Szwarcberg added that the recent reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) program through September 2029 restores an important incentive for companies developing treatments for rare childhood diseases.

“As we work to bring TA-ERT to the market as efficiently as possible, I want to commend the tireless advocacy on behalf of patients that led to the reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) program through September 2029, restoring a key incentive to develop therapies for rare pediatric diseases. The reauthorization of the PRV program has provided hope and renewed optimism to the Sanfilippo community and will enable us to successfully position TA-ERT, if approved, as potentially the first disease-modifying treatment option for MPS IIIB.”

About Sanfilippo Syndrome Type B (MPS IIIB)

Sanfilippo Syndrome Type B is an ultra-rare, inherited lysosomal storage disorder caused by a deficiency in the enzyme N-acetyl-alpha-glucosaminidase (NAGLU), which is necessary to break down heparan sulfate in cells. The accumulation of toxic levels of heparan sulfate in the brain drives progressive neurodegeneration.

The condition is estimated to affect fewer than one in 200,000 people in the United States, though the true prevalence is uncertain because it is not included in newborn screening programs. Patients typically experience cognitive decline, developmental delay, behavioral disturbances, speech impairment, hearing loss and motor deterioration. In advanced stages, severe dementia, loss of mobility and seizures are common, with most patients requiring full-time care. Life expectancy is generally between 15 and 19 years. There are currently no FDA-approved treatments.

About TA-ERT

TA-ERT is a fusion protein composed of recombinant human alpha-N-acetylglucosaminidase (rhNAGLU) designed to replace the deficient enzyme in patients with MPS IIIB. Administered via intracerebroventricular injection, the therapy is intended to restore enzyme activity in the central nervous system.

Because rhNAGLU alone is poorly taken up by cells, TA-ERT is fused to an insulin-like growth factor 2 peptide to facilitate cellular uptake and lysosomal delivery. By restoring NAGLU activity and promoting clearance of heparan sulfate and its non-reducing end fragments in the brain, the therapy aims to slow or halt neurological decline.

TA-ERT has been studied in three clinical trials—Study 201 and extension studies 202 and 401—and has been administered to 22 patients. Spruce reports an acceptable safety profile based on six years of integrated safety data.

The therapy has received Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the FDA.

About Spruce Biosciences

Spruce Biosciences is a late-stage biopharmaceutical company developing novel therapies for neurological disorders with significant unmet medical need.

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