Spruce Biosciences Appoints Dr. Jessica Cohen Pfeffer as Vice President of Clinical Development to Advance Rare Neurological Disease Pipeline
Spruce Biosciences, Inc., a late-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for neurological disorders with significant unmet medical needs, has announced the appointment of Jessica Cohen Pfeffer, M.D., as Vice President of Clinical Development, effective immediately. The appointment strengthens the company’s clinical leadership as it prepares for several important regulatory and commercial milestones, including the planned Biologics License Application (BLA) submission for its lead investigational therapy, tralesinidase alfa enzyme replacement therapy (TA-ERT), being developed for Sanfilippo Syndrome Type B (MPS IIIB).
Dr. Cohen Pfeffer joins Spruce with more than ten years of pharmaceutical industry experience across clinical development, medical affairs, portfolio management, and regulatory strategy. Throughout her career, she has focused extensively on rare genetic and neurodegenerative diseases, helping advance multiple enzyme replacement therapies from clinical development through global regulatory approvals. Her experience is expected to play an important role as Spruce continues to move its lead program toward potential commercialization.
Strengthening Leadership During a Critical Stage of Development
The appointment comes at an important point in Spruce Biosciences’ evolution as the company transitions from late-stage clinical development toward regulatory submission and commercial readiness.
Over the past several years, Spruce has concentrated its efforts on developing therapies for neurological disorders that currently have few or no effective treatment options. The company’s primary focus is on diseases where existing therapies are limited and where significant unmet medical need remains.
With TA-ERT progressing toward a planned regulatory submission, expanding the clinical leadership team has become a strategic priority.
Bringing an experienced physician-scientist with deep expertise in rare disease development is intended to support both the final stages of clinical development and future interactions with regulatory agencies worldwide.
Extensive Experience in Rare Disease Drug Development
Dr. Jessica Cohen Pfeffer has built an impressive career centered on therapies for rare inherited disorders, particularly diseases affecting children.
Her professional background includes leadership roles spanning:
- Clinical development
- Clinical sciences
- Medical affairs
- Global product strategy
- Portfolio management
- Regulatory support
She has worked extensively on enzyme replacement therapies designed to treat rare lysosomal storage disorders and inherited metabolic diseases.
Her experience includes supporting programs through clinical development while also helping prepare regulatory submissions and post-approval lifecycle management.
These capabilities closely align with Spruce’s current priorities as it advances its own enzyme replacement therapy program.
Leadership at BioMarin Pharmaceutical
Before joining Spruce Biosciences, Dr. Cohen Pfeffer spent more than a decade at BioMarin Pharmaceutical, one of the world’s leading biotechnology companies specializing in rare genetic diseases.
During her tenure, she held positions of increasing responsibility across multiple functional areas.
Most recently, she served as Executive Medical Director, where she acted as the clinical sciences lead for Brineura® (cerliponase alfa), an enzyme replacement therapy approved for CLN2 disease, a rare pediatric neurodegenerative disorder.
In this leadership position, she oversaw numerous aspects of clinical development, including:
- Clinical study completion
- Clinical study report preparation
- Regulatory documentation
- Scientific strategy
- Cross-functional collaboration
She also played an instrumental role in supporting several important regulatory achievements.
These included:
- FDA supplemental Biologics License Application (sBLA) efficacy approval
- European Medicines Agency (EMA) Type II variation approval
- United Kingdom MHRA post-authorization measure approval
These accomplishments demonstrate her familiarity with complex international regulatory pathways for biologic therapies.
Broad Portfolio Leadership
Beyond Brineura, Dr. Cohen Pfeffer contributed to several additional rare disease programs at BioMarin.
She served as an asset team leader supporting the company’s broader enzyme therapy portfolio, including Palynziq® (pegvaliase-pqpz) and multiple commercial enzyme replacement therapies.
She also held leadership responsibilities within BioMarin’s North American Medical Affairs organization while contributing to the company’s global medical leadership team.
These roles provided experience coordinating scientific strategy across clinical development, commercial planning, physician education, and lifecycle management.
Such multidisciplinary expertise will likely benefit Spruce as it prepares both regulatory filings and future commercialization activities.
Expertise in Mucopolysaccharidosis Disorders
One aspect of Dr. Cohen Pfeffer’s background is particularly relevant to Spruce’s current pipeline.
She has previously contributed to the development and lifecycle management of therapies targeting multiple forms of mucopolysaccharidosis (MPS).
Her work has included programs addressing:
- Morquio Syndrome (MPS IV)
- Maroteaux-Lamy Syndrome (MPS VI)
These disorders belong to the same family of inherited lysosomal storage diseases as Sanfilippo Syndrome Type B (MPS IIIB).
Because these diseases share similar biological mechanisms, regulatory considerations, and clinical development challenges, her previous experience provides valuable insight into advancing therapies for patients affected by MPS disorders.
Clinical Background Before Industry
Prior to entering the biotechnology industry, Dr. Cohen Pfeffer practiced medicine as a pediatric physician.
She served as an attending pediatrician and held a faculty appointment at the Mount Sinai School of Medicine in New York.
Her academic training includes:
- Medical degree from the Universidad Central de Venezuela
- Pediatric residency at Miami Children’s Hospital
- Fellowship in Clinical Genetics
- Fellowship in Clinical Biochemical Genetics at Mount Sinai School of Medicine
This combination of pediatric medicine, genetics, and rare metabolic disease expertise provides a strong scientific foundation for developing therapies targeting inherited neurological disorders.
Supporting the Development of TA-ERT
One of Dr. Cohen Pfeffer’s primary responsibilities at Spruce Biosciences will be supporting the continued development of tralesinidase alfa enzyme replacement therapy (TA-ERT).
TA-ERT is being investigated as a potential treatment for Sanfilippo Syndrome Type B (MPS IIIB), a rare inherited lysosomal storage disorder caused by deficiency of the enzyme alpha-N-acetylglucosaminidase.
The disease leads to accumulation of complex sugars within cells, resulting in progressive neurological deterioration, developmental regression, behavioral abnormalities, cognitive decline, and shortened life expectancy.
Currently, there are no approved disease-modifying therapies specifically targeting the underlying cause of MPS IIIB.
Spruce hopes that TA-ERT may become the first therapy capable of modifying disease progression rather than simply managing symptoms.
Preparing for Regulatory Submission
According to Spruce, one of the company’s highest priorities is preparing its planned Biologics License Application for TA-ERT.
The BLA submission represents one of the final regulatory steps before potential FDA review and possible product approval.
Successfully completing this process requires extensive coordination involving:
- Clinical data
- Manufacturing information
- Regulatory documentation
- Safety analyses
- Quality assurance
- Scientific communications
Dr. Cohen Pfeffer’s previous experience supporting multiple biologic approvals is expected to strengthen these efforts.
Executive Leadership Perspective
Spruce Chief Medical Officer Adrian Quartel, M.D., FFPM, welcomed Dr. Cohen Pfeffer to the organization while highlighting the importance of her experience.
He noted that she has spent much of her career developing therapies for children living with rare genetic and neurodegenerative disorders.
Her leadership on enzyme replacement therapies—including one approved for a rare pediatric neurodegenerative disease—provides expertise directly relevant to Spruce’s clinical programs.
Quartel stated that her background will be particularly valuable as the company advances its regulatory strategy for TA-ERT while preparing for a potential commercial launch should regulatory approval be obtained.
Dr. Cohen Pfeffer’s Perspective
Following her appointment, Dr. Cohen Pfeffer expressed enthusiasm about joining Spruce during what she described as an important period for both the company and the MPS IIIB community.
She emphasized that much of her professional career has focused on developing treatments for children affected by devastating rare neurodegenerative diseases.
Having worked closely with patients and families facing progressive inherited disorders, she understands the urgent need for effective therapeutic options.
She stated that she looks forward to collaborating with Spruce’s scientific and clinical teams to advance TA-ERT toward becoming what could potentially be the first disease-modifying therapy available for individuals living with Sanfilippo Syndrome Type B.
Commitment to Rare Disease Innovation
Spruce Biosciences continues to focus its research efforts on neurological disorders with significant unmet medical needs.
Rare diseases often present unique challenges due to small patient populations, limited treatment options, and complex clinical development requirements.
The appointment of an executive with deep experience in rare disease therapeutics reflects the company’s continued commitment to advancing innovative treatments in these specialized areas.
By strengthening its leadership team ahead of important regulatory milestones, Spruce aims to position itself for long-term growth while accelerating development of therapies that may improve outcomes for patients with severe inherited neurological disorders.
Inducement Equity Award
In connection with her appointment, Spruce Biosciences granted Dr. Cohen Pfeffer restricted stock units (RSUs) covering 3,800 shares of the company’s common stock.
The award was approved by the Compensation Committee of the Board of Directors on August 3, 2026, as an employment inducement in accordance with Nasdaq Listing Rule 5635(c)(4).
Under the terms of the award, the RSUs will vest over a four-year period, with 25% of the shares vesting annually beginning September 15, 2026, provided Dr. Cohen Pfeffer remains employed with the company through each applicable vesting date.
The grant is governed by the provisions of Spruce Biosciences’ 2026 Inducement Plan and the associated award agreement.
As Spruce Biosciences advances toward key regulatory and commercialization milestones, expanding its executive leadership with experienced rare disease experts represents an important strategic step.
Dr. Jessica Cohen Pfeffer brings extensive experience spanning clinical medicine, genetics, regulatory strategy, and enzyme replacement therapy development—expertise that closely aligns with the company’s mission to develop transformative treatments for patients with severe neurological diseases.
With preparations underway for the planned BLA submission of TA-ERT and continued progress toward potential commercialization, Spruce believes its strengthened clinical leadership team will help guide the next phase of development while supporting its broader goal of delivering innovative therapies to patients living with rare neurodegenerative disorders that currently have few effective treatment options.
About Spruce Biosciences
Spruce Biosciences is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need. Spruce’s lead product candidate, tralesinidase alfa enzyme replacement therapy (TA-ERT), is in late-stage development for the treatment of mucopolysaccharidoses type IIIB (MPS IIIB), or Sanfilippo Syndrome Type B, a devastating pediatric neurodegenerative disorder for which there are no FDA-approved therapies. TA-ERT has received Breakthrough Therapy Designation, Rare Pediatric Disease Designation, Fast Track Designation and Orphan Drug Designation from the FDA, as well as Orphan Drug Designation in the European Union. To learn more, visit www.sprucebio.com and follow us on X, LinkedIn, Facebook and YouTube.