Slide 1: Tralesinidase Alfa Enzyme Replacement Therapy for Sanfilippo Syndrome Type B Conference Call
Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) for the Treatment of Sanfilippo Syndrome Type B (MPS IIIB) Conference Call April 15, 2025 Javier Szwarcberg, M.D., M.P.H., CEO Samir Gharib, M.B.A., President & CFO Kirk Ways, M.D., Ph.D., CMO
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements about Spruce Biosciences, Inc. (“we,” “Spruce” or the “Company”). All statements other than statements of historical facts contained in this presentation are forward-looking statements, including statements about our st
Slide 3: Spruce Bio: Global Neuro Biopharma Company
aj Topline data anticipated in 1H 2026 Precision Psychiatry Program Novel mAb Pipeline Tildacerfont + Cortibon (Companion Diagnostic) for Major Depressive Disorder (MDD) Development partner (HMNC Brain Health) funding Phase 2 study Topline data anticipated in 1H 2026 CRH Antagonist mAb for Congenita
Slide 4: Program Overview
Program Overview sl sprucesio
Slide 5: Mucopolysaccharidosis IIIB (MPS IIIB): Progressive Fatal Neurodegenerative Pediatric Disorder
Autosomal recessive disorder affecting 1 in 200,000 newborns 1 A deficiency in alpha-N-acetylglucosaminidase (NAGLU) leads to lysosomal accumulation of Heparan Sulfate (HS)2 HS buildup impairs neuronal development ultimately leading to neurodegeneration and death Sources: 1. Disease modeling for Muc
Slide 6: Well-Defined Regulatory Pathway to Approval for Tralesinidase Alfa
HS-NRE is deemed to be a biomarker1 reasonably likely to predict clinical benefit and serve as basis for accelerated approval (AA) HS based AA in other MPS subtypes are being pursued (RARE,2 DNLI3, RGNX4 ) Prior agreement with FDA on the tralesinidase program on: Acceptability of HS based biomarker
Slide 7: Tralesinidase Alfa Anticipated Development Roadmap
NATURAL HISTORY TREATMENT Natural History Study 1-10 years of age DQ > 50 Concurrent Natural History Study 0-18 years of age No DQ requirement COMPLETED STUDIES SUPPORTING A POTENTIAL ACCELERATED APPROVAL (AA3) PLANNED STUDY FOR FULL APPROVAL 250-301 Ph 4 Confirmatory Trial N~14 (Initiating Anticipa
Slide 8: Tralesinidase Alfa Was Shown to Significantly and Durably Normalize CSF HS and HS-NRE Levels
Normalization of pathogenic substrate in CSF is the goal of therapy Sources: 1. Internal data on file; 2. J Clin Invest. 2023;133(2):e165076. https://www.jci.org/articles/view/165076 Tralesinidase alfa is designed to target lysosomal delivery and correct NAGLU deficiency Tralesinidase alfa, 300 mg w
Slide 9: Tralesinidase Alfa Was Shown to Normalize Liver and Stabilize Cortical Grey Matter Volume
Reflective of tralesinidase alfa removal of HS deposits in target organs Source: 1. J Clin Invest. 2023;133(2):e165076. https://www.jci.org/articles/view/165076 Tralesinidase alfa, 300 mg was ICV administered by a 10-minute infusion via an Ommaya reservoir weekly for 48 weeks Liver Volume 1 Cortical
Slide 10: Bayley and Kaufman Assessments: Most Benefit in Patients with Early Disease
Tralesinidase alfa treatment was associated with stable cognitive function more frequently in patients with early disease, relative to patents with late disease Early and late disease groups based on baseline cognition score3 Disease stability: No meaningful loss of cognitive function4 at endpoint e
Slide 11: Tralesinidase Alfa – Safety Profile and Immunogenicity
22 patients enrolled in the 48-week initial study and extension studies lasting up to 6 years ~6,000 total infusions have been delivered in the 48-week initial study and its extensions Tralesinidase alfa was generally well tolerated with no discontinuations due to drug or device related adverse even
Slide 12: Financials and Commercial
Financials and Commercial sl sprucesio
Slide 13: Financial Snapshot
Financial Snapshot 13 Capital Structure and Summary Financials as of December 31, 2024 Capital Structure Shares (M) Common Shares Outstanding 42.2 Equity Awards Issued and Outstanding1 5.8 Common Stock Warrants2 12.7 Fully Diluted Shares Outstanding 60.7 Cash and Debt 000’s Cash & Cash Equivalents $
Slide 14: Market Benchmark: Enzyme Replacement in MPS Driving Robust Growth
Consistent Growth Trajectories: Patient-centric commercial strategy linking awareness, diagnosis, and reimbursement using a small field team has generated multiple high-value markets Small patient numbers results in low overall payer burden: Multiple approved MPS ERTs confirm long-term potential Pre
Slide 15: Tralesinidase Alfa Enzyme Replacement Therapy for Sanfilippo Syndrome Type B Conference Call
Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) for the Treatment of Sanfilippo Syndrome Type B (MPS IIIB) Conference Call April 15, 2025 Javier Szwarcberg, M.D., M.P.H., CEO Samir Gharib, M.B.A., President & CFO Kirk Ways, M.D., Ph.D., CMO