Spruce Biosciences (SPRB) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Regulatory and clinical development

• TA-ERT, an enzyme replacement therapy for MPS IIIB, is advancing toward FDA approval with a BLA submission planned for Q4 2024 and potential approval by mid-2027.

• FDA has accepted heparan sulfate as a surrogate endpoint for accelerated approval, following consensus at a 2024 Reagan-Udall Foundation forum.

• The agency has shown flexibility on CMC requirements, allowing staggered PPQ batch submissions, and maintains a stable, collaborative review team.

• Long-term data show durable reductions in heparan sulfate and strong clinical benefits in cognition and adaptive behavior, especially when treatment starts early.

• A confirmatory trial using cognitive endpoints is planned to run during regulatory review.

Market opportunity and commercialization

• The MPS IIIB market is characterized by high unmet need, strong patient advocacy, and centralized care at specialized centers.

• TA-ERT is expected to be well-tolerated and easier to administer than existing ICV therapies, with no hypersensitivity reactions observed.

• Peak sales potential is estimated at over $1 billion, supported by improved diagnosis and extended patient survival.

• Commercialization will focus on a targeted U.S. sales force and patient support hub, with potential for direct European launch and partnerships in Asia.

• The company is open to expanding its rare disease portfolio to leverage its commercial infrastructure.

Financial position and milestones

• Cash runway extends into early 2027, with a $50 million debt facility (of which $15 million drawn) and additional tranches tied to milestones.

• Potential monetization of a priority review voucher could yield $100–$150 million.

• Exploring non-dilutive capital through regional partnerships, especially in Asia.

• Key 2026 milestones include BLA submission, manufacturing scale-up, and initiation of the confirmatory trial.

• Additional pipeline asset for CAH (a monoclonal CRH ligand) may enter development this year.