Spruce Biosciences (SPRB) investor relations material

Regulatory and clinical development updates

• Achieved Breakthrough Therapy designation from FDA, enabling productive Type B meetings on CMC and clinical matters, with positive and consistent feedback from regulators.

• FDA agreed on the use of heparan sulfate non-reducing end as a surrogate endpoint for accelerated approval, with robust supporting data and normalization in patients over six years.

• Confirmatory trial design was refined with FDA input, allowing trial initiation during BLA review and focusing on timely patient rescue and robust feasibility assessment.

• Clinical data show durable biomarker normalization, cognitive and functional improvements, and favorable safety profile, with evidence from sibling analysis and extension studies.

• BLA submission delayed to Q4 to include first PPQ batch per FDA request, with subsequent batches as mid-cycle and post-marketing commitments.

Commercial strategy and market outlook

• Hired a new Chief Commercial Officer and developed a three-pronged commercial strategy: patient identification, medical affairs and education, and pricing/market access.

• Commercial launch preparations include partnerships with specialty distributors, focus on MPS Centers of Excellence, and support for patient navigation and insurance coverage.

• Epidemiology estimates suggest a US and global peak prevalence of around 500 patients each, with potential for higher numbers as diagnosis improves.

• Market access strategy considers payer dynamics in ultra-rare diseases and the importance of early patient identification, including newborn screening initiatives.

Manufacturing and financial position

• Manufacturing transferred to Samsung Biologics, with validation and commercial launch batches in progress and no major hurdles anticipated.

• Ended 2025 with ~$50 million in cash, plus access to additional debt tranches tied to regulatory milestones, and exploring non-dilutive funding options.

• Financial runway expected to last into 2027, with a potential shortfall to be bridged by debt or strategic partnerships; priority review voucher could provide further funding if approved.