Spruce Biosciences (SPRB) Corporate presentation summary

Pipeline overview and development milestones

• Advancing tralesinidase alfa (TA-ERT) for MPS IIIB, with BLA filing anticipated in Q4 2026 and FDA Breakthrough Therapy Designation.

• TA-ERT program is BLA-ready, with no further clinical or non-clinical studies required for submission.

• SPR202, a novel anti-CRH monoclonal antibody for congenital adrenal hyperplasia (CAH), is in IND-enabling development.

• TA-ERT has first-to-market potential, orphan drug designations in US/EU, and IP exclusivity to 2038.

Clinical efficacy and safety data

• TA-ERT significantly reduces and durably normalizes CSF HS-NRE over five years, supporting accelerated approval.

• Therapy stabilizes cognition, communication, and motor skills compared to natural history in untreated children.

• TA-ERT normalizes liver and spleen volumes and stabilizes cortical grey matter volume.

• Safety profile is adequate for a serious, fatal disease; no deaths reported, most frequent serious TEAEs include vomiting and pyrexia.

Market opportunity and benchmarks

• Enzyme replacement therapies for MPS have shown consistent year-over-year growth, driven by improved diagnosis and awareness.

• Premium pricing and favorable reimbursement are supported by high unmet need and measurable clinical benefit.

• Small patient populations result in low overall payer burden, confirming long-term market potential.