Spruce Biosciences (SPRB) investor relations material

Key program developments and regulatory landscape

• Lead asset Tralesinidase alfa targets MPS IIIB, a severe pediatric neurodegenerative disorder with no approved therapies.

• FDA's 2024 forum recognized heparan sulfate as a reliable surrogate endpoint, enabling regulatory progress for multiple companies.

• Recent Type B meetings with FDA were constructive, confirming trial design and manufacturing plans; BLA submission targeted for Q4.

• Confirmatory global trial will enroll 14 patients over 5 years, with clear rescue criteria and focus on regions without commercial access.

• Regulatory tailwinds include Priority Review Voucher eligibility and positive agency engagement.

Clinical data and therapeutic impact

• Long-term data show profound reductions in heparan sulfate and improvements in cognitive, adaptive, and motor functions, especially with early treatment.

• Stability of disease in later-treated patients is considered a significant benefit in this progressive disorder.

• Safety profile is favorable, with most adverse events manageable and no discontinuations due to side effects.

• Weekly intracerebroventricular dosing is well tolerated, with up to 6 years of follow-up supporting long-term safety.

• No clear correlation between baseline heparan sulfate and functional outcomes, but robust biomarker and clinical data support disease modification.

Market opportunity and commercialization plans

• U.S. patient population estimated at 150–200, with similar or larger numbers ex-U.S.; commercial launch will require a small, specialized team.

• Diagnosis relies on genetic panels; newborn screening inclusion likely after product approval, increasing awareness and patient identification.

• Recent hire of a new head of commercial and focus on patient access infrastructure.

• Strategic partnerships, especially in Asia, are being explored to expand reach.

• Priority Review Voucher could be monetized post-approval, providing additional financial flexibility.