Spruce acquires tralesinidase alfa enzyme replacement therapy

Spruce Biosciences (SPRB) announced the company’s new corporate strategy and acquisition of tralesinidase alfa enzyme replacement therapy for the treatment of Sanfilippo Syndrome Type B. Spruce intends to seek U.S. accelerated approval of TA-ERT for MPS IIIB based on existing non-clinical and clinical data. As a condition of seeking such approval of a BLA from the FDA, Spruce will initiate a confirmatory trial. If the BLA is approved, Spruce intends to build a highly specialized commercial and medical affairs organization to support the commercialization of TA-ERT. Given that a relatively small number of clinicians and specialists treat most of the patients with MPS IIIB, the company believes this market can be effectively addressed with a modest-sized and targeted patient-centric field team, alongside various high-touch patient initiatives. Spruce seeks to commercialize TA-ERT and its other investigational products throughout the developed world, including North America, the European Union, the United Kingdom, Latin America, Turkey, Asia, and other international markets. The company intends to establish its own commercial organization in the U.S., EU, and the U.K., and seek regional strategic collaborations and a network of third-party distributors in other international markets. Spruce focuses on diseases that have biology that is well understood. The company believes that developing drugs that directly impact known disease pathways will increase the probability of success of its development programs. Spruce entered into an asset purchase agreement under which the company acquired an exclusive worldwide license agreement with BioMarin Pharmaceutical (BMRN) for TA-ERT and other enzyme replacement therapy products. TA-ERT is a fusion protein comprised of recombinant human alpha-N-acetylglucosaminidase with modified human insulin-like growth factor 2 via an amino acid linker. TA-ERT is intended as an enzyme replacement therapy for the treatment of patients with MPS IIIB who lack rhNAGLU enzyme activity. In March 2024, in a Type C meeting with the FDA, the FDA confirmed that HS-NRE is deemed to be a surrogate biomarker reasonably likely to predict clinical benefit and could serve as a basis for accelerated approval. The FDA also confirmed that the completed clinical and non-clinical studies of TA-ERT were sufficient for a BLA submission and provided guidance around key design elements of a confirmatory trial, which must be initiated prior to potential accelerated approval of TA-ERT. TA-ERT has received fast-track designation, rare pediatric disease designation, and orphan drug designation in the U.S. and EU. Spruce intends to submit the BLA of TA-ERT for the treatment of MPS IIIB in the first half of 2026.