Spruce Biosciences targets Q4 2026 BLA filing for TA-ERT in MPS IIIB
The company also plans to start a required confirmatory trial in the fourth quarter and reported $96.3 million in cash as of June 30, 2026.
Spruce Biosciences reported second quarter 2026 results on August 12, 2026, along with updates on its lead program, tralesinidase alfa enzyme replacement therapy (TA-ERT) for MPS IIIB, also known as Sanfilippo Syndrome Type B. Spruce is working on manufacturing readiness, regulatory interactions and commercial planning ahead of a Biologics License Application it expects to submit in the fourth quarter of 2026, pursuing accelerated approval using cerebrospinal fluid heparan sulfate non-reducing end as a surrogate endpoint the company considers reasonably likely to predict clinical benefit.1 There is currently no FDA-approved therapy for MPS IIIB, and current care is limited to palliative measures.1
As a condition of potential accelerated approval, the FDA asked Spruce to begin its "TrAnsform" confirmatory study while the BLA is under review.1 The trial is designed as a randomized, single-blind, controlled, multicenter study comparing intracerebroventricular TA-ERT with standard of care in children ages 1 to 5 with severe MPS IIIB.1 It is expected to enroll about 14 participants, with cognition as the primary outcome measure.1 The study is expected to start in the fourth quarter of 2026.
Separately, Cure Sanfilippo Foundation and the National MPS Society made a combined $5.5 million investment in Spruce, with proceeds earmarked to help fund an Expanded Access Program for TA-ERT.1 That open-label, single-arm program is meant for U.S. children with attenuated or severe MPS IIIB who cannot join the TrAnsform study, is expected to enroll about 10 participants receiving weekly ICV-administered TA-ERT, and is expected to begin in the fourth quarter of 2026.1
On personnel, Spruce added two clinical leaders during and after the quarter to support the BLA submission, potential approval and commercial readiness, including a chief medical officer with more than 25 years of pharmaceutical experience in enzyme replacement therapy development.1
Cash and cash equivalents stood at $96.3 million as of June 30, 2026, up from $48.9 million at the end of 2025.1 The company said this is expected to fund planned operations and debt obligations into the second half of 2027.1
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