(RTTNews) - Spruce Biosciences, Inc. (SPRB), a late-stage biopharmaceutical company developing therapies for neurological disorders, announced the launch of MPS3BStudy.com, a resource for families and caregivers of children with Sanfilippo Syndrome Type B (MPS IIIB).
The website allows families and caregivers to learn about and express interest in the planned Expanded Access Program (EAP) and the TrAnsform confirmatory study of Spruce's investigational tralesinidase alfa enzyme replacement therapy (TA-ERT), both expected to begin as early as the fourth quarter of 2026.
Sanfilippo Syndrome Type B (MPS IIIB) is an ultra-rare, fatal genetic disorder caused by deficiency of the NAGLU enzyme, leading to toxic heparan sulfate accumulation in the brain and progressive neurodegeneration.
Spruce's lead investigational candidate, tralesinidase alfa enzyme replacement therapy (TA-ERT), is in late-stage development for the treatment of mucopolysaccharidoses type IIIB (MPS IIIB), or Sanfilippo Syndrome Type B, for which there are no FDA-approved therapies.
TA-ERT is an enzyme replacement therapy designed to restore NAGLU activity in the central nervous system and promote clearance of heparan sulfate.TA-ERT has received Breakthrough Therapy, Rare Pediatric Disease, Fast Track and Orphan Drug designations from the FDA.
The TrAnsform confirmatory study is a planned randomized, single-blind, controlled, multicenter trial evaluating ICV-administered TA-ERT against standard of care in children aged 1 to 5 years with severe MPS IIIB.
Through a simple registration form in the portal, families and caregivers can express interest in participation and receive updates as U.S. sites activate and enrollment begins.
Earlier this week, Spruce announced that it had completed two pre-BLA meetings with the FDA and said it remains on track to submit a biologics license application for TA-ERT in the fourth quarter of 2026.
SPRB has traded between $7.00 and $240 over the last year. The stock closed Wednesday's trade at $61.48.
As of writing, SPRB was up 1.19% at $62.21.
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