Spruce Biosciences
From the brink to breakthrough: Delivering the first therapy for a critically underserved patient community
Key Highlights
- Back from the Brink with a Near-Commercial Asset: After disappointing data from its lead asset tildacerfont in congenital adrenal hyperplasia in 2024, Spruce was on the precipice. Delisted by NASDAQ and a cash runway through the end of 2025, the company had an ace up its sleeve with the acquisition of TA-ERT, a near-commercial therapeutic for the treatment of Sanfilippo syndrome type B, a rare and ultimately fatal neurodegenerative disease. Following its receipt of Breakthrough Therapy Designation for TA-ERT, the company raised $50 million, is now back on NASDAQ and looking forward to a potential FDA approval in 2026.
- Positioned to Bring the First Therapy to San Filippo Syndrome Type B Patients: With no FDA-approved therapies and limited options for even palliative care, the San Filippo Syndrome Type B community had been left by the wayside. A promising enzyme replacement therapy was developed by BioMarin but spun out to a company that later went bankrupt. Now Spruce Biosciences has picked up the baton with a plan to bring this potentially life-changing therapy to the patient community as early as 2026.
- 2026 Milestones: BLA Submission of TA-ERT for Sanfilippo Syndrome Type B anticipated 1Q 2026 and a PDUFA by September 30, 2026.