Clinical Trials
28
17 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
2014
Active, not recruiting
15
53.6%
Available
1
3.6%
Completed
4
14.3%
Not yet recruiting
2
7.1%
Recruiting
2
7.1%
Terminated
4
14.3%
No approval data available
- Spruce Biosciences presented six-year data at the 22nd Annual WORLDSymposium demonstrating that tralesinidase alfa enzyme replacement therapy (TA-ERT) rapidly normalized cerebrospinal fluid heparan sulfate levels and preserved cognitive function in 22 patients with Sanfilippo Syndrome Type B. - The treatment stabilized cognitive, communication, and motor skills over six years compared to natural history patients, with approximately 6,000 doses administered showing a consistent safety profile. - A sibling comparison study revealed that the treated child maintained higher cognitive and functional abilities at age 12.1 years compared to an untreated sibling at age 11.7 years. - TA-ERT represents a potential first disease-modifying treatment for MPS IIIB, a fatal genetic disorder with no currently approved therapies and an estimated life expectancy of 15-19 years.
- Spruce Biosciences secured up to $50 million in growth capital from Avenue Capital Group to support development of tralesinidase alfa enzyme replacement therapy (TA-ERT) for MPS IIIB. - The financing includes an initial $15 million tranche funded January 2026, with three additional tranches totaling $35 million contingent on regulatory and commercial milestones. - TA-ERT represents a potential first disease-modifying therapy for Sanfilippo Syndrome Type B, a devastating neurological disorder with no FDA-approved treatments currently available. - The funding extends Spruce's cash runway into 2027, supporting the biologics license application filing and pre-launch commercial activities for TA-ERT.
- Spruce Biosciences appointed Keli Walbert, a veteran pharmaceutical commercial leader with over 20 years of experience, to its Board of Directors as the company prepares for a Biologics License Application submission for TA-ERT. - The appointment comes at a pivotal time as Spruce advances TA-ERT for the treatment of Sanfilippo Syndrome Type B (MPS IIIB), which could potentially become the first disease-modifying therapy for children with this rare neurological disorder. - Walbert brings extensive rare disease commercialization expertise from her role as Executive Vice President, U.S. Commercial at Horizon Therapeutics, where she led the successful launch of blockbuster medicine TEPEZZA and managed commercial strategy for over 10 marketed brands.
- Spruce Biosciences raised $50 million in private placement financing to advance tralesinidase alfa enzyme replacement therapy for Sanfilippo Syndrome Type B through biologics license application submission. - The funding will support the late-stage development program through BLA submission in Q1 2026 and potential U.S. commercial launch in late 2026. - The private placement involved approximately 502,181 shares at $68.00 per share, with some investors purchasing pre-funded warrants exercisable for five years.
- Spruce Biosciences announced that the FDA has granted Breakthrough Therapy Designation to tralesinidase alfa enzyme replacement therapy (TA-ERT) for treating Sanfilippo Syndrome Type B, an ultra-rare and fatal genetic disease. - Clinical data demonstrated that TA-ERT produces rapid, profound, and durable effects in normalizing cerebrospinal fluid biomarkers while stabilizing brain volume and cognitive function in pediatric patients. - The company plans to submit a Biologics License Application for TA-ERT in the first quarter of 2026, marking a significant regulatory milestone for this orphan disease treatment. - Following the announcement, Spruce Biosciences' stock surged 135.4 percent to $20.83 in pre-market trading, reflecting strong investor confidence in the therapy's potential.
- Tralesinidase alfa enzyme replacement therapy (TA-ERT) demonstrated profound and durable efficacy over five years in 22 patients with Sanfilippo Syndrome Type B, significantly reducing pathogenic biomarkers to normal levels. - The FDA confirmed that cerebrospinal fluid heparan-sulfate non-reducing end (CSF HS-NRE) is a surrogate biomarker reasonably likely to predict clinical benefit and could serve as basis for accelerated approval. - Treated patients showed stabilized cognitive function and cortical grey matter volume compared to progressive decline in untreated children, with cognitive differences reaching 34.66 points by age 10. - The therapy maintained an adequate safety profile over up to 7.3 years of exposure in this fatal disease with no currently approved treatments.
- HMNC Brain Health and Spruce Biosciences have dosed the first patient in the Phase 2 TAMARIND trial, evaluating tildacerfont as a precision treatment for major depressive disorder. - The trial targets a biologically distinct subtype of MDD patients with HPA axis dysregulation, using HMNC's proprietary genetic marker-based patient selection tool. - Tildacerfont, a CRF1 receptor antagonist, has the potential to address up to 50% of MDD patients worldwide through personalized medicine approaches. - The randomized, double-blind, placebo-controlled study will enroll 88 adults across eight UK sites, with topline results expected in the first half of 2026.
- Spruce Biosciences has acquired tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B, a neurodegenerative and fatal genetic disease with no FDA-approved treatments. - Clinical studies show TA-ERT significantly normalizes cerebral spinal fluid heparan sulfate levels over a five-year period, with FDA confirming this biomarker could serve as a basis for accelerated approval. - The company plans to submit a Biologics License Application to the FDA in the first half of 2026, with intentions to commercialize globally through a specialized patient-focused organization.
• The FDA has accepted two New Drug Applications for Neurocrine Biosciences' crinecerfont, granting Priority Review for treating classic congenital adrenal hyperplasia (CAH). • Crinecerfont, a selective CRF1 receptor antagonist, aims to manage ACTH and androgen levels without glucocorticoids, potentially revolutionizing CAH treatment. • If approved, crinecerfont would be the first new CAH treatment in 70 years, offering a novel mechanism to address this rare endocrine disorder. • The CAH market is expected to grow significantly, driven by emerging therapies like crinecerfont, with a projected CAGR of ~40% to reach USD 20 billion by 2034.
