Drugs / AX 250
AX 250
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02754076 | Apr 2016 → Jun 2020 | mucopolysaccharidosis type 3B | Allievex Corporation | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT03784287 | Feb 2018 → Jan 2025 | mucopolysaccharidosis type 3B | Allievex Corporation | Unknown | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT07579910 | Dec 2026 → Aug 2033 expected | mucopolysaccharidosis type 3B | Spruce Biosciences | Not yet recruiting | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT05492799 | Dec 2022 → Oct 2027 expected | mucopolysaccharidosis type 3B | Allievex Corporation | Enrolling by invitation | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2026-06-08 | Spruce Biosciences | Results Spruce Biosciences Announces Long-Term Tralesinidase Alfa Enzyme Replacement Therapy Data in Sanfilippo Syndrome Type B (MPS IIIB) Presented at the 18th International MPS Symposium sprucebio.com ↗
The data show that long-term administration of TA-ERT resulted in rapid and durable reduction of heparan sulfate and preserved cognitive and non-cognitive outcomes relative to natural history patients. |
| 2026-02-05 | Spruce Biosciences | Results Long-Term Data Presented at the 22nd Annual WORLDSymposium™ Highlights Tralesinidase Alfa Enzyme Replacement Therapy’s Potential as the First Disease-Modifying Treatment Option for Sanfilippo Syndrome Type B (MPS IIIB) sprucebio.com ↗
TA-ERT Treatment Stabilized and Preserved Cognitive and Non-Cognitive Outcomes, such as Communication and Motor Skills for Over Six-Year Period Relative to Natural History Patients |
| 2025-10-06 | Spruce Biosciences | Regulatory Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB) sprucebio.com ↗
Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B (MPS IIIB). |
| 2025-08-14 | Spruce Biosciences | Results Spruce Biosciences Reports Second Quarter 2025 Financial Results and Provides Corporate Updates sprucebio.com ↗
The integrated long-term clinical data of TA-ERT reinforces its potentially transformative clinical impact and compelling value proposition. |
| 2025-08-14 | Spruce Biosciences | Results Spruce Biosciences Announces Integrated Long-Term Clinical Data of Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) Demonstrating Profound and Durable Efficacy and Safety in Patients with Sanfilippo Syndrome Type B (MPS IIIB) sprucebio.com ↗
Spruce Biosciences, Inc. (OTCQB: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need, today announced results from a long-term data integration of tralesinidase alfa enzyme replacement therapy (TA-ERT) clinical program in patients with Sanfilippo Syndrome Type B (MPS IIIB). |
All press releases naming this drug 6 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AX 250 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05492799 ↗ |
| Known as | rhNAGLU-IGF2 | “Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)” NCT03784287 ↗ |
| Known as | tralesinidase alfa | “A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B.” PMID 36413418 ↗ Jan 2023 |
| Action | Restore | “Enzyme replacement therapy for neuronopathic MPS requires efficient enzyme delivery throughout the brain in order to normalize HS levels, prevent brain atrophy, and potentially...” PMID 36413418 ↗ Jan 2023 |
| Modality | Protein / enzyme biologic | “Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human insulin-like growth factor 2 (rhNAGLU-IGF2)” NCT02754076 ↗ |
| Route | Other | “administered i.c.v.” PMID 36413418 ↗ Jan 2023 |