Busulfanum
Metachromatic Leukodystrophy
Floatz Rating
BB67/100
Confidence
Indicative
Sponsor
Masonic Cancer Center, University of Minnesota
Modality
Small molecule
Development Phase
Phase 2/3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
53High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT02171104CT.gov MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | — | Active Not Recruiting | — | — | — |
NCT01043640CT.gov Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Metachromatic Leukodystrophy
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Busulfanum (this asset) | Masonic Cancer Center, University of Minnesota | P2/3 | BB · 67 |
| Cyclophosphamide | Masonic Cancer Center, University of Minnesota | P2/3 | BB |
| Anti-Human T-Lymphocyte Immunoglobulin, Rabbit | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| Clofarabine | Masonic Cancer Center, University of Minnesota | P2 | B |
| Melphalan | Masonic Cancer Center, University of Minnesota | P2 | B |
| Cyclosporin A | Masonic Cancer Center, University of Minnesota | P2 | B |
| Alemtuzumab | Masonic Cancer Center, University of Minnesota | P2 | B |
| Hydroxyurea | Masonic Cancer Center, University of Minnesota | P2 | B |
| Cald Hr-D | Masonic Cancer Center, University of Minnesota | P2 | CCC |
+3 more in the Metachromatic Leukodystrophy cohort
Other indications for Busulfanum
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Myelodysplastic/Myeloproliferative Neoplasm | — | P2/3 | BBB · 76 |
| Graft Versus Host Disease | — | P4 | BBB · 74 |
| Disease Related To Hematopoietic Stem Cell Transplant | — | P3 | BBB · 74 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Citation
Floatz Terminal. Busulfanum in Metachromatic Leukodystrophy. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 13, 2026. https://terminal.floatz.ai/assets/busulfanum-metachromatic-leukodystrophy
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