Treosulfan
Disease Related To Hematopoietic Stem Cell Transplant
Explore 55 assets in Disease Related To Hematopoietic Stem Cell Transplant →Floatz Rating
B60/100
Confidence
Indicative
Sponsor
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
45Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT07316595CT.gov Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome | — | Not Yet Recruiting | — | — | — |
NCT05534620CT.gov Effect of Moderate Renal Impairment and Race/Ethnicity on Treosulfan Pharmacokinetics | — | Completed | — | — | — |
NCT07232953CT.gov Safety and Efficacy of FT14 Conditioning for Allogeneic HSCT in Acute Myeloid Leukemia | — | Active Not Recruiting | — | — | — |
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 · Disease Related To Hematopoietic Stem Cell Transplant
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Treosulfan (this asset) | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | P2 | B · 60 |
| Busulfanum | Institute of Hematology & Blood Diseases Hospital, China | P3 | BBB |
| Cyclophosphamide | The First Affiliated Hospital of Soochow University | P3 | BBB |
| Nivestim | National Cancer Institute (NCI) | P2/3 | BBB |
| Rituximab | M.D. Anderson Cancer Center | P2 | BBB |
| Decitabine | Chinese PLA General Hospital | P4 | BBB |
| Cyclosporin A | The First Affiliated Hospital of Soochow University | P4 | BB |
| Tacrolimus | Oncoimmune, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) | P3 | BB |
| Thiotepa | medac GmbH | P2 | BB |
+42 more in the Disease Related To Hematopoietic Stem Cell Transplant cohort
Other indications for Treosulfan
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Myelodysplastic Syndrome | — | P3 | BB · 68 |
| Acute Biphenotypic Leukemia | — | P2/3 | BB · 65 |
| Adult Acute Lymphoblastic Leukemia | — | P2/3 | BB · 65 |
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.
Related assets
Citation
Floatz Terminal. Treosulfan in Disease Related To Hematopoietic Stem Cell Transplant. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/treosulfan-disease-related-to-hematopoietic-stem-cell-transplant-124701
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