Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Memorial Sloan Kettering Cancer Center
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.
38Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04644016CT.gov Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders | — | Recruiting | — | — | — |
NCT01652092CT.gov Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | — | Active Not Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Moderate confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Hemophagocytic Syndrome
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Busulfanum (this asset) | Memorial Sloan Kettering Cancer Center | P2 | CCC · 53 |
| Etoposide | Beijing Tongren Hospital | P4 | BBB |
| Pegaspargase | Beijing Tongren Hospital | P4 | BB |
| Adriamycin | The First Affiliated Hospital of Soochow University | P4 | BB |
| Zanubrutinib | The First Affiliated Hospital of Soochow University | P4 | BB |
| Cis-Diamminedichloroplatinum Ii | Zhengzhou University | P4 | BB |
| Rituximab | The First Affiliated Hospital of Soochow University | P4 | BB |
| Thiotepa | University of Florida | P2 | BB |
| Dexasone | Zhengzhou University | P4 | BB |
+23 more in the Hemophagocytic Syndrome 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.
Related assets
Citation
Floatz Terminal. Busulfanum in Hemophagocytic Syndrome. 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/busulfanum-hemophagocytic-syndrome
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