Floatz Rating
BB68/100
Confidence
Indicative
Sponsor
Fred Hutchinson Cancer Center
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.
55High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04965597CT.gov Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | — | Completed | — | — | — |
NCT02179359CT.gov Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | — | Terminated | — | — | — |
NCT00176852CT.gov Stem Cell Transplant for Hemoglobinopathy | — | Completed | — | — | — |
NCT00176878CT.gov Stem Cell Transplant for Bone Marrow Failure Syndromes | — | 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 · Shwachman-Diamond Syndrome
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Fludarabine (this asset) | Fred Hutchinson Cancer Center | P2/3 | BB · 68 |
| Cyclophosphamide | Roswell Park Cancer Institute | P2/3 | BB |
| Busulfanum | Masonic Cancer Center, University of Minnesota | P2/3 | BB |
| Tacrolimus | Fred Hutchinson Cancer Center | P2 | B |
| Equine Atg | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| Methotrexate | Fred Hutchinson Cancer Center | P2 | B |
| Anti-Human T-Lymphocyte Immunoglobulin, Rabbit | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| Melphalan | Roswell Park Cancer Institute | P2 | B |
| Treosulfan | Fred Hutchinson Cancer Center | P2 | B |
+5 more in the Shwachman-Diamond Syndrome cohort
Other indications for Fludarabine
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Adult T-Cell Leukemia/Lymphoma | — | P2 | BBB · 76 |
| Acute Myeloid Leukemia With Abnormal Bone Marrow Eosinophils Inv(16)(P13q22) Or T(16;16)(P13;Q22) | — | P2 | BBB · 76 |
| Fanconi Anemia | — | P2/3 | BBB · 76 |
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. Fludarabine in Shwachman-Diamond 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/fludarabine-shwachman-diamond-syndrome
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