Floatz Rating
BB68/100
Confidence
Indicative
Sponsor
Paul Szabolcs
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.
54High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03653338CT.gov T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | — | Recruiting | — | — | — |
NCT02512679CT.gov Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | — | Terminated | — | — | — |
NCT00176852CT.gov Stem Cell Transplant for Hemoglobinopathy | — | 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 · Diamond-Blackfan Anemia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Cyclophosphamide (this asset) | Paul Szabolcs | P2/3 | BB · 68 |
| Deferasirox | Novartis | P4 | BBB |
| Fludarabine | Fred Hutchinson Cancer Center | P2/3 | BBB |
| Busulfanum | Paul Szabolcs | P2/3 | BBB |
| Thiotepa | Fred Hutchinson Cancer Center | P2 | BB |
| Methotrexate | Fred Hutchinson Cancer Center | P2 | B |
| Anti-Human T-Lymphocyte Immunoglobulin, Rabbit | Masonic Cancer Center, University of Minnesota | P2/3 | B |
| Alemtuzumab | Paul Szabolcs | P2 | B |
| Tacrolimus | Fred Hutchinson Cancer Center | P2 | B |
+16 more in the Diamond-Blackfan Anemia cohort
Other indications for Cyclophosphamide
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Adult Acute Lymphoblastic Leukemia | — | P4 | BBB · 79 |
| Mycosis Fungoides | — | P4 | BBB · 78 |
| B-Cell Childhood Acute Lymphoblastic Leukemia | — | P3 | BBB · 78 |
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. Cyclophosphamide in Diamond-Blackfan Anemia. 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/cyclophosphamide-diamond-blackfan-anemia
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