Floatz Rating
BB71/100
Confidence
Indicative
Sponsor
M.D. Anderson Cancer Center
Modality
Small molecule
Development Phase
Phase 4 / Approved
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
59High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04776850CT.gov Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies | — | Withdrawn | — | — | — |
NCT03653338CT.gov T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | — | Recruiting | — | — | — |
NCT03171831CT.gov Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major | — | Unknown | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Beta-Thalassemia Major
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Cyclophosphamide (this asset) | M.D. Anderson Cancer Center | Approved | BB · 71 |
| Deferasirox | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Hydroxyurea | Bahria University | P4 | BBB |
| Deferiprone | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Tacrolimus | M.D. Anderson Cancer Center | P4 | BBB |
| Desferrioxamine B | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Busulfanum | M.D. Anderson Cancer Center | P4 | BBB |
| Fludarabine | M.D. Anderson Cancer Center | P4 | BBB |
| Cyclosporin A | First Affiliated Hospital of Guangxi Medical University | P4 | BB |
+42 more in the Beta-Thalassemia Major 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 Beta-Thalassemia Major. 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/cyclophosphamide-beta-thalassemia-major
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