Floatz Rating
BB66/100
Confidence
Indicative
Sponsor
Memorial Sloan Kettering 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.
54High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05736419CT.gov A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) | — | Recruiting | — | — | — |
NCT03249831CT.gov A Blood Stem Cell Transplant for Sickle Cell Disease | — | Active Not Recruiting | — | — | — |
NCT03609840CT.gov Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | — | Completed | — | — | — |
NCT03609814CT.gov Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT) | — | Completed | — | — | — |
NCT02105766CT.gov Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure | — | Active Not Recruiting | — | — | — |
NCT02512679CT.gov Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | — | Terminated | — | — | — |
NCT00578292CT.gov Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO | — | Terminated | — | — | — |
NCT00176852CT.gov Stem Cell Transplant for Hemoglobinopathy | — | Completed | — | — | — |
NCT00040469CT.gov Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies | — | Terminated | — | — | — |
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 · Thalassemia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Cyclophosphamide (this asset) | Memorial Sloan Kettering Cancer Center | P2/3 | BB · 66 |
| Deferasirox | Novartis Pharmaceuticals | P4 | BBB |
| Fludarabine | Memorial Sloan Kettering Cancer Center | P2/3 | BB |
| Hydroxyurea | Riphah International University | P4 | BB |
| Ppsv23 | Fakultas Kedokteran Universitas Indonesia | P4 | BB |
| Zinc Sulfate | Fakultas Kedokteran Universitas Indonesia | P4 | BB |
| Etavopivat | Novo Nordisk A/S | P3 | BB |
| Conjugated Pneumococcal Vaccine | Fakultas Kedokteran Universitas Indonesia | P4 | BB |
| Busulfanum | University of California, San Francisco | P2/3 | BB |
+42 more in the Thalassemia 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 Thalassemia. 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-thalassemia
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →