Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
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.
38Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03579875CT.gov Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders | — | Recruiting | — | — | — |
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 · Dyskeratosis Congenita
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Cyclophosphamide (this asset) | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | CCC · 53 |
| Nivestim | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | B |
| Rituximab | Masonic Cancer Center, University of Minnesota | P2 | CCC |
| Busulfanum | Masonic Cancer Center, University of Minnesota | P2 | CCC |
| Melphalan | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | CCC |
| Fludarabine | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | CCC |
| Alemtuzumab | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | CCC |
| Tacrolimus | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | CCC |
| Danazol | Boston Children's Hospital | P1/2 | CCC |
+2 more in the Dyskeratosis Congenita 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
- NivestimSidney Kimmel Comprehensive Cancer Center at Johns HopkinsB
- RituximabMasonic Cancer Center, University of MinnesotaCCC
- BusulfanumMasonic Cancer Center, University of MinnesotaCCC
- MelphalanSidney Kimmel Comprehensive Cancer Center at Johns HopkinsCCC
- FludarabineSidney Kimmel Comprehensive Cancer Center at Johns HopkinsCCC
Citation
Floatz Terminal. Cyclophosphamide in Dyskeratosis Congenita. 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-dyskeratosis-congenita
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