Floatz Rating
B63/100
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.
48Moderate confidence
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 | — | — | — |
NCT03128996CT.gov Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | — | Recruiting | — | — | — |
NCT01962415CT.gov Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | — | Recruiting | — | — | — |
NCT01049854CT.gov CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant | — | 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 · Bone Marrow Failure Syndrome
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Melphalan (this asset) | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2 | B · 63 |
| Fludarabine | Fred Hutchinson Cancer Center | P2/3 | BBB |
| Cyclophosphamide | Memorial Sloan Kettering Cancer Center | P2/3 | BBB |
| Thiotepa | Fred Hutchinson Cancer Center | P2 | BB |
| Busulfanum | Memorial Sloan Kettering Cancer Center | P2/3 | BB |
| Alemtuzumab | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | P2/3 | BB |
| Horse Anti-Thymocyte Globulin | New York Medical College | P2/3 | B |
| Treosulfan | Fred Hutchinson Cancer Center | P2 | B |
| Rabbit Anti-Thymocyte Globulin | Columbia University | P2/3 | B |
+22 more in the Bone Marrow Failure Syndrome cohort
Other indications for Melphalan
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Diffuse Large B-Cell Lymphoma | — | P4 | BBB · 76 |
| Myelodysplastic/Myeloproliferative Neoplasm | — | P2/3 | BBB · 76 |
| Cancer Of Unknown Primary Site | — | P2/3 | BBB · 72 |
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. Melphalan in Bone Marrow Failure 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/melphalan-bone-marrow-failure-syndrome-88550
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