Melphalan
Paroxysmal Nocturnal Hemoglobinuria
Explore 6 assets in Paroxysmal Nocturnal Hemoglobinuria →Floatz Rating
BB66/100
Confidence
Indicative
Sponsor
Roswell Park Cancer Institute
Modality
Small molecule
Development Phase
Phase 3
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
52High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT00997386CT.gov Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States | — | 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 · Paroxysmal Nocturnal Hemoglobinuria
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Melphalan (this asset) | Roswell Park Cancer Institute | P3 | BB · 66 |
| Thiotepa | Fred Hutchinson Cancer Center | P3 | BB |
| Muromonab-Cd3 | St. Jude Children's Research Hospital | P3 | BB |
| Cyclophosphamide | Roswell Park Cancer Institute | P3 | BB |
| Fludarabine | Fred Hutchinson Cancer Center | P3 | BB |
| Rituximab | St. Jude Children's Research Hospital | P3 | BB |
| Eculizumab | AO GENERIUM | P3 | BB |
| Alemtuzumab | University of Arizona | P2 | B |
| Tacrolimus | Fred Hutchinson Cancer Center | P2 | B |
+11 more in the Paroxysmal Nocturnal Hemoglobinuria 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 Paroxysmal Nocturnal Hemoglobinuria. 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-paroxysmal-nocturnal-hemoglobinuria
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