Floatz Rating
B62/100
Confidence
Indicative
Sponsor
Emory University
Modality
Small molecule
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
60High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03279094CT.gov Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease | — | Recruiting | — | — | — |
NCT03128996CT.gov Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders | — | Recruiting | — | — | — |
NCT02766465CT.gov Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503) | — | Completed | — | — | — |
NCT02867800CT.gov Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease | — | Completed | — | — | — |
NCT02186418CT.gov Gene Transfer for Patients With Sickle Cell Disease | — | Terminated | — | — | — |
NCT01049854CT.gov CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant | — | Completed | — | — | — |
NCT02435901CT.gov HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity | — | Completed | — | — | — |
NCT00745420CT.gov Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601) | — | Completed | — | — | — |
NCT00152113CT.gov Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound | — | Completed | — | — | — |
NCT00029393CT.gov Induction of Stable Chimerism for Sickle Cell Anemia | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
65High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Sickle Cell Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Melphalan (this asset) | Emory University | P3 | B · 62 |
| Glutamine | Ain Shams University | P4 | BBB |
| Hydroxyurea | St. Jude Children's Research Hospital | P4 | BB |
| Crizanlizumab | Novartis Pharmaceuticals | P4 | BB |
| Magnesium Metallicum | Medical College of Wisconsin | P3 | BB |
| Nivestim | St. Jude Children's Research Hospital | P4 | BB |
| Nepenthe | Oman Medical Speciality Board | P4 | BB |
| ARG | Tanta University | P3 | BB |
| Fentanyl | University College Dublin | P4 | BB |
+42 more in the Sickle Cell Disease 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 Sickle Cell Disease. 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-sickle-cell-disease
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