Thiotepa
Paroxysmal Nocturnal Hemoglobinuria
Explore 6 assets in Paroxysmal Nocturnal Hemoglobinuria →Floatz Rating
BB69/100
Confidence
Indicative
Sponsor
Fred Hutchinson Cancer Center
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.
56High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04965597CT.gov Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | — | Completed | — | — | — |
NCT00566696CT.gov Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies | — | Completed | — | — | — |
NCT00186823CT.gov Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies | — | Completed | — | — | — |
NCT00587054CT.gov Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders | — | 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 |
|---|---|---|---|
| Thiotepa (this asset) | Fred Hutchinson Cancer Center | P3 | BB · 69 |
| Muromonab-Cd3 | St. Jude Children's Research Hospital | P3 | BB |
| Cyclophosphamide | Roswell Park Cancer Institute | P3 | BB |
| Melphalan | Roswell Park Cancer Institute | P3 | BB |
| Rituximab | St. Jude Children's Research Hospital | P3 | BB |
| Fludarabine | Fred Hutchinson Cancer Center | P3 | BB |
| Eculizumab | AO GENERIUM | P3 | BB |
| Tacrolimus | Fred Hutchinson Cancer Center | P2 | B |
| Alemtuzumab | University of Arizona | P2 | B |
+11 more in the Paroxysmal Nocturnal Hemoglobinuria cohort
Other indications for Thiotepa
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Graft Versus Host Disease | — | P4 | BBB · 73 |
| Myelodysplastic/Myeloproliferative Neoplasm | — | P2/3 | BBB · 72 |
| Juvenile Myelomonocytic Leukemia | — | P3 | 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. Thiotepa 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/thiotepa-paroxysmal-nocturnal-hemoglobinuria
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