Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Paul Szabolcs
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 |
|---|---|---|---|---|---|
NCT01962415CT.gov Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | — | 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 · Inborn Errors Of Metabolism
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Hydroxyurea (this asset) | Paul Szabolcs | P2 | CCC · 53 |
| Nexiguran Ziclumeran | Intellia Therapeutics | P3 | B |
| Mgta-456 | Magenta Therapeutics, Inc. | P2 | B |
| Cald Hr-D | Masonic Cancer Center, University of Minnesota | P2 | CCC |
| Alemtuzumab | Paul Szabolcs | P2 | CCC |
| Hpc, Cord Blood | Memorial Sloan Kettering Cancer Center | P2 | CCC |
| Cyclosporin A | Memorial Sloan Kettering Cancer Center | P2 | CCC |
| Melphalan | University of California, San Francisco | P2 | CCC |
| Clofarabine | Memorial Sloan Kettering Cancer Center | P2 | CCC |
+7 more in the Inborn Errors Of Metabolism cohort
Other indications for Hydroxyurea
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Beta-Thalassemia Major | — | P4 | BBB · 77 |
| Sickle Cell Disease | — | P4 | BB · 71 |
| Thalassemia | — | P4 | BB · 70 |
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. Hydroxyurea in Inborn Errors Of Metabolism. 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/hydroxyurea-inborn-errors-of-metabolism
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