Floatz Rating
CC46/100
Confidence
Indicative
Sponsor
Assiut University
Modality
Small molecule
Development Phase
Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
32Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05777733CT.gov NAC Effect on Iron Overload and Blood Transfusion in β-thalassemia Major | — | Unknown | — | — | — |
NCT04260516CT.gov The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major | — | Completed | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
89High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Beta-Thalassemia Major
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Acetylcysteine (this asset) | Assiut University | P1 | CC · 46 |
| Deferasirox | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Deferiprone | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Hydroxyurea | Bahria University | P4 | BBB |
| Desferrioxamine B | Pakistan Blood and Marrow Transplant (PBMT) Group | P4 | BBB |
| Tacrolimus | M.D. Anderson Cancer Center | P4 | BBB |
| Fludarabine | M.D. Anderson Cancer Center | P4 | BBB |
| Busulfanum | M.D. Anderson Cancer Center | P4 | BBB |
| Cyclophosphamide | M.D. Anderson Cancer Center | P4 | BB |
+42 more in the Beta-Thalassemia Major cohort
Other indications for Acetylcysteine
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Idiopathic Pulmonary Fibrosis | — | P4 | BBB · 74 |
| Schizophreniform Disorder | — | P4 | BBB · 74 |
| Pulmonary Fibrosis | — | P3 | 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. Acetylcysteine in Beta-Thalassemia Major. 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/acetylcysteine-beta-thalassemia-major
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