Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Ionis Pharmaceuticals, Inc.
Modality
nucleic_acid
Development Phase
Phase 2
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
38Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT04059406CT.gov Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) | — | Terminated | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Beta-Thalassemia Intermedia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Sapablursen (this asset) | Ionis Pharmaceuticals, Inc. | P2 | CCC · 53 |
| Sodium 2,2 Dimethylbutyrate | Boston University | P2 | B |
| Mitapivat | Agios Pharmaceuticals, Inc. | P3 | B |
| Hydroxyurea | Shiraz University of Medical Sciences | P2 | B |
| L-Carnitine | Shiraz University of Medical Sciences | P2 | B |
| Vit-2763 Once A Day | Vifor (International) Inc. | P2 | B |
| Vit-2763 Twice A Day | Vifor (International) Inc. | P2 | B |
| Exerenibart | Regeneron Pharmaceuticals | P2 | CCC |
| Human Apotransferrin | Prothya Biosolutions | P2 | CCC |
+3 more in the Beta-Thalassemia Intermedia cohort
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. Sapablursen in Beta-Thalassemia Intermedia. 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/sapablursen-beta-thalassemia-intermedia
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