Floatz Rating
B62/100
Confidence
Indicative
Sponsor
PTC Therapeutics
Modality
Small molecule
Development Phase
Phase 3
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
47High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03179631CT.gov Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy | — | Completed | — | — | — |
NCT02090959CT.gov An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy | — | Terminated | — | — | — |
NCT01826487CT.gov Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | — | 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 · Musculoskeletal System Disorder
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Ataluren (this asset) | PTC Therapeutics | P3 | B · 62 |
| (±)-2-(O-Chlorophenyl)-2-(Methylamino)Cyclohexanone | Stanford University | P4 | BB |
| Tirzepatide | Pennington Biomedical Research Center | P4 | BB |
| Testosterone Undecylate | Alliance for Clinical Trials in Oncology | P3 | BB |
| Duloxetine | SWOG Cancer Research Network | P3 | BB |
| Naproxen | Gary Morrow | P3 | BB |
| L-Ofloxacin | Children's Oncology Group | P3 | BB |
| Zoledronate | Alliance for Clinical Trials in Oncology | P3 | B |
| Amoxicillin | Bart Koes | P3 | B |
+10 more in the Musculoskeletal System Disorder cohort
Other indications for Ataluren
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Cystic Fibrosis | — | P4 | BB · 68 |
| Becker Muscular Dystrophy | — | P3 | BB · 64 |
| Duchenne Muscular Dystrophy | — | P3 | BB · 64 |
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. Ataluren in Musculoskeletal System Disorder. 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/ataluren-musculoskeletal-system-disorder
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