Vatiquinone
Friedreich Ataxia
Floatz Rating
BB66/100
Confidence
Indicative
Sponsor
PTC Therapeutics
Modality
UNKNOWN
Development Phase
Phase 3
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
54High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05515536CT.gov A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | — | Active Not Recruiting | — | — | — |
NCT05485987CT.gov A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | — | Completed | — | — | — |
NCT04577352CT.gov A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia | — | 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 · Friedreich Ataxia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Vatiquinone (this asset) | PTC Therapeutics | P3 | BB · 66 |
| Idebenone | Santhera Pharmaceuticals | P3 | A |
| Interferon Γ-1B | Amgen | P3 | BB |
| Omaveloxolone | Biogen | P3 | BB |
| rt001 | Biojiva LLC | P3 | BB |
| Trans-Resveratrol | Murdoch Childrens Research Institute | P2 | BB |
| Citalopram | University of Colorado, Denver | P4 | BB |
| Deferiprone | ApoPharma | P2 | BB |
| L-Methionyl-L-Tyrosylglycyl-L-Arginyl-L-Lysyl-L-Lysyl-L-Arginyl-L-Arginyl-L-Glutaminyl-L-Arginyl-L-Arginyl-L-Arginylglycylglycine Fusion Protein With Frataxin (Human Isoform 1 Precursor) | Larimar Therapeutics, Inc. | P2 | BB |
+30 more in the Friedreich Ataxia cohort
Other indications for Vatiquinone
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Mitochondrial Disease | — | P3 | NR |
| MELAS Syndrome | — | P2/3 | NR |
| Leigh Syndrome | — | P2/3 | NR |
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.
Citation
Floatz Terminal. Vatiquinone in Friedreich Ataxia. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 13, 2026. https://terminal.floatz.ai/assets/vatiquinone-friedreich-ataxia
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