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)
Friedreich Ataxia
Floatz Rating
BB64/100
Confidence
Indicative
Sponsor
Larimar Therapeutics, Inc.
Modality
protein_peptide
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
49Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06447025CT.gov An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia | — | Recruiting | — | — | — |
NCT05579691CT.gov A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia | — | Completed | — | — | — |
NCT04519567CT.gov Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia | — | Completed | — | — | — |
NCT04176991CT.gov Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia | — | Completed | — | — | — |
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 · Friedreich Ataxia
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| 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) (this asset) | Larimar Therapeutics, Inc. | P2 | BB · 64 |
| 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 |
| Vatiquinone | PTC Therapeutics | P3 | BB |
+30 more in the Friedreich Ataxia 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.
Citation
Floatz Terminal. 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) 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/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-friedreich-ataxia
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