Sepiapterin
Hyperphenylalaninemia Due To Tetrahydrobiopterin Deficiency
Floatz Rating
CCC52/100
Confidence
Indicative
Sponsor
PTC Therapeutics
Modality
Small molecule
Development Phase
Phase 1/2
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
37Moderate confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03519711CT.gov A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia | — | 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 · Hyperphenylalaninemia Due To Tetrahydrobiopterin Deficiency
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Sepiapterin (this asset) | PTC Therapeutics | P1/2 | CCC · 52 |
| (6S)-L-ERYTHRO-TETRAHYDROBIOPTERIN | University of Massachusetts, Amherst | P1 | CC |
| Phenoptin | BioMarin Pharmaceutical | P2 | NR |
| Sapropterin Dihydrochloride | BioMarin Pharmaceutical | Unk | NR |
Other indications for Sepiapterin
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Phenylketonuria | — | P3 | B · 62 |
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. Sepiapterin in Hyperphenylalaninemia Due To Tetrahydrobiopterin Deficiency. 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/sepiapterin-hyperphenylalaninemia-due-to-tetrahydrobiopterin-deficiency
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