Floatz Rating
CCC53/100
Confidence
Indicative
Sponsor
Universidade do Porto
Modality
Small molecule
Development Phase
Phase 2
Status
Dormant
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
38High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT03820804CT.gov Nutritional Status in Phenylketonuria | — | Completed | — | — | — |
NCT01977820CT.gov Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria | — | Terminated | — | — | — |
NCT01541397CT.gov Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy | — | Terminated | — | — | — |
NCT00986973CT.gov Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy | — | Completed | — | — | — |
NCT00964236CT.gov The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) | — | 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 · Phenylketonuria
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| (6S)-L-ERYTHRO-TETRAHYDROBIOPTERIN (this asset) | Universidade do Porto | P2 | CCC · 53 |
| Sapropterin Dihydrochloride | Sohag University | P4 | A |
| Phenylase | BioMarin Pharmaceutical | P4 | A |
| Ravpal-Peg | BioMarin Pharmaceutical | P2 | BB |
| Repinatrabit | Otsuka Pharmaceutical Development & Commercialization, Inc. | P3 | BB |
| Escherichia Coli Strain Synb1618 | Synlogic | P2 | BB |
| L-Carnitine | Mansoura University | P4 | BB |
| Bmn 165 | BioMarin Pharmaceutical | P3 | B |
| Sepiapterin | PTC Therapeutics | P3 | B |
+18 more in the Phenylketonuria cohort
Other indications for (6S)-L-ERYTHRO-TETRAHYDROBIOPTERIN
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Lesch-Nyhan Syndrome | — | P2/3 | B · 56 |
| Portal Hypertension | — | P2/3 | B · 56 |
| Cirrhosis Of Liver | — | P2/3 | CCC · 53 |
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. (6S)-L-ERYTHRO-TETRAHYDROBIOPTERIN in Phenylketonuria. 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/6s-l-erythro-tetrahydrobiopterin-phenylketonuria
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