Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07241234 | A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria | PHASE1 | RECRUITING | 20 | — | — | Apr 17, 2026 | Jan 17, 2028 | May 29, 2026 | 2 | United States, Poland |
| Arm | Type | Description |
|---|---|---|
| Cohort 1: AG-181 | EXPERIMENTAL | Subjects will receive AG-181 from Day 1 to Day 28. |
| Cohort 2 (Optional Cohort): AG-181 | EXPERIMENTAL | Subjects will receive AG-181. |
| Name | Type | Description |
|---|---|---|
| AG-181 | DRUG | AG-181 film-coated tablets |
Key Inclusion Criteria: * Diagnosis of PKU, defined as documented presence of 2 mutant alleles in the phenylalanine hydroxylase (PAH) gene, of which at least 1 is the R408W mutation, as determined during Screening per the genotyping performed by the study central genotyping laboratory. * At least 1...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase, BMN 307 |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923, Sepiapterin |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | — | HMI-102 |