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AG-181 · 1 trial · 1 indication
| 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...
Top 5 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase, BMN 307 |
| PTC Therapeutics, Inc. | PTCT | 1 | PHASE3 | Sepiapterin |
| Maze Therapeutics, Inc. | MAZE | 1 | PHASE2 | MZE782 |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
AG-181 is an investigational small molecule being developed for the treatment of phenylketonuria, a metabolic disorder. It is currently in Phase 1 clinical development and has not been approved for any indication. The Phase 1 study is enrolling adults with phenylketonuria in the United States and Poland.
AG-181 targets phenylalanine hydroxylase, or PAH, the enzyme that converts phenylalanine to tyrosine. In phenylketonuria, PAH activity is deficient, leading to elevated phenylalanine levels. AG-181 is designed as a small molecule directed at PAH.
AG-181 is being developed by Agios Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker AGIO. Agios is the sponsor of the Phase 1 clinical program evaluating the compound in phenylketonuria.
AG-181 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 trial is currently recruiting participants.
AG-181 is being evaluated in one active clinical trial, NCT07241234, titled A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria. The Phase 1 study is recruiting and plans to enroll 20 adults across sites in the United States and Poland.