Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AG-181 · 2 trials · 2 indications
| 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. |
| Part 1: Single Ascending Dose (SAD) | EXPERIMENTAL | Participants will receive a range of doses of AG-181 or placebo, orally, once on Day 1. AG-181 will be given under fasted conditions. |
| Part 2: Multiple Ascending Dose (MAD) | EXPERIMENTAL | Participants will receive a range of doses of AG-181 or placebo twice daily (BID) for 13 days and a single dose on Day 14 under fasted conditions. |
| Part 3: Food Effect - Sequence 1: AB | EXPERIMENTAL | Participants will receive single oral dose of AG-181 on Day 1 of Period 1 under fasted condition (A) followed by single oral dose of AG-181 on Day 1 of Period 2 under fed (high fat meal) condition (B). Each period will be separated by a washout period of 1 week. |
| Part 3: Food Effect - Sequence 2: BA | EXPERIMENTAL | Participants will receive a single oral dose of AG-181 on Day 1 of Period 1 under fed (high fat meal) condition (B) followed by single oral dose of AG-181 on Day 1 of Period 2 under fasted condition (A). Each period will be separated by a washout period of 1 week. |
| Name | Type | Description |
|---|---|---|
| AG-181 | DRUG | AG-181 film-coated tablets |
| Placebo | DRUG | AG-181 matched-placebo 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...
AG-181 is an investigational small molecule being studied for the treatment of phenylketonuria (PKU). It is also being evaluated in healthy volunteers to assess its safety and tolerability. The drug is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
AG-181 is being developed by Agios Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AGIO. The company is conducting clinical trials to evaluate the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with 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 trials are designed to evaluate safety, tolerability, and how the drug is processed in the body.
AG-181 is being studied in two Phase 1 clinical trials. The first, NCT06286033, is a completed single and multiple ascending dose and food effect study in healthy volunteers. The second, NCT07241234, is an ongoing study in patients with phenylketonuria, currently recruiting participants in the United States and Poland.
AG-181 is the primary name for this investigational drug. No alternative names have been reported for it in clinical trial registries. It is distinct from other compounds with similar names, and researchers refer to it consistently as AG-181 across studies.