Drug Profile

AG 181

Alternative Names: AG-181; PAH - Agios Pharmaceuticals

Latest Information Update: 21 Jan 2026

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At a glance

Originator Agios Pharmaceuticals
Class Small molecules
Mechanism of Action Phenylalanine hydroxylase modulators

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes
Available For Licensing Yes

Highest Development Phases

Phase I Phenylketonuria

Most Recent Events

12 Jan 2026 Agios Pharmaceuticals plans to initiate a Phase Ib proof-of-mechanism trial in patients with Phenylketonuria (PKU) in H1 2026
21 Nov 2025 Agios Pharmaceuticals plans a phase I trial for Phenylketonuria (PO) in March 2026 (NCT07241234) (CTIS2025-522630-31-00)
09 Oct 2025 Agios Pharmaceuticals completes a phase-I trial in Phenylketonuria (In volunteers) in USA (PO) (NCT06286033)

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