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AG-181

Development stage: Phase 1
Lead developer: Agios Pharmaceuticals
Modality: Small Molecules
Administration: Oral

Overview

AG-181 is an orally administered small molecule developed by Agios Pharmaceuticals as a phenylalanine hydroxylase (PAH) stabilizer. Its primary mechanism of action is to modulate and stabilize the PAH enzyme, which plays a critical role in the metabolism of phenylalanine. AG-181 is being investigated for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder characterized by deficient activity of PAH, leading to elevated levels of phenylalanine in the blood. As of early 2025, AG-181 has reached Phase I clinical trials focused on safety and tolerability in healthy volunteers[1][2][4][8].

Targets

PAH (Phenylalanine hydroxylase)

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