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HZN-457

Development stage: Phase 1
Lead developer: Amgen
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Subcutaneous

Overview

HZN-457 is a development-stage small interfering RNA (siRNA) drug candidate designed to treat gout by silencing the expression of xanthine dehydrogenase in the liver, thereby reducing uric acid production. The siRNA is conjugated to N-acetylgalactosamine (GalNAc) for targeted delivery to hepatocytes. The drug operates via RNA interference (RNAi), leading to selective knockdown of xanthine dehydrogenase mRNA and protein levels. HZN-457 was originally developed as ARO-XDH by Arrowhead Pharmaceuticals and is being further developed through an exclusive collaboration with Horizon Therapeutics. As of 2023, it has completed Phase 1 clinical trials in healthy volunteers for the treatment of gout.

Other names: HZN-457HZN457HZN 457ARO-XDH

Targets

XO (Xanthine Oxidoreductase)

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