Drug intelligence / Profile preview

ATX-GD-59

Development stage
Phase 1
Lead developer
Worgpharma
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Peptides
Administration
Intradermal
01

Overview

ATX-GD-59 is an investigational, peptide-based immunotherapy designed for the treatment of Graves' disease. It consists of a lyophilized equimolar mixture of two synthetic peptides (5D-K1 and 9B-N) derived from sequences in the human thyrotropin receptor (TSHR). These peptides act as apitopes—antigen-processing independent epitopes—that bind with high affinity to HLA-DR molecules on immature dendritic cells, aiming to restore immune tolerance specifically to TSHR and thereby reduce pathogenic autoantibody production. The drug is administered intradermally and has shown promising safety and tolerability in Phase I clinical trials, with preliminary evidence suggesting efficacy in normalizing thyroid hormone levels in patients with Graves' hyperthyroidism. Unlike conventional therapies that broadly suppress the immune system or ablate thyroid tissue, ATX-GD-59 targets the underlying autoimmune mechanism by modulating antigen-specific T cell responses[1][2][3][4][6][7][8].

Other names
thyroid-stimulating hormone receptor apitopesTSHR apitopesApitopes derived from TSHR
02

Targets

MHC II (Major histocompatibility complex class II receptor)TSHR (Thyroid-stimulating hormone receptor)

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