Drug intelligence / Profile preview

AX-024

Development stage
Phase 1
Lead developer
Artax Biopharma
Modality
Small Molecules
Administration
Oral
01

Overview

AX-024 is an orally available, first-in-class small molecule inhibitor developed to modulate T cell activation by targeting the T cell receptor (TCR) signaling pathway. It was originally designed to inhibit the interaction between the adaptor protein Nck and the CD3ϵ subunit of the TCR complex, thereby selectively suppressing TCR-triggered T cell activation without affecting B cell or innate immune responses[2][5]. Although initial studies suggested that AX-024 binds to the SH3.1 domain of Nck and blocks its interaction with CD3ϵ, later research indicated that its mechanism may involve additional targets in T cells and likely exhibits polypharmacology[1][8]. Preclinical models demonstrated protective effects in autoimmune diseases such as multiple sclerosis, psoriasis, allergic asthma[5], and more recently intracerebral hemorrhage (ICH), where it reduced neuroinflammation and improved outcomes by inhibiting antigen-specific T-cell responses without increasing infection risk[6][7]. The drug was developed by Artax Biopharma and has completed phase I clinical trials in healthy volunteers; however, no recent development has been reported for asthma or multiple sclerosis indications[3].

02

Targets

NCK1 SH3.1 (NCK1 SH3 domain 1)

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