Drug intelligence / Profile preview

ATX-F8-117

Development stage
Unknown
Lead developer
Worgpharma
Modality
Peptides
Administration
Subcutaneous
01

Overview

ATX-F8-117 is a peptide-based antigen-specific immunotherapy composed of two optimized “apitopes” (P1 and P17) derived from immunodominant CD4 T‑cell epitopes of human coagulation factor VIII, designed to re‑establish immune tolerance to FVIII and thereby prevent or reduce the formation of neutralizing anti‑FVIII antibodies in hemophilia A patients receiving FVIII replacement therapy.[1][2] The peptides are engineered for solubility and direct binding to multiple HLA‑DRB1 alleles without prior antigen processing, promoting presentation by steady‑state dendritic cells and the induction or expansion of tolerogenic Tr1‑like regulatory T cells that suppress FVIII‑specific helper responses.[1][2] In HLA‑DR2 transgenic mouse models, subcutaneous administration of ATX‑F8‑117 using a dose‑escalation regimen markedly suppresses FVIII‑specific T‑cell proliferation, reduces inflammatory cytokine production, and significantly decreases both total anti‑FVIII IgG and inhibitor titers, supporting its development as a targeted tolerance‑inducing therapy for patients with or at risk of FVIII inhibitors in hemophilia A.[1][2][8][14]

Other names
FVIII apitope peptide cocktail P1 P17
02

Targets

Factor VIII-specific B-cell responseHLA-DRB1 (Human leukocyte antigen DR beta chain)Factor VIII-specific T-cell receptor

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