LTX-401, targets the Golgi apparatus, and is an oncolytic amino acid derivative.

LTX-401 displays the highest cytotoxic activity against the human malignant melanoma cell line MDA-MB-435S (13.5 μM), and is least active against the human hepatocellular carcinoma cell line HEPG2 (35.4 μM). For the remaining cell lines, LTX-401 exhibits similar IC50 values, varying slightly within the range of 19-32 μM. No in vitro hemolytic activity against RBCs is observed using the same concentrations required for the induction of cell death in cancer cell lines. A 50% hemolysis is observed using higher concentrations of LTX-401 (400 μg/mL=1087 μM)[1].LTX-401, an oncolytic amino acid derivative, targets the Golgi apparatus[2].?LTX-401 effectively reduces the viability of several tumor cell lines in vitro, with a similar degree of cytotoxicity against non-malignant cell lines such as HUV-EC-C endothelial cells, HaCat keratinocytes and MRC-5 fibroblasts.

The majority ?of the animals demonstrate a complete and lasting tumor regression after intratumoral treatment with LTX-401.?Samples are collected from selected animals in the treatment group and control group on days 2 and 7 post-treatment with a single i.t injection.?The immunolabelling of tumor tissue with anti-CD3 antibody reveals that a majority of the infiltrating cells are CD3+ T cells, whereas tumors injected with vehicle only exhibit tumors with a viable tumor tissue, with minimal necrosis and few lymphocytes[1].