US-based clinical stage bio-pharmaceutical company Rgenix has begun its Phase Ia/b study of RGX-104 to treat advanced solid malignancies and lymphoma.

RGX-104 is an orally administered, bioavailable, small molecule immunotherapy developed to target the liver X receptors (LXR) to activate expression of the ApoE protein.

It affects both myeloid-derived suppressor cells (MDSCs), as well as tumour blood vessels. MDSCs are known to inhibit T-cells and other immune system cells from attacking tumours.

The study will be divided into an initial dose-escalation stage, which will determine the pharmacokinetic, pharmacodynamic, biomarker and safety of an optimal biological dose RGX-104 while treating patients with advanced solid malignancies and lymphoma.

"RGX-104 represents the first LXR-ApoE targeting drug in clinical development for cancer."

The expansion stage of the study will assess the preliminary efficacy of RGX-104, while examining patients with at least four types of cancers.

Rgenix co-founder and CEO Masoud Tavazoie said: “RGX-104 represents the first LXR-ApoE targeting drug in clinical development for cancer.

“Because of its ability to inhibit tumour growth and metastasis as a single agent in drug-resistant cancer types via a novel mechanism, RGX-104 has the potential to transform the treatment landscape for several common cancer types, addressing urgent unmet needs in oncology.”

RGX-104 has demonstrated efficacy in reversing the immunosuppressive and pro-angiogenic effects of cancers by activating ApoE expression in cancer and highly specific circulating immune cells, which finally leads to killing these cells and blood vessels within the tumour microenvironment.