RGX-104 is a novel oral small molecule immunotherapy in development for the treatment of solid tumors, including drug-resistant malignancies. It reverses the immunosuppressive effects of cancer by targeting the LXR/ApoE pathway that regulates the innate immune response to cancer.
Mechanism of Action
This next-generation therapy has two key effects on the innate immune system that drive tumor immunity: it depletes myeloid-derived suppressor cells (MDSCs) and stimulates dendritic cells (DCs). MDSCs block the ability of T cells to become active, while stimulated DCs are required for proper activation (priming) of T cells.
By effecting these two types of innate immune cells (MDSCs and DCs), RGX-104 can unleash tumor-targeting T cells to induce anti-tumor activity.(1,2)
RGX-104 depletes MDSCs and activates T cells in patients with cancer2
9/10 evaluable patients achieved significant MDSC depletion (>66%) on RGX-104 monotherapy
9/10 evaluable patients achieved >50% increase in activated PD-1+CD8+ T-cells
RGX-202 is an oral small molecule cancer metabolism inhibitor in development for the treatment of gastrointestinal cancer.
1) Tavazoie et al. Cell. 2018 Feb 8;172(4):825-840
2) Mita M et al. Proceedings of the 2017 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics