Rgenix is a privately-held clinical-stage biopharmaceutical company developing first-in-class drugs that target key pathways in cancer progression.

310 E 67th St, Suite 1-12‎ New York, NY
(646) 856-9261


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 is an investigational drug candidate. In pre-clinical models RGX-104 demonstrates significant single-agent activity as well as additivity with approved therapies, such as immune check-point inhibitors. In an ongoing phase 1/2 clinical trial in advanced cancer patients, RGX-104 has demonstrated immune-stimulatory and anti-tumor activity as a single agent.

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.



RGX-019 is a monoclonal antibody that targets key pathway that drives tumor progression and metastasis of several cancer types, including triple-negative breast 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