Developing new cancer therapies and improving the quality of patients’ lives is at the heart of everything we do. By focusing on novel first-in-class drug development, we aim to develop the therapies of the future. Our goal is to create effective therapies that provide meaningful and durable benefit to cancer patients.
We are actively recruiting patients for a phase 1, first-in-human, dose escalation and expansion study of RGX-104, an oral small molecule targeting the liver X receptor (LXR), as a single agent and in combination with nivolumab.
Clinical Trial Format
During the dose escalation stage, multiple doses and schedules of orally administered RGX-104 with or without nivolumab (single agent or combination therapy) will be evaluated in patients with advanced solid tumors and lymphoma (i.e., locally advanced and unresectable, or metastatic) who have had progressive disease (PD) on available standard systemic therapies or for which there are no standard systemic therapies of relevant impact.
In the expansion stage of the study, additional patients with epithelial ovarian carcinoma (EOC), melanoma, non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), renal cell cancer (RCC), bladder cancer (BLC), or triple negative breast cancer (TNBC) will be treated at the MTD (or maximum tested dose if no MTD is identified, or dose below the MTD if there is evidence suggesting a more favorable risk/benefit profile).
This stage will provide further characterization of the safety, efficacy, PK, and pharmacodynamics, including biomarkers of immunologic activity and LXR target activation, of RGX-104 as a single agent (EOC) and in combination with nivolumab (melanoma, NSCLC, SCLC, RCC, BLC, and TNBC).
RGX-104 is a small molecule immunotherapy that activates LXR, resulting in depletion of both myeloid-derived suppressor cells (MDSCs) as well as tumor blood vessels. MDSCs block the ability of T-cells and other cells of the immune system from attacking tumors.
- Epithelial Ovarian Cancer (EOC)
- Non-Small Cell Lung Cancer (NSCLC)
- Small Cell Lung Carcinoma (SCLC)
- Renal Cell Carcinoma (RCC)
- Bladder Cancer (BLC)
- Triple-Negative Breast Cancer (TNBC)
- Metastatic, locally advanced or unresectable cancer
- Any type of a malignant solid tumor
RGX-104 is an investigational small molecule immunotherapy that activates LXR, resulting in depletion of both myeloid-derived suppressor cells (MDSCs) as well as tumor blood vessels. MDSCs block the ability of T-cells and other cells of the immune system from attacking tumors.