Rgenix Announces Publication In Cell Demonstrating Activation of LXR/ApoE with RGX-104 Enhances Antitumor Immunity

LXR/ApoE Pathway Identified as Key Regulator of Innate Immune Suppression in Cancer


New York, NY – January 11, 2018– Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today the publication of clinical and pre-clinical results by research collaborators at Rgenix and The Rockefeller UniversityThe results of the collaboration, published in the January 11 online issue of Cell, reveal that the Liver-X Receptor/Apolipoprotein E (LXR/ApoE) pathway regulates anti-tumor immunity via effects on myeloid-derived suppressor cells (MDSCs).

MDSCs are an immunosuppressive cell population that have been found to be circulating at high levels in cancer patients who are also commonly found to be non-responders to immunotherapy. RGX-104 was able to deplete MDSCs both in cancer patients participating in the dose escalation portion of the Phase 1a/b trial and in mice. This resulted in robust activation of relevant T cell populations in both settings.

“We are pleased to have this opportunity to share our latest research results in Cell that illustrate the effects our first-in-class RGX-104 compound has on one of the key cells that are responsible for suppressing the immune response in cancer patients,” said Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer and co-founder of Rgenix. “We believe RGX-104 represents a novel strategy for stimulating anti-tumor immunity, and this data cements this belief and furthers our resolve to continue developing this compound both as monotherapy as well as in combination with checkpoint inhibitor therapy.”

Sohail Tavazoie, M.D., Ph.D., Leon Hess Associate Professor and Head of Elizabeth and Vincent Meyer Laboratory of Systems Cancer Biology at the Rockefeller University who was the senior-author of the study as well as co-founder of Rgenix, added: “The data our research has generated at this stage is exciting and supports our perspective that RGX-104 has the potential to modulate the immune response in a broad array of cancers. We will continue to progress in our research and our pursuit of unique treatments for patients affected by cancers with a high unmet medical need.”

The paper, titled “LXR/ApoE Activation Restricts Innate Immune Suppression in Cancer”, was published today and is available online.

About Rgenix

Rgenix, Inc., is a privately-held clinical-stage biopharmaceutical company focused on the discovery and development of novel cancer drugs that target key pathways in cancer progression. The company is pursuing several first-in-class drug candidates to treat cancers of high unmet need. Rgenix identifies novel cancer targets using a microRNA based target discovery platform originally developed by Rgenix’s scientific co-founders at The Rockefeller University and now exclusively licensed to Rgenix. The company brings together distinguished scientific founders, a seasoned Board, and a leadership team comprised of experienced drug developers. The company is funded by leading biotechnology investors, including Novo A/S, Sofinnova Partners, and Alexandria Venture Investments. For more information, please visit www.rgenix.com.

About RGX-104

RGX-104 is an orally-administered potent small molecule agonist of the Liver X Receptor (LXR) that is currently being evaluated in clinical studies. Activation of the LXR-ApoE pathway by RGX-104 stimulates the innate immune response in cancer via depletion of myeloid-derived suppressor cells and activation of dendritic cells, leading to stimulation of T cells and anti-tumor immunity. LXR activation also blocks the ability of tumors to recruit blood vessels. These combined effects result in suppression of tumor growth and metastasis in a broad array of animal tumor models. The LXR-ApoE pathway was originally identified as a cancer target using a novel microRNA-based discovery platform developed by Rgenix’s scientific co-founders at The Rockefeller University.

Rgenix is conducting a Phase 1a/b clinical trial of RGX-104 in patients with advanced solid malignancies and lymphoma—for more information about the clinical trial, please visit: https://clinicaltrials.gov/ct2/show/NCT02922764.

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