Rgenix is a clinical-stage biopharmaceutical company developing first-in-class drugs that target key pathways in cancer progression. Using a discovery platform developed by Rgenix's founding scientists at Rockefeller University, we have discovered several novel cancer targets that drive tumor growth and cancer progression. These targets regulate key components of the tumor micro-environment including immune cells and cancer metabolism pathways.

Our discovery platform - which can be applied to virtually any tumor type - has yielded novel therapeutic targets for several high-unmet need cancer types to date. We aim to develop effective therapies against these targets that will provide durable and meaningful responses for cancer patients that lack effective therapies.

Our lead drug candidate (RGX-104) is a small molecule immunotherapy that targets the immune system to treat cancer progression. RGX-104 has obtained FDA Orphan Drug Designation in several solid tumor types of high-unmet need and is currently being evaluated in a Phase 1 clinical trial in cancer patients.


Science underlying Rgenix’s technology and pipeline has been published in leading scientific journals.


Rgenix has multiple therapeutic programs in development, impacting several prevalent cancer types

Rgenix's product pipeline addresses cancers diagnosed in more than 500,000 patients annually in the US, including drug-resistant cancer types. These cancers result in more than 250,000 deaths per year in America, with 5-year survival rates often as low as 10%-20% for patients with metastatic disease. Rgenix has exclusive worldwide rights to each of its programs below.


RGX-104 is an orally bioavailable small molecule immunotherapy that targets LXR. Rgenix in-licensed RGX-104 and other LXR intellectual property from GlaxoSmithKline as part of a world-wide exclusive licensing agreement. RGX-104 depletes both myeloid-derived suppressor cells (MDSCs) as well as tumor blood vessels. MDSCs block T-cells and other cells of the immune system from attacking tumors, resulting in immune evasion. Treatment with RGX-104 reverses immune evasion, resulting in robust anti-tumor activity in established animal models of various cancer types, including drug-resistant tumors. In pre-clinical models, RGX-104 has demonstrated remarkable single-agent activity as well as synergy with approved therapies, such as immune check-point inhibitors. RGX-104 is currently being evaluated in a Phase 1 clinical study. For more information about the clinical trial visit: http://clinicaltrials.gov/ct2/show/NCT02922764


RGX-202 is a small molecule that inhibits a novel cancer metabolism pathway involved in supplying energy to cancer cells. The target of RGX-202 is genetically amplified in several prevalent cancer types, including colon cancer, lung cancer and lymphoma. RGX-202 has demonstrated anti-tumor activity in several tumor models in pre-clinical studies, both as a single agent as well as in combination with standard-of-care therapies. IND-enabling studies for RGX-202 have been initiated in preparation for clinical development.


RGX-014/019 are two monoclonal antibodies that target independent components of a key pathway that drives tumor progression and metastasis of several cancer types, including triple-negative breast cancer.


Lung Cancer


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Colorectal Cancer


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Trip Neg Breast Cancer


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Addressable Cancers


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Rgenix’s management comprises biotech professionals with deep scientific, clinical and operational expertise.


Jan 6, 2017

Rgenix Announces Treatment of First Patient in Phase 1 Trial of RGX-104, its Lead Immuno-Oncology Therapeutic

Jan 6, 2017

Rgenix Announces Treatment of First Patient in Phase 1 Trial of RGX-104, its Lead Immuno-Oncology Therapeutic   

First-in-Class Cancer Therapeutic Targets the Liver X Receptor (LXR)-ApoE Pathway Identified Using Rgenix’s Discovery Platform

NEW YORK CITY, N.Y., January 6, 2017 – Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics using its unique target discovery platform, today announced treatment of the first patient with its lead candidate RGX-104 in a Phase 1a/b study.  The 80-patient study consists of an initial dose-escalation stage involving patients with advanced solid malignancies and lymphoma, in which an optimal biological dose is being defined using pharmacokinetic, pharmacodynamic, biomarker and safety studies. This will be followed by an expansion stage in at least four cancer types, in which detection of preliminary efficacy signals will be determined.  

RGX-104 is an orally administered small molecule that selectively targets the nuclear receptor LXR to activate the expression of the ApoE protein that becomes silenced in human cancers as they grow, become invasive, and metastasize.  ApoE silencing is related to reduced survival in cancer patients. 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 who demonstrated that the pathway regulates the ability of cancers to evade the immune system and recruit blood vessels. 

Rgenix’s scientists showed that RGX-104 reverses the immune suppressive and pro-angiogenic effects of cancers by potently activating ApoE expression in cancer and highly specific circulating immune cells, leading to the elimination of these immunosuppressive cells and blood vessels in the tumor microenvironment. RGX-104 treatment confers robust anti-cancer activity and suppression of metastases in a wide range of well-established animal models of multiple human cancer types, including drug-resistant malignancies.  In addition to its profound and diverse actions as a single agent, RGX-104 blocks resistance to immune checkpoint inhibitors, providing a rationale to combine RGX-104 with these therapies in patients who either do not initially respond to immune checkpoint inhibitor therapy alone, or those who at first benefited, but soon after developed resistance.  RGX-104 was granted Orphan Drug Designation by the FDA for the treatment of several cancer types of high unmet need.

“RGX-104 represents the first LXR-ApoE targeting drug in clinical development for cancer,” said Masoud Tavazoie, MD, PhD, Chief Executive Officer and co-founder of Rgenix.  He added, “Because of its ability to inhibit tumor 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 cancers types, addressing urgent unmet needs in oncology.” 

Dr. Michael Postow, a member of the Melanoma and Immunotherapeutics Service at Memorial Sloan Kettering Cancer Center, one of the RGX-104 study sites, commented, "We are excited about RGX-104's potential to treat a broad array of cancers given its unique effect on the immune system and strong pre-clinical activity both as a single agent and in combination with a checkpoint inhibitor.”

The same discovery platform used to identify the ApoE pathway in cancer has been used to discover several other key regulators of cancer progression that Rgenix is targeting with first-in-class small-molecule and antibody drug candidates as part of its novel therapeutics pipeline.

For more information about the clinical trial visit: https://clinicaltrials.gov/ct2/show/NCT02922764

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. Using a miRNA based target discovery platform developed by Rgenix’s founders at The Rockefeller University, the company is pursuing several first-in-class drug candidates to treat cancers of high unmet need. 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.

Media Contact:


Marion Janic




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Jun 15, 2016

Rgenix Announces $33 Million Series B Financing to Develop First-in-Class Cancer Therapeutics

Jun 15, 2016

Novo A/S and Sofinnova Partners are leading this latest financing round

NEW YORK, New York ― June 15, 2016 ― Rgenix, a cancer therapeutics company developing first-in-class drugs targeting novel cancer pathways, announced a $33 million Series B financing led by Novo A/S and Sofinnova Partners, with participation from existing investors including Partnership Fund for New York City, Alexandria Venture Investments, and Conegliano Ventures LP. The financing will support clinical development of Rgenix’s lead drug candidates, RGX-104 and RGX-202, as well as further development of its therapeutics pipeline. 

“We are thrilled to have attracted top-tier investors to advance development of our novel cancer therapeutics,” said Masoud Tavazoie, M.D. Ph.D., Chief Executive Officer and co-founder of Rgenix. “This financing validates the potential of our lead immunotherapy RGX-104, which will be entering clinical trials this fall, and also demonstrates the strength of our discovery platform, developed in the laboratory of Rgenix co-founder Dr. Sohail Tavazoie at The Rockefeller University. The funding will enable our team to deliver an innovative therapy to cancer patients while simultaneously pushing forward our pipeline of other novel drug candidates.”  

Antoine Papiernik, Managing Partner at Sofinnova Partners, commented: “We are very excited to back such a high quality team at Rgenix. We also believe that RGX-104 could revolutionize treatment to cancer patients that today lack effective therapies.”

Despite recent advances in cancer therapy, most patients will eventually succumb to their disease due to drug resistance and immune evasion. RGX-104 is a small molecule that reverses immune evasion and drug resistance by targeting immunosuppressive cells in the tumor microenvironment via a novel pathway, resulting in strong anti-tumor activity in several drug-resistant cancer types in pre-clinical models, both as a single agent and in combination with approved immunotherapies such as PD-1 inhibitors. The target of RGX-104 was discovered using Rgenix’s miRNA platform that has yielded several new cancer targets across multiple prevalent cancer types.

“We are excited to support Rgenix’s novel approach to treating cancers of high unmet need,” said Nilesh Kumar, Senior Principal of Novo Ventures*. “Rgenix has an exciting platform founded on strong science from The Rockefeller University; the lead program is a first in class opportunity addressing a key mechanism in tumor immunosuppression in various cancer types.”

In connection with the financing, Nilesh Kumar of Novo Ventures and Antoine Papiernik of Sofinnova Partners will join the Rgenix Board of Directors together with existing members, including Executive Chairman Eric Rowinsky, M.D., Masoud Tavazoie, M.D. Ph.D., Nancy Chang, Ph.D., and Saeed Tavazoie, Ph.D.

Jun 15, 2016

Rgenix Raises $33M, Preps Cancer Trial For Drug Shelved By GSK

Jun 15, 2016

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Jun 15, 2016

Rottweiler tested, Novo and Sofinnova back $33M round for Rgenix

Jun 15, 2016

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Aug 31, 2015

In New York, Rgenix Rises From Rockefeller and Takes Aim at Cancer

Aug 31, 2015

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