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

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

Pipeline

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

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

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

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

 

diagnosed in US/ year

 

Colorectal Cancer

 

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Trip Neg Breast Cancer

 

diagnosed in US/ year

 

Addressable Cancers

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diagnosed in US/ year

Team

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

News

Nov 17, 2017

Rgenix Reports Phase I Data for Cancer Compound RGX-104

Nov 17, 2017

Rgenix Inc. (New York, N.Y.) reported preliminary data from a Phase Ia/Ib trial showing that oral RGX-104 led to 4 cases of stable disease among 12 evaluable patients with advanced solid malignancies and lymphoma. To read the full article, please click here.

Nov 17, 2017

Liver X Factor

Nov 17, 2017

How Rgenix’s Liver X Agonist Could Help Overcome Checkpoint Resistance in Cancer. To read the full article, please click here.

Oct 30, 2017

Rgenix Reports Preliminary Phase 1a/b Clinical Data Demonstrating Immune-stimulatory Activity with RGX-104 in Advanced Cancer Patients

Oct 30, 2017

Preliminary Phase 1a/b data demonstrate RGX-104 modulates the innate immune system to stimulate T cell activation in a diverse group of heavily pre-treated patients with refractory solid cancers.  

Phase 1 dose escalation continues, and future plans include initiation of an expansion component of the trial as monotherapy as well as in combination with a checkpoint inhibitor.

FOR IMMEDIATE RELEASE

New York, NY – October 29, 2017 – Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today preliminary data from an ongoing Phase 1a/b clinical trial with its lead oral investigational agent, RGX-104. These data demonstrate immune-stimulatory activity in solid tumor patients with highly-refractory malignancies, including patients who have failed prior checkpoint inhibitors. Also presented were pre-clinical data establishing the immune-modulatory and anti-tumor effects of RGX-104. The company presented the data at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Philadelphia.

RGX-104 is a liver X receptor (LXR) agonist that upregulates the expression of the target gene, Apolipoprotein E (ApoE), triggering several downstream effects via ApoE receptors. In pre-clinical data presented today, treatment with RGX-104 in mouse models resulted in dual effects on myeloid-derived suppressor cells (MDSCs) and dendritic cells (DCs), both innate immune cells that play a central role in regulating anti-tumor immunity and response to checkpoint inhibitors. Innate immune activation with RGX-104, coupled with a reduction in tumor blood vessels, resulted in anti-tumor activity as a monotherapy as well as synergy with checkpoint inhibitors (CPI) in several drug-resistant mouse models. These data provide rationale for Rgenix’s ongoing Phase 1a/b trial of RGX-104 in advanced cancer patients and support evaluation of RGX-104 as both a monotherapy as well as in combination with CPIs.

As part of the ongoing Phase 1a/b clinical trial, 15 patients with a variety of solid tumors have been treated with escalating doses of RGX-104 monotherapy. Patients treated with RGX-104 had a median of six prior therapies with a range of 1-12, highlighting a population of patients with profoundly resistant disease.

Activation of the LXR-ApoE pathway with oral administration of RGX-104 was associated with immune-stimulatory activity in 9 of 10 evaluable patients. This was demonstrated by an increase (up to 11-fold) in activated circulating PD-1+CD8+ T cells during treatment. T cell activation was observed in patients who experienced modulation of the innate immune system during treatment. The effect of RGX-104 on the innate immune system consisted of both MDSC depletion (up to 95% decrease) as well as DC activation as indicated by induction of PD-L1 expression (up to 100% increase). In most cases these effects were observed within two weeks of treatment initiation and generally preceded the onset of T cell activation.

Safety data demonstrate good tolerability with on-target safety findings in the first three dosing cohorts. One patient experienced a DLT of grade 4 reversible neutropenia - a known potential effect of LXR agonism – that reversed within one week, allowing the patient to subsequently tolerate a 50% dose reduction. No MTD has been reached to date. Stable disease has been observed in 4 of 12 evaluable patients, including three who have failed prior checkpoint inhibitor therapy, for periods of at least 8 weeks.

“We are very pleased to see robust evidence of immune stimulation in such highly-pretreated patients,” said Roger Waltzman, MD, MBA, and Chief Medical Officer of Rgenix. “CPI therapy is now commonplace but only a minority of patients derive clinical benefit. We hope the effects of RGX-104 on modulating barriers to innate and adaptive immune function will enable a larger number of patients to benefit from this therapy. These preliminary results also highlight the potential for development of RGX-104 as a monotherapy.”

Rgenix plans to enroll subsequent dose-escalation cohorts of the RGX-104 monotherapy trial in Q4 2017. Additionally, Rgenix is planning to initiate the Phase 1b expansion component of the study, comprised of disease directed cohorts receiving RGX-104 monotherapy as well as cohorts receiving RGX-104 combined with a CPI, projected to begin in 1H 2018.

“These preliminary data establish RGX-104 as a potential first-in-class oral immunotherapy agent with broad immune-stimulatory activity and a unique dual mechanism targeting innate immunity,” said Masoud Tavazoie, MD, PhD, and Chief Executive Officer of Rgenix. “These results also further validate our discovery platform at Rgenix, as well as our pipeline of other drug candidates slated to begin entering clinical-stage development in 2018.”

The LXR-ApoE pathway was discovered as a cancer target using a microRNA (miRNA) based target discovery approach originally developed at The Rockefeller University and now exclusively licensed to Rgenix.

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 a potent small molecule agonist of the Liver X Receptor (LXR). 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 in tumor models. 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 pre-clinical 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.

Media Contacts:

RooneyPartners

Marion Janic

212-223-4017

mjanic@rooneyco.com

 

Oct 26, 2017

Rgenix to Present Early Clinical Data on RGX-104 at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics

Oct 26, 2017

FOR IMMEDIATE RELEASE

New York, NY – October 26, 2017 - Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today that Dr. Monica Mita of Cedars-Sinai Medical Center and Principal Investigator of the RGX-104-001 study, will present at the 2017 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics. The conference is scheduled to take place from Thursday, October 26 to Monday, October 30 in Philadelphia.

The details of Rgenix’s presentation are as follows:

Event:              AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics: Discovery, Biology, and Clinical Applications

Date:               October 29, 2017

Time:               12:30 P.M. – 4:00 P.M. EST

Description:     Poster B001, “A phase 1 trial of RGX-104, a first-in-class immunotherapy targeting the liver-X nuclear hormone receptor (LXR), in patients with refractory malignancies”

Location:         Hall E, Pennsylvania Convention Center, 1101 Arch St, Philadelphia, PA 19107

Dr. Mita will present data from the Phase 1 trial of RGX-104, a first-in-class investigational immunotherapy.

 

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 a potent small molecule agonist of the Liver X Receptor (LXR). 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 in tumor models. 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 pre-clinical 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.

 

Media Contacts:

RooneyPartners

Marion Janic

212-223-4017

mjanic@rooneyco.com

Oct 9, 2017

Meet the 30 biotech leaders under 40 who are searching for breakthrough treatments and shaping the future of medicine

Oct 9, 2017

It's an exciting time to be in the biotech and pharmaceutical industries. To read the full piece, please click here.

Sep 14, 2017

For Rgenix CEO Masoud Tavazoie, fighting cancer is a family business

Sep 14, 2017

Rgenix CEO Masoud Tavazoie was recently interviewed for an article in The Pharma Letter. To read the full piece, please click here.

Apr 28, 2017

Rgenix Appoints Chief Medical Officer

Apr 28, 2017

For Immediate Release

New York, NY – April 27, 2017 – Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today the appointment of Roger Waltzman, M.D., M.B.A. as its Chief Medical Officer. Dr. Waltzman brings more than 20 years of experience in oncology across the entire new drug development and approval process, including serving in various executive roles at Novartis for nearly a decade.

In his role, he will oversee clinical development at Rgenix, including clinical trial design and the new drug application process with regulators. He will also support Rgenix’s goals of providing a meaningful response for cancer patients lacking effective therapies by overseeing the patient experience.

“Dr. Waltzman has an extraordinary background in oncology, which is our focus at Rgenix, and his expertise will be crucial as we progress with the clinical development of our lead therapy, RGX-104, and our other drug candidates in development,” said Masoud Tavazoie, M.D., Ph.D., Chief Executive Officer and co-founder of Rgenix. “We look forward to his contributions to our work at Rgenix as we develop novel treatments for patients who suffer from cancer types with a high, unmet need.”

“Rgenix has innovation at its core, and I’m pleased to be joining an organization that is looking to fill a gap in oncology that could benefit hundreds of thousands of patients. I look forward to collaborating with the Rgenix team on its therapies and clinical developments as it looks to drive positive change in the industry,” said Dr. Waltzman.

Prior to Rgenix, Dr. Waltzman worked at Novartis in both Medical Affairs and Clinical Development roles. In oncology, he successfully filed an sNDA as the Clinical Development Head for Tasigna/Glivec and led the successful development and MAA/NDA filing of Jakavi/Jakafi, in collaboration with Incyte.  His last position at Novartis was as Global Development Head for anti-malarials, where he continued Phase 2 development of two novel anti-malarials and facilitated a cost-saving Collaboration Agreement with the Bill and Melinda Gates Foundation and Medicines for Malaria Venture.

His most recent position was with Jaguar Animal Health and Napo Pharmaceuticals in San Francisco where he was the Chief Scientific Officer and Chief Medical Officer, leading the development of a first-in-class, FDA-approved botanical as a novel anti-diarrheal.

Dr. Waltzman received his medical degree from Brown University School of Medicine and his MBA from Columbia Business School. He completed his residency in Internal Medicine at Harvard’s Beth Israel Hospital, and his fellowship in hematology/oncology at Memorial Sloan-Kettering Cancer Center. He has been a member of the board of directors of GoDocGo and the Brown Medical Alumni Association since 2015.

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 Contacts:

RooneyPartners

Marion Janic

212-223-4017

mjanic@rooneyco.com

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:

RooneyPartners

Marion Janic

212.223.4017

mjanic@rooneyco.com

 

Jun 29, 2016

What does it take to succeed as a New York biotech startup?

Jun 29, 2016

View article

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

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

Jun 15, 2016

View article

Jun 15, 2016

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

Jun 15, 2016

View article

Aug 31, 2015

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

Aug 31, 2015

View article

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To learn more about our science and products, please send us a message below.

 
 

Address

310 East 67th Street, Suite 1-12‎

New York, NY 10065, USA

 

Phone number

(646) 856-9261