New York, NY – June 22, 2020 – RGENIX, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today it is presenting an abstract on RGX-104, RGENIX’s lead therapy in development. RGENIX’s abstract, “Correlative analysis of pharmacokinetics and pharmacodynamics of RGX-104, a first-in-class Liver-X-Receptor (LXR) agonist, and clinical outcomes in patients with advanced solid tumors” was accepted for the 2020 American Association for Cancer Research (AACR) Annual Meeting, which this year was scheduled as two virtual meetings. The abstract results will be presented as a virtual poster presentation (#LB-133/7) in the Late-Breaking Research: Clinical Research 1 session on June 22, by clinical investigator Dr. Monica Mita, Co-Director, Experimental Therapeutics Program, Samuel Oschin Comprehensive Cancer Institute at Cedars-Sinai Medical Center, who is lead author on the study.

RGX-104 is a small-molecule LXR agonist that modulates innate immunity via transcriptional activation of the ApoE gene. RGX-104 inhibits tumor angiogenesis and depletes myeloid derived suppressor cells (MDSC), thereby activating cytotoxic T-lymphocytes. MDSCs are associated with resistance to both checkpoint inhibitors (CPI) and chemotherapy, providing a rationale for combination therapy with RGX-104.

For the dose escalation stage of the Phase 1 study, RGX-104 was tested as a monotherapy or in combination with either nivolumab, ipilimumab, or docetaxel in heavily pre-treated patients with refractory or relapsed solid tumors, including patients who had progression on prior checkpoint inhibitors (CPI). As outlined in the presentation, objective clinical activity was observed in all treatment arms, including the monotherapy arm, with partial responses achieved in patients with NSCLC, SCLC, melanoma, SCCHN, and endometrial cancer.

Of note, in the combination arms, a 28.6% objective response rate was observed in all evaluable patients who had previously progressed on CPI, with 4 of 14 evaluable patients achieving PRs. Responses included ongoing durable PRs – some exceeding 11 months – in CPI refractory/resistant patients whose tumors were PD-L1 low/negative.

Importantly, clinical activity across all treatment arms was associated with RGX-104 related pharmacodynamic effects, including ApoE activation, MDSC depletion, and CD8 T cell activation with associated induction of IFNg. Robust ApoE induction was achieved with BID dosing of RGX-104 and was correlated with the magnitude of MDSC depletion. Additionally, in comparison to PD-L1 positive tumors, PD-L1 negative tumors were found to have significantly lower baseline (pre-treatment) levels of ApoE, a feature associated with higher likelihood of clinical benefit to RGX-104. The data demonstrate that MDSC depletion via ApoE induction with RGX-104 can overcome resistance to CPI or chemotherapy, resulting in durable clinical activity.

As a result, RGX-104 is being evaluated in combination with the front-line standard-of-care regimen of pembrolizumab plus carboplatin/pemetrexed in a phase 1b/2 study currently enrolling patients with advanced non-squamous non-small cell lung cancer (NSCLC) whose tumors are PD-L1 negative. RGX-104 is also being evaluated in combination with docetaxel in a phase 1b/2 expansion study that has begun enrolling patients with relapsed/refractory extensive stage small-cell lung cancer (ES-SCLC) or high grade-neuroendocrine tumors (HG-NET).

Monica Mita, M.D., principal investigator from Cedars-Sinai Medical Center and lead author and presenter of the poster, said, “These exciting results provide clinical validation of the novel MDSC-targeting mechanism of RGX-104. The durable clinical responses observed in patients who have previously progressed on checkpoint inhibitors are very promising.”

Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer of RGENIX, said, “We are very encouraged by the results presented today as they demonstrate that our first-in-class drug candidate RGX-104 can robustly target MDSCs, a key drug resistance mechanism, to provide durable clinical benefit to patients. The findings further strengthen the foundation for our ongoing Phase 1b/2 studies. We look forward to sharing results from these ongoing studies.”

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Exor Seeds, and the Partnership Fund for New York City. 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 a Phase 1b/2 clinical study. 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. 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.

Media Contact:

RooneyPartners
Jeanene Timberlake
646-770-8858
jtimberlake@rooneyco.com

New York, NY – June 22, 2020 – RGENIX, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today it is presenting an abstract on RGX-019, RGENIX’s pre-clinical antibody program in development. RGENIX’s abstract, “In Vivo Safety and Efficacy of RGX-019, a MerTK Targeting Monoclonal Antibody” was accepted for the 2020 American Association for Cancer Research (AACR) Annual Meeting, which this year was scheduled as two virtual meetings. The abstract results will be presented as a virtual poster presentation (LB-090) during the Late-Breaking Research: Immunology 1 session on June 22, by RGENIX’s Vice President of Research Dr. Isabel Kurth, who is senior author on the abstract.

MerTK is a member of the TAM family of receptor tyrosine kinases and is predominantly expressed on macrophages as well as on cancer cells from a number of solid and hematologic malignancies. The binding of ligands to MerTK on cancer cells activates signaling that increases proliferation, angiogenesis, and drug resistance. On immune-suppressive M2 macrophages, MerTK signaling promotes immune tolerance. Therefore, there is rationale to target MerTK both to suppress tumor growth and activate anti-tumor immunity.

RGX-019 is a novel humanized MerTK targeting monoclonal antibody with a unique mechanism of action. As outlined in the presentation, RGX-019 was found to bind with high affinity to human and monkey MerTK, without detectable binding to related TAM receptors AXL or Tyro.  RGX-019’s unique mechanism drives MerTK degradation through receptor internalization and lysosomal trafficking, resulting in the elimination of MerTK from the surface of treated cells.  In vitro, RGX-019 inhibited human cancer cell viability and induced immune-stimulatory cytokine expression in M2 macrophages, consistent with robust inhibition of MerTK signaling. RGX-019 treatment in vivo led to near complete elimination of MerTK from the surface of tumor cells, which was associated with anti-tumor efficacy in mouse xenograft models.

Importantly, RGX-019 demonstrated a favorable safety profile in a 28-day dose-range finding toxicology study in monkeys. Of note, retinal toxicity – a finding associated with other MerTK targeting approaches – was not observed at any dose of RGX-019. The results overall demonstrate that RGX-019 can potently target MerTK signaling on both cancer cells and immune-suppressive M2 macrophages with a novel mechanism, resulting in anti-tumor activity with a wide therapeutic window.

Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer of RGENIX, said, “The results presented today demonstrate the potential for RGX-019 to be a best-in-class MerTK inhibitor. Its unique characteristics have yielded a favorable safety and efficacy profile in animals that provides a solid foundation for further development of RGX-019. We are excited to move RGX-019 through further IND-enabling studies and ultimately into clinical development.”

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, Exor Seeds, and the Partnership Fund for New York City. For more information, please visit www.rgenix.com.

About RGX-019

RGX-019 is humanized monoclonal antibody that targets MerTK, a receptor kinase that mediates tumor growth as well as immune evasion. RGX-019 has a unique mechanism-of-action that results in degradation of MerTK in target cells, which is associated with in vivo anti-tumor activity. RGX-019 is currently in pre-clinical development. MerTK was revealed as a driver of metastatic cancer progression using a novel microRNA-based discovery platform developed by RGENIX’s scientific co-founders at The Rockefeller University.

Media Contact:

RooneyPartners
Jeanene Timberlake
646-770-8858
jtimberlake@rooneyco.com

Data from Phase 1 Trial of RGX-202 Presented at 2020 ASCO Virtual Scientific Program

New York, NY – May 29, 2020 – RGENIX, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today it is presenting data showing clinical activity in patients with advanced colorectal (CRC) tumors from a clinical trial of RGX-202, a first-in-class small molecule that inhibits a novel cancer metabolism pathway involved in supplying energy to cancer cells. RGENIX’s abstract, “Phase 1 trial of RGX-202, a first-in-class oral inhibitor of the SLC6a8/CKB pathway, in patients with advanced gastrointestinal (GI) solid tumors” is being presented by Dr. Johanna Bendell, Director of the Drug Development Unit at Sarah Cannon Research Center in Nashville, and clinical investigator and lead author on the study as part of the 2020 American Society of Clinical Oncology Virtual Scientific Program, during an oral abstract session available on demand starting on May 29.

RGX-202 is an oral small molecule inhibitor of the creatine transporter SLC6a8 that depletes intracellular phospho-creatine and ATP levels, leading to the death of cancer cells. The SLC6a8/CKB pathway was discovered using RGENIX’s proprietary miRNA-based target-discovery platform applied to KRAS mutant CRC. Due to increased metabolic demand, KRAS mutant tumors are highly susceptible to blockade of SLC6a8. In CKB-expressing (CKB+) pre-clinical models, RGX-202 treatment triggers tumor regressions across KRAS mutant subtypes. The ongoing Phase 1 trial of the compound is an escalation/expansion study of RGX-202 with or without FOLFIRI in patients with advanced GI tumors, and the trial will identify the maximum tolerated dose, or the dose at which multiple dose-limiting toxicities (DLTs) are not observed, and to determine the antitumor activity of the RGX-202.

As of April 29, 2020, 17 patients with advanced relapsed/refractory gastrointestinal cancers have been treated in 4 single agent dose escalation cohorts, with doses ranging from 600mg twice a day (BID) up to 3600mg BID. RGX-202 was well tolerated at all doses, with the majority (69%) of RGX-202-related adverse events (AEs) being grade 1 in severity with the most common AEs being nausea and vomiting. No DLTs were observed at any dose; notably, in the two highest cohorts, drug exposures predicted to be sufficient to inhibit human tumor growth from preclinical models were achieved along with concomitant pharmacodynamic effects.

10 patients were evaluable for response across all dose cohorts. Among the 5 evaluable patients with KRAS mutant CRC, 1 achieved a PR and 2 achieved stable disease, for an ORR of 20% and a disease control rate of 60%. Durable clinical benefit was observed in the highest dose cohort; a patient with KRAS G12V mutant CRC had a confirmed partial response of 40 weeks duration, and a patient with KRAS G13D mutant CRC experienced stable disease for 16 weeks. The ORR in all evaluable CRC patients – regardless of KRAS status – was 10% with a disease control rate (DCR) of 40%. A dose-escalation study assessing RGX-202 plus the chemotherapy regimen FOLFIRI is ongoing, with a planned expansion cohort in CKB+ CRC.

Masoud Tavazoie, MD, PhD, and Chief Executive Officer of RGENIX, said, “Though RGX-202 is still in an early stage of development, the results obtained to date are exciting. The single agent safety and activity profile provide a foundation for further development of RGX-202, including in combination regimens.  Importantly, the results further validate our target discovery approach, which has now yielded two first-in-class clinical compounds, RGX-104 and RGX-202, both with single agent activity against novel targets discovered using our RNA-based platform technology. We look forward to sharing additional data from our programs as they progress through clinical development.”

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments,LLC, and the Partnership Fund for New York City. For more information, please visit www.rgenix.com.

About 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, the SLC6a8/CKB pathway, is over-expressed in several prevalent cancer types, including KRAS mutant colorectal cancer. RGX-202 has demonstrated anti-tumor activity in preclinical studies, both as a single agent as well as in combination with standard-of-care therapies. In a Phase 1 dose escalation, RGX-202 demonstrated clinical activity as a single agent in the KRAS mutant CRC setting. A Phase 1b study assessing the safety and clinical activity of RGX-202 in combination with FOLFIRI is ongoing.

Media Contact:

Media Contact:
RooneyPartners
Jeanene Timberlake
646-770-8858
jtimberlake@rooneyco.com

New York, NY – May 21, 2020 – RGENIX, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today that Dr. Johanna Bendell, Director of the Drug Development Unit at Sarah Cannon Research Center in Nashville, will present an oral abstract related to the RGX-202 program at the 2020 American Society of Clinical Oncology (ASCO) Virtual Scientific Program. The meeting is scheduled to take place May 29 – 31, 2020.

The details of Rgenix’s presentation are as follows:

Event: 2020 ASCO Virtual Scientific Program

Date: May 29, 2020

Time: 8:00 AM EDT (available for download)

Description: Abstract #3504, “Phase I monotherapy dose escalation of RGX-202, a first-in-class oral inhibitor of the SLC6a8/CKB pathway, in patients with advanced gastrointestinal (GI) solid tumors.”

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments,LLC, and the Partnership Fund for New York City. For more information, please visit www.rgenix.com.

About 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, the SLC6a8/CKB pathway, is over-expressed in several prevalent cancer types, including KRAS mutant colorectal cancer. RGX-202 has demonstrated anti-tumor activity in preclinical studies, both as a single agent as well as in combination with standard-of-care therapies.

Media Contact:

Media Contact:
RooneyPartners
Jeanene Timberlake
646-770-8858
jtimberlake@rooneyco.com

New York, NY – April 27, 2020 – Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today it is presenting an abstract on RGX-104, RGENIX’s lead therapy in development. RGENIX’s abstract, “RGX-104, a first-in-class immunotherapy targeting the liver-X receptor (LXR); Initial results from the phase 1b RGX-104 plus Docetaxel combination dose escalation cohorts” was accepted for the 2020 American Association for Cancer Research (AACR) Annual Meeting, which this year was scheduled as two virtual meetings. The abstract results will be presented as a virtual poster presentation (CT-146) during the VPO.CT01 – Phase I Clinical Trials session on April 27, by clinical investigator Dr. Emerson Lim from Columbia University Herbert Irving Comprehensive Cancer Center, who is lead author on the study.

For the dose escalation stage of this Phase 1b study, RGX-104 was tested in combination with docetaxel across three different dose escalation cohorts. The presentation will outline the safety profile, pharmacodynamic effects, and clinical activity of the combination in unselected heavily pre-treated patients with refractory or relapsed solid tumors. The results support further development of this regimen.

RGX-104 is a small-molecule LXR agonist that modulates innate immunity via transcriptional activation of the ApoE gene. RGX-104 inhibits tumor angiogenesis and depletes myeloid derived suppressor cells (MDSC), thereby activating cytotoxic T-lymphocytes. MDSCs are associated with resistance to both checkpoint inhibitors (CPI) and chemotherapy, including docetaxel, providing a rationale for combination therapy with RGX-104. Consistent with this, RGX-104 plus docetaxel combination therapy is highly efficacious in pre-clinical models that demonstrate MDSC-associated docetaxel resistance.

As of February 7, 2020, 11 patients with refractory solid tumors were treated with the combination across 3 different dosing cohorts. The safety profile was consistent with the individual profiles of RGX-104 and docetaxel, with neutropenia the most common drug-related adverse event observed, but dose-limiting in only one patient in cohort one, where RGX-104 was administered at 80mg BID every day with docetaxel administered at 35mg/m2 weekly x 3. In cohorts two and three, in which patients received RGX-104 dosing five consecutive days out of seven (at either 80mg or 100mg BID) and a reduced dose of docetaxel (28mg/m2), no dose-limiting toxicity was observed, and sustained pharmacodynamic activity (ApoE activation and MDSC depletion) was achieved. The overall response rate (ORR) in all evaluable patients was 22%, with a disease control rate (DCR) of 56%. Across cohorts 2 and 3, where target RGX-104 pharmacodynamic effects were achieved, the ORR was 33% with a 67% DCR.  Clinical responses included partial responses (PRs) in CPI refractory/resistant patients, including an ongoing confirmed PR in a CPI resistant melanoma patient treated in cohort 2 that remains on study at 10 months. Clinical activity was associated with increases in T cell activation markers exceeding that generally observed with RGX-104 alone.

As a result, the RGX-104/docetaxel regimen is being evaluated in a phase 1b/2 expansion study that has begun enrolling patients with relapsed/refractory extensive stage small-cell lung cancer (ES-SCLC) or high grade-neuroendocrine tumors (HG-NET).

Emerson Lim, M.D., principal investigator from Columbia University Herbert Irving Comprehensive Cancer Center and lead author and presenter of the poster, said, “The clinical activity seen in the dose escalation of RGX-104 combined with Docetaxel is quite encouraging; especially notable is the ongoing PR of greater than 10 months duration in a patient with metastatic melanoma who had previously progressed with combination Nivolumab and Ipilimumab as his second line therapy. I am hopeful this combination will provide a therapeutic option for patients with ES-SCLC/HG-NET as currently being tested in the expansion phase.”

RGX-104 is also being evaluated in combination with the front-line standard-of-care regimen of pembrolizumab plus carboplatin/pemetrexed in a phase 1b/2 study currently enrolling patients with advanced non-squamous non-small cell lung cancer (NSCLC).

Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer of RGENIX, said, “The data from the RGX-104/docetaxel combination dose escalation cohorts are encouraging, providing early safety and efficacy data that support further development of the combination.  Though we are still in the early stages of the RGX-104 Phase 1b/2 studies, the results show the potential of RGX-104 to provide durable clinical activity in refractory patients through a novel mechanism-of-action. We look forward to sharing additional findings from these ongoing studies.”

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments,LLC, and the Partnership Fund for New York City. 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 a Phase 1b/2 clinical study. 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. 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.

Media Contact:

Media Contact:
RooneyPartners
Jeanene Timberlake
646-770-8858
jtimberlake@rooneyco.com

Masoud Tavazoie, Rgenix CEO and Co-Founder speaks at Immuno-Oncology 360°

From February 26th – 28th 2020, the sixth IO360 conference in New York City gathers key representatives from industry, non-profits, regulatory bodies, investors and patients to discuss advances and challenges in immuno-oncology (IO) in the fight against cancer. Stakeholders will present and contrast strategies in IO from a scientific, clinical and regulatory viewpoint. In spite of the immeasurable positive impact that IO has had for patients, recent failures of multiple agents suggest that in order to advance the field, new angles are required in the investigation of immunology and disease biology.

Masoud Tavazoie, CEO of RGENIX, will present rationale and development strategies for novel modalities impacting the innate immune system. These first or best in class molecules target myeloid derived suppressor cells and macrophages and represent new strategies to overcome resistance or to improve response to IO- and chemotherapy and achieve a more durable benefit for more patients.

Masoud Tavazoie
Masoud Tavazoie

Robert Wasserman, MD, joins Rgenix with more than 20 years of oncology clinical development experience and prior senior roles in the biopharma industry.

Wasserman will oversee new Phase 1b/2 trial of lead compound RGX-104 in lung cancer patients; and RGX-202 Phase 1b/2 trial in patients with colorectal and gastric cancer.

New York, NY – September 16, 2019 – Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today that Robert Wasserman, MD, has joined the Rgenix management team as its new Chief Medical Officer. Dr. Wasserman takes on his new role after a 20-year career that includes senior positions at large pharma companies including Roche, Novartis and Merck.

In his new role, Dr. Wasserman will have oversight of clinical development for Rgenix’s two lead clinical programs, RGX-104 and RGX-202. In particular, he will oversee the imminent launch of a Phase 1b/2 trial of Rgenix’s lead therapy, RGX-104, a first-in-class oral LXR agonist that has demonstrated anti-tumor activity as a single agent and in combination with other therapies – including chemotherapy and checkpoint blockade – in patients with refractory cancers.

For the Phase 1b/2 trial, RGX-104 will be tested in combination with the standard-of-care regimen consisting of chemotherapy (carboplatin/pemetrexed) plus the checkpoint inhibitor immunotherapy pembrolizumab as a first-line treatment in non-small cell lung cancer (NSCLC) patients whose tumors lack expression of the PD-L1 protein (<1% expression). NSCLC patients whose tumors lack PD-L1 expression have significantly lower response rates to standard-of-care in the first line setting, and thus represent a patient population of high unmet need. Resistance to standard-of-care immunotherapy has been associated with the presence of high levels of immunosuppressive myeloid cells known as MDSCs. RGX-104 has demonstrated broad-spectrum depletion of MDSCs in refractory cancer patients – including NSCLC patients – and therefore represents a novel approach to overcome drug resistance. As part of the Phase 1b/2 trial, RGX-104 will also be tested in patients with small cell lung cancer (SCLC/HG-NET) in combination with docetaxel chemotherapy as a second line treatment.

“Bob’s vast experience in oncology and clinical research is a perfect fit for Rgenix given our expanding clinical programs and pending milestones,” said Masoud Tavazoie, MD, PhD, Chief Executive Officer and co-founder of Rgenix. “As we prepare to launch Phase 1b/2 studies for our first-in-class compounds RGX-104 and RGX-202, we are eager to leverage his knowledge and expertise to help expand our efforts for patients with cancers considered to have high unmet need.”

“I look forward to working with the team at Rgenix to develop novel treatments for these patients by applying the company’s unique platform technology and methodology to discover drivers of cancer growth. With its roots in strong science and its dedication to a novel approach, Rgenix’s reputation as a premier innovator in the oncology space is growing and I’m excited to be a part of such a dynamic effort at this key stage for the company,” said Dr. Wasserman.

Dr. Wasserman started his career in the industry working with major pharmaceutical companies. His last large pharma appointment was at Roche, where he ended a six-year period at the company as the Chairman of the Clinical Biomarker Leadership Team and where he also served for a time as the Global Head of Oncology Translational Medicine (Early Clinical Development) for Roche Pharma Research/Early Development. Prior to Roche, he was the Deputy Global Head of Oncology Biomarkers at Novartis and, before that at Merck.

His most recent biotech role was as the Chief Medical Officer at Northern Biologics, a Canadian  company developing first-in-class immuno-oncology products. He currently maintains a position as an Advisory Board Member of Sectoral Asset Management out of Montreal.

Dr. Wasserman earned his B.A. from the University of Pennsylvania and his MD from the Johns Hopkins University School of Medicine. He completed his pediatric residency and pediatric hematology-oncology fellowship training at The Children’s Hospital of Philadelphia, at the University of Pennsylvania and then was on faculty as an Assistant Professor.

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments, LLC, and the Partnership Fund for New York City. For more information, please visit www.rgenix.com.

About RGX-104

RGX-104 is an investigational orally-administered potent small molecule agonist of the Liver X Receptor (LXR) that is currently being evaluated in a Phase 1b/2 clinical trial. 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 1b/2 clinical trial of RGX-104 in patients with lung cancer —for more information about the clinical trial, please visit: https://clinicaltrials.gov/ct2/show/NCT02922764.

About RGX-202

RGX-202 is an investigational orally-administered small molecule inhibitor of the SLC6a8/CKB pathway that is currently being evaluated in a Phase 1a clinical trial. RGX-202 induces apoptosis (cell death) of gastrointestinal cancer cells that over-express the CKB protein (CKB+). Blockade of the SLC6a8 pathway by RGX-202 starves cancer cells of the metabolite phosphocreatine which is required for cancer cell survival in CKB+ tumors. Pre-clinical research demonstrates that RGX-202 is active both as a single agent as well as in combination with standard-of-care therapies in several gastrointestinal cancer models, including KRAS mutant tumor models.

A phase 1a study of RGX-202 in patients with gastrointestinal cancers, including colorectal and gastric cancer, is in progress. For more information about our clinical trial visit:

https://clinicaltrials.gov/ct2/show/NCT03597581

Media Contacts:

RooneyPartners

Marion Janic

212-223-4017

mjanic@rooneyco.com

Pre-clinical data demonstrates first-in-class MERTK selective inhibitor with novel mechanism of action

Rgenix has initiated IND-enabling studies of RGX-019

 FOR IMMEDIATE RELEASE 

New York, NY – April 3, 2019 – Rgenix, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and antibody cancer therapeutics, announced today it is presenting pre-clinical data from ongoing research of RGX-019, a monoclonal antibody that targets MERTK, for the treatment of advanced cancer. In a presentation of a late-breaking abstract, “Characterization of the anti-cancer and immunologic activity of RGX-019, a novel pre-clinical stage humanized monoclonal antibody targeting the MERTK receptor”, which was accepted for the 2019 American Association of Cancer Research Annual Meeting, Isabel Kurth Ph.D., Rgenix VP of Research, discussed data showing RGX-019 to be a potent and selective inhibitor of MERTK signaling, resulting in suppression of cancer growth and activation of the innate immune response.

RGX-019 is a humanized monoclonal antibody that selectively targets MERTK, a receptor tyrosine kinase of the TYRO3/AXL/MERTK (TAM) family. MERTK is expressed in immune cells such as macrophages, dendritic cells and NK cells, and is also overexpressed in a wide variety of liquid and solid cancers.

Activation of MERTK on cancer cells via ligand binding activates several tumor-promoting signaling pathways, stimulating tumor proliferation, migration and angiogenesis, and decreasing apoptosis and chemosensitivity. When activated on macrophages, MERTK promotes an immune-suppressive M2 phenotype.

RGX-019 binds to human MERTK with high affinity and selectivity, without detectible binding to other related TAM kinases. RGX-019 has a unique mechanism of action that not only leads to blockade of ligand binding, but also to MERTK degradation via receptor internalization.

This novel mechanism of action leads to inhibition of cancer growth in vitro and in vivo as well as activation of M1 (pro-inflammatory) cytokine release from immune-suppressive M2 macrophages.

Masoud Tavazoie, MD, PhD, and Chief Executive Officer of Rgenix, said, “We are excited to have an opportunity to reveal the pre-clinical data that demonstrates the positive progress we are making with the development of RGX-019, our third proprietary program. The ability of RGX-019 to selectivity degrade MERTK in cancers cells and M2 macrophages provides a distinct advantages over current small-molecule approaches to targeting MERTK which are hampered by off-target binding to other related kinases. This data provides a strong foundation for IND enabling studies.”

Sohail Tavazoie, MD, PhD, and Chair of Rgenix’s Scientific Advisory Board, said, “RGX-019 shows great potential as a cancer therapeutic and its ability to selectively modulate both cancer growth and innate immune activation is illustrative of that possibility. We look forward to continuing our research on this antibody and its impact on various MERTK expressing cancers.”

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 Holdings A/S, Sofinnova Partners, Lepu Holdings Limited, Oceanpine Capital, WuXi PharmaTech Healthcare Fund I, LP, Alexandria Venture Investments,LLC, and the Partnership Fund for New York City. For more information, please visit www.rgenix.com.

About RGX-019

RGX-019 is an investigational drug candidate and a humanized monoclonal antibody that targets MERTK being developed by Rgenix for the treatment of advanced cancer. Rgenix in-licensed RGX-019 related antibody intellectual property from Rockefeller University as part of a world-wide exclusive licensing agreement.

MERTK is a receptor tyrosine kinase of the TAM family and is over-expressed in several human cancers. The MERTK pathway drives cancer progression, tumor angiogenesis, and innate immune suppression. Targeting MERTK inhibits the growth of MERTK-expressing cancer cells and induces an M1 pro-inflammatory cytokine expression profile in tumor-associated macrophages. Rgenix has initiated IND-enabling studies of RGX-019.

Media Contacts: 

RooneyPartners

Marion Janic

212-223-4017

mjanic@rooneyco.com