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

NEW YORK–(BUSINESS WIRE)– 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 cancer 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.

Image may be NSFW.
Clik here to view.

View source version on businesswire.com: http://www.businesswire.com/news/home/20170106005115/en/