On October 11, 2018, VCBeat (WeChat ID: VCbeat) learned that Rgenix, a biopharmaceutical company focused on the research and development of novel small-molecule and antibody-based cancer therapies, had secured $40 million in Series C financing. The round was led by Lepu Medical, a global healthcare company, with participation from new investors Oceanpine Capital and WuXi AppTec’s Corporate Venture Fund, as well as existing investors Novo Holdings A/S, Sofinnova Partners, Alexandria Venture Investments, LLC, and New York City’s Innovation NYC Fund along with its affiliated partner funds. Jefferies LLC served as the financial advisor to Rgenix in this financing round. Previously, Rgenix raised $33 million in Series B financing in June 2016, co-led by Novo Holdings and Sofinnova Partners. Since its establishment in 2010, Rgenix has raised a total of $83.5 million.
Rgenix will use the proceeds from this financing round to advance its clinical and preclinical oncology drug development programs and support general corporate operations. The funds will be primarily allocated to progressing the Phase 1b/2 clinical trial of RGX-104 for multiple cancer indications, as well as supporting the early-stage clinical development of RGX-202.
Pu Zhongjie, CEO and Chairman of Lepu Medical, stated, “Lepu Medical is honored to invest in Rgenix. We are highly optimistic about Rgenix’s unique mRNA discovery platform, which enables the development of first-in-class drugs targeting various cancer cell antigens. We also believe that RGX-104 holds significant potential as a checkpoint inhibitor across many types of cancer. Currently, Lepu Medical is conducting clinical trials on PD-1 and PD-L1 checkpoint inhibitors as well as oncolytic viruses. Therefore, we look forward to exploring potential collaboration opportunities with Rgenix to achieve our shared goal of further expansion in the oncology market.”
Rgenix’s drug discovery platform can identify novel RNA-regulated targets in cancer cells across various types of solid tumors. This mRNA platform was initially developed by Rgenix’s scientific co-founders at Rockefeller University, and Rgenix has now secured exclusive licensing rights to the platform. Currently, Rgenix’s R&D pipeline primarily includes:
RGX-104 is a novel, first-in-class oral small-molecule drug that prevents tumor immune evasion and overcomes drug resistance. RGX-104 targets the liver X receptor (LXR) and activates the APOE gene to modulate innate immunity, thereby treating cancer by regulating the activity of two types of innate immune cells: myeloid-derived suppressor cells (MDSCs), which it depletes, and dendritic cells (DCs). MDSCs block T-cell activation, whereas DCs activate T cells; RGX-104 depletes MDSCs and stimulates DCs, thereby inducing antitumor activity of the human immune system. RGX-104 demonstrates potential efficacy both as a monotherapy and in combination with immunotherapies such as PD-1 inhibitors.
RGX-104 has been granted Orphan Drug Designation by the U.S. FDA for the treatment of melanoma, glioblastoma multiforme (GBM), and ovarian cancer. Meanwhile, Rgenix has also obtained a license from GlaxoSmithKline (GSK) for RGX-104 and other LXR-related intellectual property. Currently, RGX-104 is undergoing Phase 1b/2 clinical trials in combination with Opdivo for the treatment of various cancers, including ovarian cancer and non-small cell lung cancer. Trial results indicate that monotherapy also demonstrates immunostimulatory and antitumor activities.
RGX-202 is a novel small-molecule compound that inhibits cancer cell metabolism by targeting the creatine transporter SLC6A8 to suppress creatine synthesis, thereby blocking the conversion of creatine into ATP and depriving cancer cells of energy for their activities. Preclinical studies have demonstrated that RGX-202 can be used as a monotherapy or in combination with standard chemotherapy for the treatment of gastrointestinal cancers. Rgenix plans to initiate clinical studies of RGX-202 in mid-2018, encompassing colorectal cancer (including colon and rectal cancers) and gastric cancer.
RGX-019 is a monoclonal antibody targeting MerTK for the treatment of solid tumors. In collaboration with Rockefeller University and leveraging its proprietary microRNA platform, Rgenix identified MerTK (a receptor tyrosine kinase of the TAM family) as a novel target in triple-negative breast cancer (TNBC). Rgenix will conduct investigational new drug (IND)-enabling studies for RGX-019 in 2019.

Rgenix’s mRNA Platform (from Rgenix’s official website)
About Rgenix
Rgenix, founded in 2010 and headquartered in New York, USA, is a privately held clinical-stage biopharmaceutical company. Rgenix focuses on the development of novel anticancer drugs targeting key processes in cancer progression. Currently, the company is developing several first-in-class therapies to address the treatment needs of cancer patients. Rgenix’s microRNA platform, which enables the identification of novel cancer targets, represents a significant innovation in cancer biology.