Rgenix Treats First Patient in Phase 1 Trial of RGX-202

RGX-202 targets the creatine transporter SLC6a8. Creatine metabolism - driven by SLC6a8 and the creatine kinase CKB - has been implicated in the progression of gastrointestinal cancers. The SLC6a8/CKB pathway is activated by gastrointestinal cancers to generate the energy required to grow and spread throughout the body. By inhibiting SLC6a8, RGX-202 suppresses the growth of colorectal, gastric, and pancreatic cancers as a single agent and in combination with chemotherapy in preclinical models. RGX-202 has also demonstrated efficacy in KRAS mutant tumor models which are generally resistant to approved targeted therapies.

The Phase 1 a/b study is a dose escalation and expansion study of RGX-202 alone and in combination with chemotherapy. RGX-202 will be evaluated in approximately 60 participants with advanced gastrointestinal tumors who have had progressive disease on available therapies, or for which there are no standard systemic therapies with relevant clinical impact. In the expansion stage, additional patients with advanced gastrointestinal malignancies will be treated at the recommended Phase 2 dose, to characterize safety, efficacy, PK, and pharmacodynamics of RGX-202 as a single agent and as part of a combination therapy. A biomarker directed approach will be utilized to select patients for the trial most likely to benefit from RGX-202 treatment.

“The launch of our Phase 1 trial for RGX-202 is a result of our commitment at Rgenix to develop treatments for patients suffering from prevalent cancers that lack effective therapies,” said Roger Waltzman, MD, and Chief Medical Officer of Rgenix. He added: “We are very excited to start this new trial and move RGX-202 as our second drug candidate into the clinic, as this illustrates the strength of Rgenix’s proprietary RNA discovery platform, the diversity of our pipeline, and ultimately our dedication to the cause of addressing unmet medical needs in oncology.”

For more information about the clinical trial please visit: https://clinicaltrials.gov/ct2/show/NCT03597581

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, Lepu Medical, Oceanpine Capital, WuXi AppTec’s Corporate Venture Fund, Alexandria Venture Investments, and the Partnership Fund for New York City’s Innovate NY Fund and associated entities. 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, SLC6a8, is over-expressed in several prevalent cancer types, including gastrointestinal malignancies. RGX-202 has demonstrated anti-tumor activity in pre-clinical studies, both as a single agent as well as in combination with standard-of-care therapies.

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