NEW YORK, NY, USA I November 04, 2021 I Inspirna, Inc., a clinical stage biopharmaceutical company developing first-in-class small molecule and biologic cancer therapeutics, today announced a collaboration with Bristol Myers Squibb on a clinical trial of a combination therapy using RGX-104 (abequolixron), the Company’s small molecule agonist of the Liver X Receptor/Apolipoprotein E (“LXR/APOE”) pathway, and Yervoy® (ipilimumab), Bristol Myers Squibb’s Cytotoxic T-Lymphocyte Associated protein 4 (CTLA-4) inhibitor. Under the terms of the agreement, Bristol Myers Squibb will provide ipilimumab for a Phase 1b/2 expansion study investigating the combination therapy for the 2nd and 3rd line treatment of patients with metastatic endometrial cancer whose genomes possess the E2 or E4 APOE genetic biomarker, including those patients who have progressed on prior checkpoint inhibitor therapy. Inspirna will sponsor the study and be responsible for study costs.

RGX-104 is an orally administered LXR agonist that potently activates APOE protein expression in tumors to inhibit cancer progression. Approximately 40% of the human population harbors either the E2 or E4 variants of the APOE gene in their genomes. These APOE genetic variants can be readily identified by analyzing DNA from blood samples. The presence of either of the E2 or E4 variants has been shown to increase the likelihood of a favorable clinical response to RGX-104 treatment in some cancers, including endometrial cancer, in a Phase 1 clinical trial, thus providing a potential biomarker for RGX-104 therapy. RGX-104 is currently being investigated in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for dysregulation of the APOE gene, including small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC).

“We are proud to embark on this collaboration with Bristol Myers Squibb to uncover an additional possible application of RGX-104 beyond our current areas of study,” said Masoud Tavazoie, M.D., Ph.D., and Chief Executive Officer of Inspirna. “This collaboration validates the work we are doing with RGX-104, and follows our recent phase 1 combination dose escalation trial results, in which treatment with RGX-104 combination regimens resulted in a 60% response rate in patients with relapsed or refractory cancers that had progressed on prior checkpoint inhibitor therapy and possessed the E2 or E4 APOE genetic biomarkers. We look forward to working with Bristol Myers Squibb and unlocking the potential of RGX-104 as we aim to improve the lives of people suffering with cancer.”

Yervoy® is a trademark of Bristol Myers Squibb Company.

About RGX-104 (abequolixron)

RGX-104 (abequolixron) is an orally administered small molecule agonist of the Liver X Receptor (LXR) which activates expression of the APOE tumor suppressor protein. APOE expression becomes dysregulated (silenced) in the tumors of select patients with solid cancers. APOE dysregulation results in increased tumor angiogenesis (tumor blood vessel growth) as well as a shifting of the tumor myeloid cell population from immune-stimulatory to immune-suppressive, which are both counteracted by RGX-104. RGX-104 is currently being tested in a Phase 1b/2 clinical trial in combination with standard-of-care regimens in several lung cancer indications that are enriched for APOE dysregulation, including SCLC and NSCLC. Inspirna expects to present data from this clinical study in 2H 2022.

About Inspirna

Inspirna, 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. Inspirna identifies novel cancer targets using its RNA-based target discovery platform, RNA-DRIVErTM, which was originally developed by Inspirna’s scientific co-founders at The Rockefeller University and exclusively licensed to Inspirna. The Company brings together distinguished scientific founders, a seasoned board of directors, 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, Sixty Degree Capital, K2 HealthVentures, 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

SOURCE: Inspirna