Oncology Pipeline Overview
Rexahn’s mission is to develop highly targeted cancer therapies with the potential for enhanced efficacy and safety – an approach which we believe could improve the lives of cancer patients. Building on a strong foundation in cancer research and discovery, we have assembled a diverse portfolio of novel oncology assets that includes three clinical-stage investigational anti-cancer compounds currently in human clinical trials, and a robust oncology research and discovery platform.
RX-3117 is a next generation, orally bioavailable nucleoside analog that is activated (phosphorylated) by the enzyme Uridine Cytidine Kinase 2 (UCK2) and inhibits both DNA and RNA synthesis which induces apoptotic cell death of tumor cells. UCK2 is overexpressed in multiple human tumors, but has a very limited presence in normal tissues. This unique specificity for cancer cells may lead to an improved efficacy and safety profile in cancer patients. This profile differs from existing nucleoside compounds (such as the widely used chemotherapy gemcitabine), which are activated by the enzyme DCK which is expressed in both cancer cell and in normal healthy tissue leading to significant toxicities at therapeutic doses. RX-3117 has demonstrated broad spectrum anti-tumor activity against 100 different human cancer cell lines (including Non-Small Cell Lung Cancer (NSCLC), breast, ovarian, pancreas, colon, renal, brain, bladder, cervical) and efficacy in 17 different mouse xenograft models (including colorectal, non-small cell lung, pancreatic and renal cell carcinoma) superior to that of gemcitabine.
Supinoxin (RX-5902) is an orally administered, first-in-class, small molecule inhibitor of phosphorylated-p68 (P-p68). P-p68, which is selectively expressed in cancer cells and is absent in normal tissue, increases the activity of multiple cancer related genes including cyclin D1, c-jun and c-myc, and plays a role in tumor progression and metastasis. Over-expression of P-p68 has been observed in solid tumors, such as melanoma, colon, ovarian and lung.
Archexin® is a unique anti-sense drug candidate that specifically targets the cancer cell signaling protein Akt-1. Archexin is the only specific inhibitor of Akt-1 in clinical development. The activated form of Akt-1, which is involved in cancer cell growth, survival, angiogenesis, and drug resistance, has been shown to be present or elevated in more than 12 different human cancer cell lines, including pancreatic and renal cell carcinoma. By inhibiting Akt-1, Archexin has shown to inhibit the growth of human renal cell carcinoma cell lines and exhibit a longer survival benefit in the human renal cell carcinoma animal xenograft model. Thus, while Akt-1 is a very specific anti-cancer target, it may have broad therapeutic potential across multiple types of cancer.