Rexahn's Pancreatic Cancer Drug Receives FDA Orphan Status
Clinical stage biopharmaceutical company Rexahn Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has designated Orphan Drug status to RX-3117 as a treatment for patients with pancreatic cancer.
RX-3117 is a novel, small molecule anti-metabolite incorporated into cell DNA or RNA. The drug inhibits DNA/RNA synthesis to trigger apoptotic cell death of tumor cells. RX-3117 also influences the downregulation of a certain enzyme targeted for cancer therapy. Results from preclinical studies demonstrate that the drug is able to inhibit the growth of different human cancer xenograft models and overcome drug resistance in chemotherapy.
Dr. Peter D. Suzdak, Rexahn's CEO, said that the designation represents a major step in the drug’s clinical development. “RX-3117 has shown to be effective in treating cancer cells that have become gemcitabine resistant. If the results of our Phase 1b trial and future trials show similar results in gemcitabine resistant patients, this would represent a major breakthrough in the treatment of pancreatic cancer patients.”
In May, the company announced additional data from preclinical studies showing the drug’s efficacy in animal models with tumors which have become resistant to gemcitabine. Gemcitabine, marketed by Eli Lilly as Gemzar, is observed to be a suboptimal therapy due to its 5.4 percent response rate in pancreatic cancer and its ability to prolong progression-free survival by approximately four months. However, growing resistance against gemcitabine gives Rexahn’s RX-3117 a better chance to improve treatment outcome for patients with pancreatic cancer. Analysts predict a bright future for RX-3 117, thanks to its promising tumor metabolic action against 80 distinct human cancer cell lines, including brain, lung, breast, renal, bladder, and ovarian cancer, among others.
The FDA’s orphan drug designation confers the sponsor drug with a range of benefits including exemption from application fees and tax credits for qualified clinical studies.
The company has previously stated that it is seeking partners for the development of RX-3117. Roth Capital noted that the new orphan status could jumpstart discussions for a drug development partnership soon.