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news.US-based Rexahn Pharmaceuticals has secured orphan drug designation from the US Food and Drug Administration (FDA) for its RX-3117, a new small molecule anti-metabolite, to treat patients with pancreatic cancer.
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Currently, the company is enrolling cancer patients in a Phase Ib clinical trial and expects to complete the trial in the first quarter of 2015.
In preclinical trials, RX-3117 has shown to maintain its anti-tumor activity in human cancer cell lines that are resistant to gemcitabine, a chemotherapy drug used to treat pancreatic cancer.
About 40% of patients with pancreatic cancer who have been treated with gemcitabine became resistant to gemcitabine after 30 days.
RX-3117 is incorporated into DNA or RNA of cells and inhibits both DNA and RNA synthesis which induces apoptotic cell death of tumor cells.
It also mediates the downregulation of DNA methyltransferase 1 (DNMT1), an enzyme responsible for the methylation of cytosine residues on newly synthesized DNA and also a target for anticancer therapies.
Rexahn chief executive officer Peter Suzdak said receiving orphan drug designation for RX-3117 in the treatment of pancreatic cancer is an important milestone for this clinical development program.
"RX-3117 has shown to be effective in treating cancer cells that have become gemcitabine resistant. If the results of our Phase Ib trial and future trials show similar results in gemcitabine resistant patients, this would represent a major breakthrough in the treatment of pancreatic cancer patients," Suzdak said.