Rexahn Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its RX-3117 drug used to treat patients with pancreatic cancer.
RX-3117 is a small molecule anti-metabolite that is integrated into DNA or RNA of cells. It 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 synthesised DNA and is also a target for anticancer therapies.
Rexahn CEO Peter Suzdak said: "Receiving orphan drug designation for RX-3117 in the treatment of pancreatic cancer is an important milestone for this clinical development programme.
"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."
At present, the company is enrolling patients in a Phase Ib clinical trial in cancer patients, which is expected to be completed in the first quarter of 2015.
In preclinical trials, RX-3117 has maintained its anti-tumor activity in human cancer cell lines that are resistant to gemcitabine, a chemotherapy drug used to treat pancreatic cancer.
Around 40% of patients with pancreatic cancer who have been treated with gemcitabine became resistant to gemcitabine after 30 days.
Currently, the company has three clinical stage oncology candidates, Archexin, RX-3117 and Supinoxin.
Image: Micrograph of pancreatic ductal adenocarcinoma (the most common type of pancreatic cancer). H&E stain. Photo: courtesy of KGH.