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Rexahn Receives FDA Orphan Drug Designation for RX-3117 in Treatment of Pancreatic Cancer

ROCKVILLE, Md., Sept. 24, 2014 (GLOBE NEWSWIRE) -- Rexahn Pharmaceuticals, Inc. (NYSE MKT:RNN), a clinical stage biopharmaceutical company developing best-in-class therapeutics for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its drug RX-3117 for the treatment of patients with pancreatic cancer. RX-3117 is a next generation, cancer-specific nucleoside analog. Rexahn is currently enrolling patients in a Phase Ib clinical trial in cancer patients and expects to complete this trial in the first quarter of 2015.

The FDA's office of Orphan Drug Products grants orphan status to support development of medicines for rare disorders, defined as diseases that affect fewer than 200,000 people in the United States. Orphan drug designation provides Rexahn with certain benefits, including limited market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

Preclinical studies have shown that RX-3117 maintains its anti-tumor activity in human cancer cell lines that are resistant to gemcitabine, a chemotherapy drug used to treat pancreatic cancer. Gemcitabine resistance represents a major problem in the treatment of cancer patients. In pancreatic cancer patients who have been treated with gemcitabine, up to 40% of patients became resistant to gemcitabine after 30 days.

"Receiving Orphan Drug Designation for RX-3117 in the treatment of pancreatic cancer is an important milestone for this clinical development program," commented Rexahn's Chief Executive Officer Peter D. Suzdak, PhD. "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."

About RX-3117

RX-3117 is a novel small molecule anti-metabolite that is incorporated into DNA or RNA of cells and inhibits both DNA and RNA synthesis which induces apoptotic cell death of tumor cells. RX-3117 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. Preclinical studies have shown RX-3117 to be effective in both inhibiting the growth of various human cancer xenograft models, including colon, lung, renal and pancreas, as well as overcoming chemotherapeutic drug resistance.

RX-3117 has demonstrated a broad spectrum anti-tumor activity against 50 different human cancer cell lines and efficacy in 12 different mouse xenograft models. The efficacy in the mouse xenograft models was superior to that of gemcitabine. In addition, RX-3117 still retains its full anti-tumor activity in human cancer cell lines made resistant to the anti-tumor effects of gemcitabine. These findings have either been previously presented at the American Association of Cancer Research Meeting in 2012 or will be the subject of a peer reviewed publication to be published in late 2014. In August 2012, Rexahn reported the completion of an exploratory Phase I clinical trial of RX-3117 in cancer patients conducted in Europe, to investigate the oral bioavailability, safety and tolerability of the compound. In this study, oral administration of RX-3117 demonstrated an oral bioavailability of 56% and a plasma half-life (T1/2) of 14 hours. In addition, RX-3117 was safe and well tolerated in all subjects throughout the dose range tested. In January 2014, Rexahn initiated a Phase Ib multi-center dose-escalation study which will evaluate the safety, tolerability, dose-limiting toxicities and maximal tolerated dose (MTD) of RX-3117 in patients with solid tumors. Secondary endpoints will include characterizing the pharmacokinetic profile of RX-3117 and evaluating the preliminary anti-tumor effects of RX-3117. Rexahn has completed testing of three doses (30, 60 and 100mg) and is in the middle of enrolling the fourth dose group (150mg) for the Phase Ib trial.

About Rexahn Pharmaceuticals, Inc.

Rexahn Pharmaceuticals is a clinical stage biopharmaceutical company dedicated to developing best-in-class therapeutics for the treatment of cancer. Rexahn currently has three clinical stage oncology candidates, Archexin®, RX-3117 and SupinoxinTM (RX-5902) and a robust pipeline of preclinical compounds to treat multiple types of cancer. Rexahn has also developed proprietary drug discovery platform technologies in the areas of Nano-Polymer-Drug Conjugate Systems (NPDCS), nano-medicines, 3D-GOLD, and TIMES. For more information, please visit www.rexahn.com.

Safe Harbor

To the extent any statements made in this press release deal with information that is not historical, these are forward-looking statements under the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements about Rexahn's plans, objectives, expectations and intentions with respect to cash flow requirements, future operations and products, enrollments in clinical trials, the path of clinical trials and development activities, and other statements identified by words such as "will," "potential," "could," "can," "believe," "intends," "continue," "plans," "expects," "anticipates," "estimates," "may," other words of similar meaning or the use of future dates. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. Uncertainties and risks may cause Rexahn's actual results to be materially different than those expressed in or implied by Rexahn's forward-looking statements. For Rexahn, particular uncertainties and risks include, among others, the difficulty of developing pharmaceutical products, obtaining regulatory and other approvals and achieving market acceptance; the success and design of clinical testing; and Rexahn's need for and ability to obtain additional financing. More detailed information on these and additional factors that could affect Rexahn's actual results are described in Rexahn's filings with the Securities and Exchange Commission, including its most recent annual report on Form 10-K and subsequent quarterly reports on Form 10-Q. All forward-looking statements in this news release speak only as of the date of this news release. Rexahn undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

CONTACT: The Trout Group LLC Tricia Truehart (646) 378-2953 ttruehart@troutgroup.com

Source:Rexahn Pharmaceuticals