Rexahn Initiates Phase Ib Clinical Trial of RX-3117 for Treatment of Solid Tumors

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RX-3117 has Shown Efficacy Against Gemcitabine-Resistant Human Cancer Cell Lines and a Broad-Spectrum of Anti-Cancer Activity

Rockville, MD, January 8, 2014 – Rexahn Pharmaceuticals, Inc. (NYSE MKT: RNN) a clinical stage biopharmaceutical company, announced today the initiation of a Phase Ib clinical trial to study the safety and efficacy of RX-3117 in cancer patients with solid tumors.

RX-3117 is a next-generation cancer cell specific drug candidate that possesses a broad spectrum of anticancer activity with reduced side effects. In an exploratory Phase I clinical trial conducted in Europe, RX-3117 demonstrated oral bioavailability, and safety and tolerability in cancer patients with solid tumors. The compound has also shown efficacy in human cancer cell lines resistant to gemcitabine (one of the most widely used chemotherapy drugs on the market today). Resistance to the anti-cancer effects of gemcitabine represents a major clinical issue in the treatment of cancer patients, as it has been estimated that up to 25% of cancer patients receiving one or more cycles of gemcitabine rapidly become resistant to its anti-cancer activity.

The Phase Ib trial is a 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.

The trial is expected to enroll up to 30 patients from multiple sites in the United States. Patients will receive RX-3117 orally 3 times a week for 3 weeks followed by 1 week off, and will have the ability to continue on the drug for up to 8 cycles of treatment. The decision to enroll the next group of patients and escalate the dose will be made after 1 cycle of treatment, based on safety and tolerability. Patients will be assessed for tumor progression by CT or MRI prior to the start of therapy and after every 2 cycles of therapy. Rexahn expects to complete enrollment of patients by the end of 2014, and data is expected in the first half of 2015.

“The initiation of this Phase I clinical trial of RX-3117 represents an important milestone for Rexahn,” added Peter D. Suzdak, Ph.D., Rexahn’s Chief Executive Officer. “This compound is exciting to work with, as it has a unique profile with demonstrated activity against gemcitabine-resistant human cancer cells. We were pleased to see reduced side effect liability and oral bioavailability in cancer patients in our initial exploratory Phase I clinical trial conducted in Europe, and we believe that RX-3117 may represent a significant advancement in the treatment of cancer patients with solid tumors.”

Dr. Drew Rasco, MD, Clinical Investigator, South Texas Accelerated Research Therapeutics in San Antonio, TX commented, “Gemcitabine resistance represents a major problem in the treatment of cancer patients with solid tumors. RX-3117 represents a very interesting new approach for the treatment of cancer patients with solid tumors who have become resistant to gemcitabine.”

About RX-3117

RX-3117 is a nucleoside analog that is activated (phosphorylated) by Uridine Cytidine Kinase (UCK) and inhibits both DNA and RNA synthesis which induces apoptotic cell death of tumor cells. UCK is overexpressed in multiple human tumors, but has a limited presence in normal tissues. This unique specificity for cancer cells may lead to an improved safety profile in cancer patients. RX-3117 also mediates the downregulation of DNA methyltransferase 1 (DNMT1), which is an enzyme responsible for the methylation of cytosine residues on newly synthesized DNA and is also a key target for anti-cancer therapies. RX-3117 has broad spectrum anti-tumor activity against 80 different human cancer cell lines (including NSCLC, breast, ovarian, pancreas, colon, renal, brain, bladder, cervical) and efficacy in 12 different mouse xenograft models (including colorectal, non-small cell lung, pancreatic and renal cell carcinoma) superior to that of gemcitabine.

In August 2012, Rexahn reported the completion of an exploratory Phase I clinical trial of RX-3117 in cancer patients conducted in Europe. The trial investigated the oral bioavailability, safety and tolerability of RX-3117 in nine cancer patients with solid tumors. Groups of three patients received single doses of RX-3117 and were assessed for safety, tolerability and plasma exposure. In this study, oral administration of RX-3117 demonstrated an oral bioavailability of up to 56% and a plasma half-life (T1/2) of 14 – 21 hours. In addition, RX-3117 was safe and well tolerated in all subjects throughout the dose range tested with no post-dose adverse events, laboratory abnormalities or ECG changes emerging through 7 days of follow-up.

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 future operations and products 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 timing and success of clinical testing; the timing of the conduct of clinical testing; the timing of the receipt and disclosure of clinical data 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.

News Source : Rexahn Initiates Phase Ib Clinical Trial of RX-3117 for Treatment of Solid Tumors

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