Rexahn Pharmaceuticals, Inc. (NYSEMKT:RNN), a clinical stage biopharmaceutical company developing next generation therapeutics for the treatment of cancer, announced additional interim clinical data from an ongoing Phase IIa study of its novel anti-cancer drug candidate, Archexin® were recently presented at the 2016 American Society for Clinical Oncology (ASCO) Genitourinary Cancers Symposium.

“We are excited about the results from our ongoing Phase IIa study of Archexin in renal cell carcinoma, which continue to look promising,” said Dr. Ely Benaim, Chief Medical Officer for Rexahn.  “Interim data presented in November 2015 showed that at the lowest dose of Archexin tested (125 mg/m2/day), one patient had stable disease for over a year and a 16% reduction in the size of the patient’s primary tumor following four cycles of treatment.  The latest data show a reduction in tumor size of 36% in a second patient receiving 200 mg/m2/day of Archexin following 2 treatment cycles, suggestive of a dose and time-dependent clinical benefit.”

Stage 2 of the clinical trial — a randomized, open-label, two-arm dose expansion study of Archexin in combination with everolimus, versus everolimus alone, is expected to start in early 2016 and will further evaluate the efficacy of Archexin in metastatic renal cell carcinoma (RCC).  Rexahn has received U.S. Food and Drug (FDA) Orphan Drug Designation for Archexin for metastatic RCC as well as four other cancers.

Archexin Clinical Data

New interim data from the Phase IIa Archexin clinical trial were presented on Saturday, January 9, 2016 by study investigators, Drs. S. Tagawa, G. Chatta, N. Agarwal, and Rexahn scientists in a poster presentation entitled “Archexin, A Novel AKT-1 Specific Inhibitor for the Treatment of Metastatic Renal Cancer.”

The results indicated that at the dose levels tested to date, Archexin appeared to be safe and well tolerated.  The most commonly reported adverse event in patients taking both Archexin and everolimus is thrombocytopenia.  To date, no adverse events have been dose limiting.

Early evidence of the potential clinical activity of Archexin in combination with everolimus has been observed. Among the patients enrolled in the study, three patients have experienced stable disease, which has persisted for 383, 170 and 115 days (as of January 6, 2016).  In addition, two patients have experienced a reduction from baseline in their tumor size of 16% (125 mg/m2/day) and 36% (200 mg/m2/day) following four and two treatment cycles, respectively.

Scott Tagawa, MD, MS, Medical Director, Genitourinary Oncology Research Program, Richard A. Stratton Associate Professor in Hematology & Oncology, Weill Cornell Medicine, commented, “We continue to be encouraged by the data from the Phase IIa clinical study, suggesting a potential clinical benefit of Archexin in combination with everolimus.  Its novel mechanism of action and potential synergistic benefit, when used in combination with everolimus, make Archexin a promising new anti-cancer drug candidate.  By suppressing AKT-1 — an increasingly well understood cancer pathway — it is possible that Archexin could both inhibit the growth of cancer cells and also play a role in overcoming resistance to mTor inhibitors, which would be an important breakthrough in cancer therapy if confirmed in future clinical study.  I look forward to completing Stage 1 of the current clinical trial and commencing the randomized phase of the study.”

The Phase IIa clinical study is designed to evaluate the efficacy of Archexin in combination with everolimus (Afinitor®) to treat metastatic RCC patients and is being conducted in two stages. Stage 1 is an open-label, dose-escalation study designed to identify a safe and tolerable dose of Archexin when given in combination with everolimus. Stage 2 is a randomized, open-label, 2-arm dose expansion study of Archexin in combination with everolimus versus everolimus alone to determine safety and efficacy of the combination.

In Stage 1, escalating doses of Archexin of 125, 200 and 250 mg/m2/day are administered by continuous IV infusion for 14 days followed by 1 week of rest.  Based on previous clinical data, the target dose of Archexin is anticipated to be no more than 250 mg/m2 per day. Patient assessments include safety, pharmacokinetics, laboratory and physical exams.  Once the maximum tolerated dose of Archexin in combination with everolimus has been determined, thirty RCC patients will be randomized to receive either Archexin in combination with everolimus, or everolimus alone, in a ratio of 2:1.

The primary endpoint of Stage 2 is the percentage of progression free patients following eight cycles of therapy. Patients are scanned (CT or MRI) for the assessment of tumor progression after every 2 cycles of therapy.  Secondary endpoints include pharmacokinetic profile, incidence of adverse events, changes in clinical laboratory tests and vital signs over time, tumor response, duration of response, time to response, and response rate. Exploratory endpoints include blood levels of AKT pathway biomarkers, tumor apoptosis biomarkers, or other relevant biomarkers.

In preclinical studies, Archexin has been shown to inhibit the growth of human renal cell carcinoma cells in tissue culture. Archexin has also been shown to exhibit an additive anti-tumor effect when combined with other cancer drugs in inhibiting the growth of human RCC cells in tissue culture. (Original Source)

Shares of Rexahn Pharmaceuticals closed last Friday at $0.335, down $0.01 or -4.26%. RNN has a 1-year high of $0.96 and a 1-year low of $0.33. The stock’s 50-day moving average is $0.41 and its 200-day moving average is $0.50.

On the ratings front, Roth Capital analyst Joseph Pantginis maintained a Buy rating on RNN, with a price target of $3, in a report issued on November 6. The current price target implies an upside of 782.4% from current levels.

According to, Pantginis has a total average return of -10.3%, a 32.6% success rate, and is ranked #3592 out of 3610 analysts.

Rexahn Pharmaceuticals Inc is a clinical stage biopharmaceutical company dedicated to the discovery, development and commercialization of treatments for cancer and other medical needs.