Rexahn Pharmaceuticals, Inc. (NYSEMKT:RNN), a clinical stage biopharmaceutical company developing next generation therapeutics for the treatment of cancer, announced today that final data from the dose-escalation segment (Stage 1) of a Phase Ib/IIa clinical trial of Archexin® were presented at the 2016 American Association for Cancer Research Annual Meeting (AACR) in New Orleans, Louisiana.
The Phase Ib/IIa clinical trial is evaluating Archexin, in combination with everolimus (a widely used chemotherapy drug) in patients with metastatic renal cell carcinoma (mRCC). The results were presented during the Late Breaking and Clinical Trials Session at AACR by Drs. S. Tagawa, G. Chatta, N. Agarwal and Rexahn scientists in a poster presentation entitled “Phase Ib/IIa Study of RX-0201 (Archexin®), A Novel Akt-1 Antisense Combined with Everolimus to Treat Metastatic Clear Cell Renal Carcinoma.”
“The results from the Archexin dose escalation clinical study are promising and offer an early signal of the potential safety and clinical activity of Archexin in combination with everolimus,” commented Scott Tagawa, MD, MS, Medical Director of the Genitourinary Oncology Research Program at Weill Cornell Medicine. “The combination of Archexin and everolimus is rational based upon potential complementary mechanisms of action. While the primary objective of the initial dose escalation phase of the study was not intended to provide an efficacy evaluation, we nevertheless observed evidence of stable disease and tumor burden reduction in a significant number of patients in the study. When you consider that these are patients with very advanced disease, who have stopped responding to other therapies, we believe these data are especially encouraging.”
Archexin Phase Ib/IIa Clinical Data
The results from Stage 1 of the Phase Ib/IIa study presented at AACR showed that in mRCC patients that have previously received multiple anti-cancer therapies, Archexin treatment produced both stable disease (which persisted for up to 383 days) and a reduction in tumor burden. Compared to baseline CT scans, three patients experienced reductions in the size of their tumors of up to 36%. At the lowest dose level of Archexin administered (125 mg/m2/day) one patient had a 16% tumor reduction after four cycles of treatment. At the second dose level (200 mg/m2/day) one patient experienced a 36% tumor reduction after two cycles of treatment. At the highest dose level (250 mg/m2/day), which has been determined to be the maximum tolerated dose, one patient had a 32% tumor reduction following six cycles of treatment.
In the present study Archexin appeared to be safe and well tolerated at each of the dose levels tested with no dose limiting adverse events. The most commonly reported adverse event in patients taking the combination of Archexin and everolimus was thrombocytopenia.
“The emerging clinical data from our Archexin program suggest a potentially unique profile in metastatic renal cell carcinoma with both a dose and time dependent clinical benefit,” said Dr. Ely Benaim, Chief Medical Officer for Rexahn. “Based on these encouraging results, and our identification of a maximum tolerated dose, Rexahn has commenced enrollment in the second part of the study, which is a randomized, open-label, two-arm dose expansion study to further evaluate the safety and efficacy of Archexin in combination with everolimus, versus everolimus alone. We look forward to the possibility of confirming these results in the randomized part of the study.”
The second part (Stage 2) of the Phase Ib/IIa study will enroll up to 30 patients with mRCC who will be randomized to receive either Archexin in combination with everolimus, or everolimus alone, in a ratio of 2:1. The maximum tolerated dose of 250 mg/m2/day of Archexin — identified in Stage 1, will be administered along with 10 mg of everolimus, versus 10 mg everolimus alone.
The primary endpoint in Stage 2 of the Phase Ib/IIa clinical trial 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 two 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.
Additional Poster Presentations at AACR
Rexahn scientists and their collaborators also presented two additional posters detailing advancements in the Company’s RX-3117 and Archexin-Nano programs.
In a poster presentation entitled “Inhibition of DNA Methyltransferase by RX-3117 Leads to Upregulation of Hypomethylated Targets,” Rexahn scientists presented new preclinical data further describing the novel mechanism of action of RX-3117, an orally bioavailable, small molecule, investigational anti-cancer therapy that targets a unique biological pathway implicated in the development and metastasis of numerous cancers.
In a separate poster presentation entitled “Folate Receptor-Targeted Lipid Coated Albumin Nanoparticles (F-LCAN) for Therapeutic Delivery of RX-0201 (Archexin), An Antisense Oligonucleotide against Akt-1,” preclinical study results of a nanoparticle-Archexin combination under development by Rexahn (Archexin-Nano) were presented. Preclinical results from in vitro cell studies, pharmacokinetic and biodistribution studies and in vivo xenograft efficacy studies showed increased potency and a prolonged duration of action for Archexin-nano, suggesting that the F-LCAN formulation could be an effective formulation for therapeutic delivery of antisense agents such as Archexin. (Original Source)
Shares of Rexahn are up nearly 4% to $0.334 in pre-market trading. RNN has a 1-year high of $0.77 and a 1-year low of $0.26. The stock’s 50-day moving average is $0.34 and its 200-day moving average is $0.39.
On the ratings front, Rexahn has been the subject of a number of recent research reports. In a report issued on March 31, FBR analyst Vernon Bernardino assigned a Buy rating on RNN. Separately, on February 26, Roth Capital’s Joseph Pantginis maintained a Buy rating on the stock .
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Vernon Bernardino and Joseph Pantginis have a total average return of -15.8% and 1.2% respectively. Bernardino has a success rate of 30.8% and is ranked #3776 out of 3812 analysts, while Pantginis has a success rate of 37.4% and is ranked #1259.
Rexahn Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company. It dedicates to the discovery, development and commercialization of innovative treatments for cancer and other medical needs. The company currently has three clinical stage oncology candidates, Archexin, RX-3117, and Supinoxin and a robust pipeline of preclinical compounds to treat multiple types of cancer. It has also developed proprietary drug discovery platform technologies in the areas of nano-medicines, 3D gold and times. The company was founded by Chang Ho Ahn on March 19, 2001 and is headquartered in Rockville, MD.