November 27, 2013
VBL Therapeutics Receives FDA Fast Track Designation for Lead Compound VB-111

VBL Therapeutics, a clinical-stage biotechnology company committed to the development of novel treatments for cancer and immune-inflammatory diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead oncology drug VB-111, for prolongation of survival in patients with Recurrent Glioblastoma Multiforme (rGBM). GBM is an aggressive form of brain cancer which carries a very poor prognosis with current therapy. VB-111 was already granted Orphan Drug status for GBM in the U.S. and in Europe.

VB-111, given as a simple IV infusion, is a novel gene-therapy drug that targets endothelial cells in the tumor vasculature, acting as a “biological knife”.

Based on a non-replicating adenoviral vector, VB-111 harbors a proprietary promoter which regulates transcription of a Fas-Chimera transgene, leading to targeted cell-death of endothelial cells in tumor-feeding blood vessels, with no harm to normal vasculature and non-cancerous tissues in the body. VB-111 is the first agent based on transcriptional targeting of tumor endothelium to be assessed in a clinical trial.

VBL has recently published Phase I/II results for VB-111 in the journal of Clinical Cancer Research. The Phase I/II trial for VB-111 demonstrated safety and tolerability in patients with advanced metastatic cancer at a single administration. Notably, tumor response and superior overall survival were found in the 1×1013 VPs cohort compared to sub-therapeutic doses. The data confirmed pre-clinical findings in animal models and validated VB-111’s mechanism of action, resulting in a targeted expression of the Fas-Chimera transgene selectively in tumor vasculature. For access to the manuscript please see:
http://www.ncbi.nlm.nih.gov/pubmed/23589178

The Company has recently presented results from Phase II clinical study in patients with recurrent glioblastoma at the 2013 annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago in June 2013. Data from the trial evaluating the effect of VB-111 on 28 patients with recurrent Glioblastoma Multiforme (rGBM) demonstrate that VB-111 was safe and well tolerated with repeat doses of up to 1×1013 VPs. Tumor responses and significant attenuation of tumor growth rate were seen. Overall survival was 12 months, which is at least 3 months longer compared to historical data in rGBM with the standard of care with chemotherapy and/or anti-angiogenic agents.

VBL’s platform and clinical data also attracted significant attention at a satellite symposium on VB‐111 held at the 2013 Scientific Meetings of the Society for Neuro-Oncology (SNO) and the World Federation of Neuro-Oncology in San Francisco last week.

“The Fast Track designation is another significant milestone reached in our program for approval of VB-111 as a unique drug for rGBM. We are very pleased that the FDA recognized the potential for this novel therapy to treat this serious and devastating cancer” said Dror Harats, MD, VBL’s Chief Executive Officer. “Building on the promising data from our Phase 2 trial, we plan ahead for a pivotal trial with VB-111 for rGBM under the Fast Track program which should expedite the development of VB-111.”

In addition to GBM, VB-111 is also evaluated in multi-dose Phase 2 clinical trials for differentiated thyroid cancer and ovarian cancer.

About VB-111

VB-111 is a novel IV-administered anti angiogenic agent that utilizes VTS™, VBL’s proprietary platform technology to target endothelial cells in the tumor vasculature for cancer therapy. VB-111 contains a non-replicating adenovector, a proprietary modified murine pre-proendothelin promoter (PPE-1-3x) and a Fas-Chimera transgene. The modified promoter specifically targets the expression of the Fas-Chimera transgene to angiogenic tumor blood vessels, leading to their apoptosis. VB-111 is the first agent based on transcriptional targeting of tumor endothelium to be assessed in a clinical trial.

VB-111 has successfully completed a Phase I/II “all comers” clinical trial, which demonstrated

multiple cases of objective tumor response and disease control and excellent safety and tolerability. VB-111 has been advanced into tumor specific, repeat-dose trials in glioblastoma, thyroid cancer and ovarian cancer.

About VBL Therapeutics

VBL Therapeutics is an innovative, clinical-stage biotechnology company committed to the development of novel treatments for immune-inflammatory diseases and cancer. VBL has a proprietary Vascular Targeting System (VTS™) technology platform that has yielded VB-111, an anti-angiogenic agent for cancer, which is currently in multiple Phase 2 clinical trials. In addition, VBL has pioneered the Lecinoxoid class of oral anti-inflammatory agents. VB-201 is the company’s lead candidate from this program, currently in Phase 2 clinical development in patients with psoriasis or inflammatory bowel disease. Both programs target multibillion dollar markets with unmet need for safe and well-tolerated treatments, and provide differentiation from current or proposed treatments.

Founded in 2000, VBL is based in Tel Aviv, Israel. The company has more than 120 granted patents and more than 150 applications pending.

For more information on the company, please visit www.vblrx.com.