July 21, 2011 – Jerusalem, Israel — BioCancell Therapeutics, Inc. (TASE:BICL) today announced that it had received approval from the Food and Drug Administration (FDA) to commence a Phase IIb pancreatic cancer clinical trial in the USA, using BioCancell’s lead product, BC-819 in sequence with the FDA-approved drug Gemzar (Gemcitabine), in patients with locally advanced unresectable pancreatic cancer who have never received treatment.
The clinical trial is expected to include approximately 100 participants in 25 to 30 medical centers in locations including the U.S.A., Europe and Israel. One group, comprised of half of the participants, is to be treated with the combination of BC-819 and Gemzar (the current standard chemotherapy for pancreatic cancer), while a second group is to be treated with Gemzar alone. The primary endpoint of the trial is progression-free survival.
An interim analysis to assess the safety of the treatment is planned after the recruitment of the first 18 patients, in order to determine the dose of BC-819 for the remainder of the clinical trial.
In advance of the commencement of the clinical trial, BioCancell will be required to receive regulatory approvals, including from local government authorities and participating medical centers.
The clinical trial comes in addition to two clinical trials currently being run by BioCancell – a Phase IIb clinical trial of BC-819 as a treatment for superficial bladder cancer, and a Phase I/IIa clinical trial of BC-819 as a treatment for ovarian cancer. It follows a Phase I/IIa clinical trial of BC-819 as a treatment for pancreatic cancer, and a pre-clinical hamster study comparing BC-819 used in sequence with Gemzar, to Gemzar alone.
BioCancell’s Technology – Patient-Oriented, Targeted Therapy
BioCancell’s technology is both Personal and Targeted. The approach is based on the identification of particular genes that are highly expressed only in tumors (“Target Genes”). The regulatory sequences of these Target Genes are used to drive the expression of a toxin gene exclusively within tumor cells, enabling targeted tumor-cell destruction, leaving normal cells intact. In effect, the plasmid acts as “smart bombs”, activated only inside their targets thus destroying only the cancerous cells, while leaving healthy cells intact.
The patient’s eligibility for the treatment is determined by analyzing the patient’s tumor for the expression of the specific Target Genes. The diagnosis of the expression of the Target Genes are, therefore, a prerequisite for treatment and is made possible through the Company’s proprietary diagnostic technology that enables detection of even a single malignant cell. Only those patients with high expression levels of the Target Genes in their tumor are eligible for treatment with high confidence of success.
The Company has designated two genes as Target Genes – H19 and IGF2.
Discovered by Professor Avraham Hochberg in humans, H19 is an oncofetal gene that encodes RNA (with no protein product) that is expressed at high levels in over 30 types of human cancer tissues, while existing at a nearly undetectable level in the surrounding normal tissues, thus making it an optimal weapon in the fight against cancer.
The gene is expressed abundantly in the human placenta and in several embryonic tissues, but is repressed post-natally and only re-expressed with the appearance of cancer, within cancer cells. Studies show that H19 fulfills an important role in the process of tumorigenesis, and it is thought that the gene enables tumor cells to survive and proliferate under stress conditions.
BioCancell Therapeutics Inc. is a biopharmaceuticals corporation specializing in the development of Patient-Oriented, Targeted Therapy for the treatment of numerous types of cancer. The Company’s proprietary technology constitutes a novel paradigm for the targeted destruction of cancer cells, with no effect on normal surrounding tissue and no observed side effects, allowing for long-term, safe treatment and prevention of cancer.
BioCancell was co-founded in 2004 by Professor Avraham Hochberg, Professor of Molecular Biology at the Hebrew University of Jerusalem, based on technology developed by him over the past 15 years.
BioCancell’s securities are traded on the Tel Aviv Stock Exchange.
This press release contains “forward-looking” statements, including statements with respect to the further development and potential safety and efficacy of BC-819, in the treatment of superficial bladder cancer and BioCancell’s development strategy. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. There are a number of important factors that could cause the results of BioCancell to differ materially from those indicated by these forward-looking statements, including, among others, the risk that the U.S. Food and Drug Administration may require changes to the protocols and informed consents for clinical trials of BC-819, which changes may have a material adverse effect on the timing of, and BioCancell’s ability to conduct, those clinical trials, risks related to the clinical advancement of its BC-819 plasmid, including, but not limited, to the risk that clinical trials for this product candidate may not demonstrate safety and efficacy sufficient to obtain the requisite regulatory approvals or to result in a marketable product and risks related to the potential for others to develop products containing or based on BC-819. BioCancell does not undertake any obligation to update forward-looking statements.