BioCanCell Announces Phase I/IIa Ovarian Cancer Trial Results

May 8, 2012 – Jerusalem, Israel — BioCancell Therapeutics, Inc. (TASE:BICL) today announced that it had been notified by the Food and Drug Administration that the primary endpoint of its Phase IIb clinical trial of the drug-candidate BC-819 as a treatment for superficial bladder cancer in patients refractory to standard treatment (BCG or chemotherapy), would be lack of recurrence of the disease, three months after the commencement of treatment, in at least nine of the 21 patients participating in the second part of the trial. Together with the 18 participants in the first part of the trial, the trial is expected to include 39 participants.

On May 2, 2012, the Company published the following additional information:

  • Of the first 12 participants treated (out of the 21 participants in the second part of the trial), there was lack of recurrence in nine (subject to the caveat below), and the Company has therefore already met the updated primary endpoint for the trial.
  • Of the 30 participants in the entire trial (first and second parts) who have completed three months after the commencement of treatment, 19 (63%) have demonstrated lack of recurrence.
  • Of the 25 participants in the entire trial who have completed 12 months after the commencement of treatment, 14 (56%) have demonstrated lack of recurrence.

In addition, in BioCancell’s Phase IIb clinical trial of BC-819 as a treatment for pancreatic cancer, eight patients have commenced treatment, and the results regarding the first 12 patients are expected later this year.After the trial is completed, BioCancell intends to disclose final results and the aforementioned data are subject to change. As discussed with the FDA, after the completion of the Trial, the aforementioned updated criterion will be analyzed for all 39 participants in the Trial.

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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.

H19 Gene

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.

About BioCancell

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.