March 13, 2012 – Jerusalem, Israel — BioCancell Therapeutics, Inc. (TASE:BICL) today announced the results of its Phase I/IIa clinical trial of BC-819 as a treatment for patients with advanced ovarian cancer who have previously failed at least one standard treatment. The results show that BioCancell met the trial’s primary endpoint (treatment safety). In addition, at the highest dose given (240 mg), signs of efficacy were observed.
The trial took place in a number of medical centers in Israel, with 11 late-stage, heavily pretreated patients participating (following an average of four complete cycles of chemotherapy each). The patients each received one of three doses of BC-819 – 60 mg, 120 mg and 240 mg (the highest dosage group). As required by the FDA, the trial protocol was designed to focus on testing the safety of BC-819, as this was the first time that dosages of this magnitude had been used in a clinical trial, and that an intraperitoneal mode of administration was used. Therefore, the protocol included a 4-week break in the treatment in order to evaluate any possible adverse events.
The primary endpoint of the trial was evaluating the safety of the treatment (establishing the maximum tolerated dose and the presence or absence of any dose-limiting toxicity). As no dose-limiting toxicity was recorded, the optimal dosage was determined to be the maximum dosage given in the trial – 240 mg. In addition, the results show that no serious adverse events were linked to the drug, meaning that BioCancell has fully met the trial’s primary endpoint.
During the course of the trial, ascitic fluid levels dropped and the ascites disappeared for all patients in the high dosage group as long as they received treatment. Ascites are a fluid containing cancerous cells that builds up in the peritoneal cavity. They are a significant side effect for ovarian cancer patients that severely affect quality of life, and are an indicator of deterioration of the patient’s condition.
In light of these trial results, BioCancell will act to expand the trial by treating an additional group of patients at the highest dosage (240 mg), in a treatment regime designed to provide more information regarding treatment efficacy and safety, with details to be provided one regulatory approvals are received.
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.