Findings pave the way for new human clinical trials, following strong results in Phase I bladder cancer
Jerusalem, Israel — BioCancell Therapeutics, Inc. (TASE:BICL) reported today that the BC-819 drug it developed for various forms of cancer has been proven effective in pre-clinical trials for treatment of ovarian cancer and metastatic liver cancer. BC-819 had previously proven effective and non-toxic in a Phase I clinical trial concluded in September 2007. This latest result will enable BioCancell to submit an FDA request to carry out human Phase I clinical trials in these additional cancer types.
BioCancell also reported interim results of pre-clinical trials in BC-820 and BC-821 – additional drugs being developed for the treatment of cancer. The results show efficacy of BC-820 in the treatment of ovarian cancer, and of BC-821 in the treatment of superficial bladder carcinoma. These drugs complement BC-819 and are expected to significantly enlarge the number of treatable patients.
BioCancell CEO Avi Barak said, “We are pleased that these positive results will enable us to proceed on the path of broadening the range of treatments available for different types of cancer. BioCancell Therapeutics is continuing to invest its efforts in new pre-clinical trials while making progress in human clinical trials in additional indications.”
A team of scientists will present BioCancell’s latest findings at the conference of the European Society of Gene and Cell Therapy between October 27th and 30th, in Rotterdam, the Netherlands. As co-chairman of the urology session, BioCancell’s Chief Scientist Prof. Avraham Hochberg will present the results of the Phase I/IIa clinical trial in BC-819 for bladder cancer, where 72% of patients responded to treatment, including those not receiving the optimal dose. The preliminary evaluation of the efficacy of BC-819 was that it has the ability to cause tumor ablation and regression at doses that are well tolerated, while preventing the appearance of new growths. The evaluation was considered especially significant given the fact that all participants had previously failed standard treatments for bladder cancer.
At the same time, scientists Doron Amit and Aya Mizrahi will present the positive pre-clinical results of BC-819 for use in ovarian and metastatic liver cancer, BC-820 for use in ovarian cancer and BC-821 for use in bladder cancer.
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.
BC-819 is BioCancell’s leading product. It is a plasmid in which H19 regulatory sequences drive the expression of Diphtheria Toxin A gene. Besides the treatment of bladder cancer, BC-819 is in the process of being evaluated as treatment for liver, pancreatic and ovarian cancer. A Phase I/IIa clinical trial in refractory bladder cancer led to the conclusion that it has the ability to cause tumor ablation and regression at doses that are well tolerated, while preventing the appearance of new growths.
BC-820 is a plasmid in which H19 regulatory sequences drive the expression of Diphtheria Toxin A and a protein from the cytokine family (TNF) for the destruction of cancerous cells. BC-820 has shown efficacy as a treatment for ovarian cancer in pre-clinical studies, including cases which were resistant to treatment with Diphtheria Toxin alone.
BC-821 is a plasmid in which IGF2 regulatory sequences drive the expression of Diphtheria Toxin A gene for the destruction of cancerous cells. BC-821 has shown efficacy as a treatment for superficial bladder cancer ovarian cancer in pre-clinical studies. BC-821 is a complementary treatment to BC-819, which is expected to enlarge the number of treatable cancer patients.
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.
In 2006, BioCancell successfully completed a private round of funding and an initial public offering totaling $8.5 million. Its securities are traded on the Tel Aviv Stock Exchange, with the major stockholders being Clal Biotechnology Industries, a member of the IDB group of companies, and Prof. Hochberg.
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.