Jerusalem, Israel — BioCancell Therapeutics, Inc. (TASE:BICL) today announced that the US Food and Drug Administration (FDA) has granted orphan drug status to BioCancell’s lead drug-candidate, BC-819 (currently in clinical trial stages), for use in treating pancreatic cancer.
An “orphan drug” is a drug for a disease that affects a relatively small number of people in a population. In the USA, an orphan drug is defined as one that treats a disease affecting less than 200,000 people each year. In order to encourage the development of drugs for such diseases, benefits and incentives can be granted to the drug developers. The main standard benefit for orphan drugs in the USA is the right to market the drug exclusively for 7 years from the date it is approved. Additional benefits include tax benefits on R&D expenses, and waived FDA fees.
As previously reported, BioCancell has been conducting a Phase I/IIa clinical trial for this application since November 2009, pursuant to an Investigational New Drug Approval from the FDA. The trial is expected to include nine patients, who each receive four treatments, twice per week for two weeks. The University of Maryland Medical Center in Baltimore, and Hadassah, Sheba and Meir Medical Centers in Israel are participating in the trial. Treatments are partially funded by the US-Israel Binational Industrial Research and Development Foundation (BIRD), which approved a grant in the amount of $950,000, or 50% of the predicted expenses of this project.
In January 2010, BioCancell reported completion of the first of two dosage cohorts in the clinical trial, with no treatment-related serious adverse events reported. Following a review of the safety results to confirm that the safety target of the first cohort had been achieved, the clinical monitor of the Clinical Trial approved the commencement of a second dosage cohort, with patients receiving the maximum planned dose of eight milligrams.
This orphan drug approval is in addition to a similar approval of orphan drug status for BC-819 for use in treating ovarian 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.
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.
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