BioCancell Announces Success in Phase I/IIa Pancreatic Cancer Clinical Trial
BioCancell demonstrates significant decrease in local pancreatic tumors and prevention of metastatic disease in most patients treated with the higher of two dosages tested and tested after three months.
- BC-819 demonstrated an excellent safety profile
- In 60% of patients treated with the higher dosage and examined after three months, the local pancreatic tumor shrank significantly
- Two patients with non-resectable (inoperable) pancreatic cancer became resectable after treatment, and one underwent a successful operation
- BioCancell has been granted “orphan drug” status from the FDA for use of BC-819 in pancreatic cancer
- BioCancell is preparing to apply to the FDA to commence a Phase IIb clinical trial to test the sequential use of BC-819 and Gemzar, after this combination showed greater efficacy on animals than Gemzar alone
JERUSALEM--(BUSINESS WIRE)--Nov 1, 2010 - Success for biopharmaceutical company BioCancell (TASE:BICL): BioCancell, a developer of anti-cancer drugs, announced success in attaining the goals of its Phase I/IIa clinical trial of BC-819 as a treatment for pancreatic cancer, which demonstrated an excellent safety profile and preliminary efficacy. The primary endpoint, drug safety, was achieved with no side effects definitely connected to BC-819, and no patients complaining about pain or discomfort as a result of the drug.
A secondary endpoint was preliminary efficacy of the treatment on the local pancreatic tumor alone. Results show that within a month from commencement, the local tumor had not increased in size in 8 out of the 9 patients, and of those already examined three months after commencement, 3 out of 5 (60%) of the higher-dose cohort showed significant shrinkage of the tumor and no appearance of metastases (the RECIST criteria for Partial Response). Two additional patients met the criteria for Stable Disease (no significant change in the size of the pancreatic tumor and no appearance of metastases) after three months. In total, 5 out of 8 patients (62.5%) already tested after 3 months showed significant tumor reduction or stability, together with prevention of metastases. The results show that the higher dosage given in the trial (8 mg), shows greater efficacy than the lower dosage given (4 mg).
Following treatment with BC-819, two patients in the higher dosage group that were non-resectable became resectable as a result of tumor shrinkage. The first showed reduction of 15% in tumor length and had the tumor successfully removed at the University of Maryland Medical Center, Baltimore, MD, USA. The second showed reduction of 52% in tumor length, and was operated upon, but the discovery of liver metastases during the operation prevented its successful completion in order to prevent risk to the patient. An operation to remove pancreatic tumors (the Whipple procedure) is the best chance for survival a patient can be offered, but most patients are not operable on account of the complexity, location and size of the tumor.
The Phase I/IIa clinical trial included nine patients that each received four doses of BC-819, twice per week for two weeks, in the USA at the University of Maryland Medical Center, MD, and in Israel at Hadassah, Sheba, Meir and Hillel Yafe Medical Centers. Three patients received the lower dose (4 mg), while six others received the higher dose (8 mg).
It is common to examine drug combinations for the treatment of cancer, and the combination of BC-819 and Gemzar is designed to reach maximum efficiency in treating a disease with a high level of mortality. Results of pre-clinical testing has shown that in animals treated with the sequence of BC-819 and Gemzar, the volume of tumors shrank significantly, in comparison with the animals treated with Gemzar alone. In addition, there was no appearance of metastases in the animals treated with the sequential administration of BC-819 and Gemzar, while 63% of animals treated with Gemzar alone showed metastatic growths. These results gain special importance on account of BioCancell's intention to apply to the Food and Drug Administration (FDA) to commence a Phase IIb clinical trial of BC-819 in sequence with Gemzar, for the treatment of pancreatic cancer.
Uri Danon, CEO of BioCancell, said today that, “The safety and efficacy results that BC-819 has demonstrated in this pancreatic cancer clinical trial, shows the potential of BioCancell's technology, with most participants that received the higher dose and were tested three months after treatment initiation, showing tumor shrinkage or stability and a prevention of metastases. We believe that the results of this trial, together with the results of our pre-clinical BC-819/Gemzar combination study that showed better efficacy than either of the drugs alone, show a firm basis for continued proof of efficacy in the upcoming Phase IIb trial.”
BioCancell's Technology - Targeted Cancer Therapy
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.
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 is, 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.
About the H19 Gene
Discovered in human cancer by Professor Avraham Hochberg, H19 is an oncofetal gene that encodes RNA (with no protein product) that is expressed at high levels in over 40 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 Targeted Cancer 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 20 years.
BioCancell's securities are traded on the Tel Aviv Stock Exchange (TASE:BICL), with the major stockholders being Clal Biotechnology Industries, a member of the IDB group of companies, and Professor Hochberg.
For more information, please visit http://www.biocancell.com.
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, pancreatic cancer and ovarian 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 and other factors described in our public filings. BioCancell does not undertake any obligation to update forward-looking statements.
Contact: BioCancell Therapeutics
Uri Danon, CEO
Posted: November 2010