Jerusalem, Israel, October 1, 2015, BioCancell Ltd. (TASE: BICL), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapies to treat cancer-related diseases, today announced the results of a pilot clinical trial using a combination of BC-819 and BCG in intermediate-risk and high-risk patients, in preparation for BioCancell’s Phase III clinical program for the treatment of bladder cancer.
Thirty-eight (38) patients were recruited and divided into two groups. The first, comprised of six patients, received sequential weekly treatments (a course of BC-819/PEI followed by a course of BCG). In the second group, thirty-two (32) patients received alternating treatments of BC-819 followed by BCG, once or twice weekly (89% were classified as high-risk and 34% were pre-treated). BioCancell plans to utilize the alternating-treatment regime in Phase III pivotal studies.
Analysis of the results received from all patients shows that the combination treatment is safe. No severe adverse events occurred related to BC-819/PEI. 77% of the alternating-treatment patients who completed three months from the commencement of treatment, were recurrence-free (up from 64% in BioCancell’s Phase IIb bladder cancer trial of BC-819/PEI alone). Only one additional patient of the 12 alternating-treatment patients (8%) who have already completed six months from the commencement of treatment, experienced recurrence. The high rate of patient response to the combination therapy is believed to reflect this therapy’s better efficacy, which BioCancell intends to investigate in its Phase III pivotal studies.
Jonathan Burgin, Chief Executive Officer of BioCancell, stated, “We are very pleased with the results of this combination therapy clinical trial. These results pave our way forward to the commencement of the Phase III pivotal study next year. We look forward to developing a leading new therapy to treat the unmet need of so many bladder cancer patients. ”
BioCancell’s lead product candidate, BC-819, is a double-stranded DNA plasmid construct that uses the H19 gene to activate the synthesis of diphtheria toxin after entering a cell in which H19 transcription factors exist, destroying only that cell. The result of this mechanism is highly-selective tumor cell destruction. BioCancell has successfully completed Phase I/IIa and Phase IIb clinical trials for BC-819 as a treatment for bladder cancer, as well as a pilot study of combination therapy using BC-819 and the current standard of care, Bacillus Calmette-Guérin (BCG). BC-819 is a Phase III-ready asset for the treatment of bladder cancer. In 2016, BioCancell plans to initiate two Phase III trials in intermediate- and high-risk non-muscle-invasive bladder cancer (NMIBC). The first Phase III study will be performed under an FDA Special Protocol Assessment, and will use BC-819 in combination with BCG for patients who have failed at least one course of BCG. The second Phase III study is designed for patients who are unresponsive or intolerant to BCG and will be treated with BC-819 as monotherapy. These indications cover about 70% of all NMIBC patients and received fast track designation from the FDA. Although most patients respond well initially to BCG treatment, its efficacy diminishes upon repeated administrations, leaving patients with no effective treatment and in danger of disease progression.
About Bladder Cancer
Bladder cancer is the fourth most common in men in the United States. In 2013, there were 577,000 cases in the United States. Annual incidence estimated for 2015 in the United States is 74,000. Approximately 75% of bladder cancer cases are superficial, not penetrating into the muscular layer, and are classified as Non-Muscle-Invasive Bladder Cancer (NMIBC). However, as many as 70% of NMIBC patients will suffer from recurrences within five years of treatment. The primary treatment objective for NMIBC patients is to slow or stop cancer recurrence and progression to muscle-invasive disease, which both increase risk of mortality considerably. Bladder cancer has the highest lifetime per-patient treatment cost of all cancers and one of the highest recurrence rates. BCG has been the standard of care for decades, but includes significant drawbacks such as high recurrence rates, side effects and resistance to the treatment.
BioCancell is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapies to treat cancer-related diseases. Its most advanced product candidate, BC-819, is under development as a treatment for non-muscle-invasive bladder cancer (NMIBC). BC-819 will enter two Phase III confirmatory studies in 2016. BioCancell is also developing a second generation drug, BC-821 for the systemic treatment of advanced malignant neoplasms. Preclinical studies of BC-821 have showed significant efficacy in different cancer animal models such as metastatic lung cancer and metastatic liver cancer. BioCancell’s third product candidate, BC-830, is a “liquid biopsy,” which is intended to replace invasive and costly cystoscopies for the follow-up of NMIBC patients.
BioCancell’s R&D activities build upon the research of Professor Abraham Hochberg of the Hebrew University of Jerusalem. Professor Hochberg isolated the human H19 gene and determined that the gene is expressed in over forty different forms of cancer. H19-based therapy combines a highly selective method of killing cancer cells with a strong safety profile. Each of BioCancell’s product candidates relies on a proprietary H19 technology platform, with the potential to provide benefits that are competitive with existing cancer treatment methods.
This press release contains “forward-looking statements” that are subject to risks and uncertainties. These forward-looking statements include information about possible or assumed future results of clinical trials, the anticipated effects of receiving Fast Track designation, the anticipated timeframe for conducting additional clinical trials and making regulatory submissions, and other strategic and business plans and objectives. These forward-looking statements are based on information BioCancell has when those statements are made or its management’s good faith belief as of that time with respect to future events, and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. These risks and uncertainties include, but are not limited to: the success of the approach to discover and develop prospective therapeutic products, which is new and may never lead to marketable products; a lack of history of commercial sales; a dependence on the success of BC-819, the development of which will require significant additional clinical testing before regulatory approval can be sought and commercial sales launched; a need to raise substantial additional funds to complete R&D activities; an ability to overcome scientific or technological difficulties that may be encountered and that may impede R&D activities; and an ability to obtain and maintain intellectual property protection for product candidates, including pursuant to licensed patents.
Kirin M. Smith
Chief Operating Officer
Chief Communications Officer