BioCancell Receives FDA Fast Track Designation for BC-819 for Treatment of Bladder cancer Patients

JERUSALEM, ISRAEL--(Marketwired - Sep 10, 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 that the United States Food and Drug Administration (FDA) has granted Fast Track designation to BC-819 for use in bladder cancer patients. BC-819 is being developed as a treatment for non-muscle-invasive bladder cancer (NMIBC), and will enter two Phase III confirmatory studies in the first half of 2016. The FDA Fast Track designation has been granted for both of BC-819's planned Phase III indications: for patients who have failed treatment with BCG (the current standard of care) and for patients who are unresponsive or intolerant to BCG treatment and will be treated with BC-819 as a monotherapy.

The FDA Fast Track program is designed to expedite the development and review of drugs that demonstrate the potential to address unmet medical needs by treating serious or life-threatening conditions. Companies that receive Fast Track designation are allowed to submit sections of their final marketing application (BLA) on a rolling basis as data becomes available, expediting the FDA review process. They also benefit from more frequent interactions with the FDA review team, including meetings to discuss the drug's development plan to support potential drug approval.

Jonathan Burgin, Chief Executive Officer of BioCancell, stated, "It is encouraging that BC-819 has received FDA Fast Track designation. This is an important step towards initiating two Phase III studies in 2016, and we look forward to the opportunity to work closely with the FDA as we further the development of BC-819 as a potential new therapy for bladder cancer patients."

About BC-819

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 an ongoing 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, whom the FDA and the American Urology Association have identified as suffering from high unmet need. 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.

About BioCancell

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 the first half of 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.

FORWARD-LOOKING STATEMENTS

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.