Cambridge, MA, Feb. 9, 2018 – BioCanCell Ltd. (TASE: BICL), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapies to treat cancer, today announced it presented a final analysis of its Phase 2 trial in early stage bladder cancer entitled: “Phase 2 Trial of BC-819 Intravesical Gene Therapy in Combination with BCG in Patients with Non-Muscle Invasive Bladder Cancer (NMIBC)” during the bladder cancer poster session at the 2018 ASCO Genitourinary Cancers Symposium being held on February 8-10 in San Francisco, CA.
The Phase 2 study tested the administration of BC-819 with an induction course of BCG according to three schedules. The trial included 38 patients with NMIBC eligible for therapy with BCG. The mean age was 69 years, 92% were males. All patients had NMIBC that was resected prior to treatment, 16% of patients had CIS lesions, and 47% had multiple recurrences prior to participation. 42% had any prior intravesical therapy, and 29% of patients had prior BCG therapy. At the final analysis with a median follow-up of 18 months, the overall median time to recurrence was not reached. At the 3-month timepoint, the first assessment following the induction treatment, 95% of patients were recurrence-free, and none had progressed. At 6-months, 78% were recurrence-free and 5% had progressed. The 24-month recurrence-free and progression rates were 54% and 24%, respectively. Therapy was associated with 5 adverse events related to BC-819 (in 5 patients; 13%): 2 urinary tract infections (1 mild/1 moderate), 2 reports of hematuria (both mild), and 1 report of dysuria (mild) and 29 adverse events related to BCG. Three serious adverse events were reported, none of which were related to BC-819.
The abstract can be viewed online at https://meetinglibrary.asco.org/record/157519/abstract.
“These data demonstrate that administration of BC-819 in combination with BCG is feasible and exhibits clinically meaningful activity in all three induction schedules tested,” stated Sarel Halachmi, M.D., principal investigator of this study.
Frank G. Haluska, M.D., Ph.D., chief executive officer of BioCanCell added: “We are pleased with the results of the trial as it demonstrates the feasibility of administering BC-819 and BCG together. We intend to use data from this study as the basis for the conduct of our pivotal Phase 3 study of BC-819 and BCG, which we plan to initiate under an SPA later in 2018.”
BioCanCell’s lead product candidate, BC-819, is a double-stranded bacterial DNA plasmid construct containing the diphtheria toxin A chain engineered to be expressed under the control of regulatory sequences from the H19 gene. H19 is expressed mainly in cancer cells in adults. In cells where H19 transcription is active, such as bladder cancer cells, BC-819 selectively activates the synthesis of the diphtheria toxin resulting in highly selective tumor cell destruction. BioCanCell has successfully completed three clinical trials of BC-819, including the combination trial presented here. The development of BC-819 has received Fast Track designation from the U.S. Food and Drug Administration (FDA).
In 2018, BioCanCell plans the initiation of its registrational clinical trial program comprising two pivotal trials in patients with NMIBC. The trials are designed to address two related but distinct potential indications. The first trial is a single arm registrational trial of BC-819 monotherapy in patients whose disease is unresponsive to two courses of BCG treatment. The second study is a randomized controlled trial of BC-819 in combination with BCG in patients whose disease has failed a single course of BCG. This phase 3 trial has been granted a special protocol assessment (SPA) from the FDA.
About Bladder Cancer
Bladder cancer is a common illness, the ninth most common cancer worldwide and the fourth most common cancer in men in the United States (US) (American Cancer Society 2018). An estimated 81,190 new cases of bladder cancer are expected to occur in the US in 2018. Bladder cancer incidence is about 4 times higher in men than in women and almost 2 times higher in Caucasian men than in African American men (American Cancer Society, https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2018/cancer-facts-and-figures-2018.pdf). As most cases are detected early, the mortality rate for bladder cancer is only about 20% of its incidence, and its prevalence rate is high relative to its incidence rate. An estimated 17,240 deaths from bladder cancer will occur in 2018. Most bladder cancers are transitional cell carcinomas (TCCs), and most are superficial (i.e., do not penetrate the muscularis layer), hence the term non-muscle invasive bladder cancer (NMIBC). The estimated proportion of patients with bladder cancer that is non-muscle invasive in the US is 70-80%. Of these new NMIBC cases, approximately 70% are Ta (confined to bladder epithelium), 20% are T1 lesions (invasion of lamina propria), and 10% are Tis disease (carcinoma in situ [CIS] lesions). As many as 80% of patients with Ta disease can be expected to experience disease recurrence, and up to 45% of patients with T1 or CIS lesions will experience disease progression without treatment. Although most patients respond well initially to adjuvant BCG treatment after surgical resection of NMIBC, its efficacy diminishes upon repeated administrations, leaving patients with no effective treatment and in danger of disease progression.
BioCanCell is a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies to treat cancer, with offices in Cambridge, MA, and Jerusalem, Israel. The Company’s most advanced product candidate, BC-819, is in development as a treatment for early stage, NMIBC. For additional information please go to www.biocancell.com.
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.
Frank Haluska, M.D., Ph.D.
President and Chief Executive Officer
Ashley R. Robinson
LifeSci Advisors, LLC