Bioniche Life Sciences Inc., a research-based, technology-driven Canadian biopharmaceutical company, today announced that its first Phase III clinical trial with a formulation of Mycobacterial Cell Wall-DNA Complex (MCC), trademarked Urocidin, for the treatment of bladder cancer, has been authorized to continue the trial unmodified.
The Data Safety Monitoring Committee held its sixth scheduled meeting regarding this clinical trial last week. The role of this independent body is to confirm that the safety of enrolled patients is being appropriately addressed and that, from an efficacy point of view, there is an appropriate basis for continuing the trial. After its meeting, the Committee has again recommended that Bioniche "continue the trial unmodified until the next scheduled or triggered meeting". The next scheduled meeting of the Committee will be held in three months.
The protocol calls for 105 evaluable patients to be enrolled in the trial. These are patients with non-muscle-invasive bladder cancer that is refractory (unresponsive) to bacillus Calmette-Guerin (BCG) therapy. BCG, the standard of care for non-muscle-invasive bladder cancer, has limited efficacy, especially as second-line therapy. It is associated with a number of treatment-limiting side effects that include the potential for both local (bladder) and systemic mycobacterial infections.
Recruitment of patients continues in our first pivotal Phase III clinical trial with Urocidin. In this trial, patients with non muscle-invasive bladder cancer whose cancer is specifically refractory (unresponsive) to BCG will receive Urocidin in an open label study. Thirty-one urology centres in North America are participating in this study and enrolment is expected to be completed in the first quarter of calendar 2009.
The patients at participating sites that are eligible and who choose to participate in this trial represent a very small subset of the BCG refractory patient population in North America. In order to satisfy regulatory authorities, the trial protocol is quite specific and patients must meet numerous criteria to be eligible. A significant proportion of patients who qualified for inclusion in the study based upon local pathology assessment (per normal clinical practice) were later found to have cancer of a different grade or stage when assessed by a central pathologist (per the trial's protocol) and will have to be replaced. These patients will still be allowed to continue treatment and will be evaluated for safety and efficacy.
Ultimately, 105 evaluable patients must be enrolled in the trial, and disqualifications require the Company to recruit replacement patients. Data from the 105 evaluable patients involved in the trial, coupled with additional safety information to be collected from a comparative trial, will allow full results to be reported one year after recruitment is completed for the efficacy and safety datasets. These results may also support regulatory submissions under U.S. Food and Drug Administration (FDA)'s Accelerated Approval program.
This indication was given Fast Track designation by the U.S. Food and Drug Administration (FDA) in April of 2006. The Company commenced this trial in November, 2006. An investigators' meeting was held in April, 2007 and the 31st site was validated in June, 2008.
Bioniche is planning to conduct a second registration trial which will directly compare the efficacy and safety of MCC with BCG in the treatment of non-muscle-invasive bladder cancer. In September, 2007, the Company announced that an agreement has been reached with the U.S. Food and Drug Administration (FDA) under the Special Protocol Assessment (SPA) procedure on the design of the trial, including its endpoints, data analysis and conduct. It provides assurance that, if the trial endpoints are met, they will serve as the basis for product approval under a Biologics Licensing Application (BLA). An SPA gives a clear pathway to registration of MCC when the trial endpoints are achieved. This study received Fast Track designation by the FDA earlier this year.