IRVINE, CA – September 10, 2012 – Cold Genesys, Inc. (“CGI”, the “Company”) announced today that the Company, CGI, has reached agreement with the U.S. Food and Drug Administration (FDA) under its Special Protocol Assessment (SPA) process for the Company’s proposed Phase 2/3 pivotal BOND (Bladder Oncolytic virus for Non-muscle invasive bladder cancer Disease) Trial of intravesical (bladder instillation) CG0070 (a CGI proprietary adenovirus conditionally replicative competent oncolytic vector expressing GM-CSF) for non-muscle invasive bladder cancer patients with carcinoma-in-situ (“Cis”) or Cis with Ta and/or T1 (Ta and T1 are referring to non-muscle invasive bladder cancer tumor staging) who have previously failed BCG (Bacillus Calmette-Guerin, a standard intravesical treatment for high risk non-muscle invasive bladder cancer) and have refused cystectomy. The approval was based on the results of the Phase I/2 V0046 study, a single-arm, multicenter clinical trial that enrolled 35 patients with muscle invasive bladder cancer who have previously failed BCG. The SPA provides FDA agreement that the study design and planned statistical analysis of this Phase 2/3 pivotal trial adequately address objectives necessary to support a regulatory submission, provided other conditions, such as CMC compliance in phase 3 manufacturing of a comparable product which can be adequately tested in the phase 3 portion and success in a well conducted and internally consistent clinical trial that can reach a convincing statistical conclusion are satisfied. The FDA agreement also specifically addressed the importance of obtaining CMC approval of all the necessary analyses for products that need to be newly manufactured before the Company can initiate the Phase 3 portion of the trial.
“We are pleased to have reached this agreement with the FDA and on the design of this pivotal Phase 2/3 BOND trial of an intravesical treatment using CG0070 for non-muscle invasive patients with Cis or Cis with Ta and/or T1 after they have failed BCG therapy. The only other alternative for these patients is radical cystectomy, which carries an unacceptable percentage of life long morbidity as well as a small but significant numbers of mortality. Furthermore, the frequent and prolonged need for cumbersome and costly medical care and post-cystectomy specific personal hygienic conditions will certainly and severely affect the self-esteem of this elderly population. Practically no such patient wants a cystectomy.” said Dr. Alex Yeung, Cold Genesys’s President. “Key issues in this SPA are confirmation of intended positioning of CG0070 as a good bladder preservation therapy for these patients after they have failed the standard BCG treatment, which is likely to be found in about 50% of these patients after a follow up period of five years. In addition, since we will be using CG0070 products in storage that had been transferred to us from BioSante Pharmaceuticals to initiate the Phase 2 portion of the trial, we understand the importance of additional and stringent requirements as specified in this SPA (from FDA) for our Company to manufacture new and comparable products for the Phase 3 portion of the trial. We will be searching for the right partner for this manufacturing process practically as of now to ensure that the Phase 3 portion of our trial will not be delayed. As a conclusion, we are glad that we will be able to see if the high response rate and long durable responses found in the first phase 1/2 trial can be confirmed. We sincerely hope that CG0070 intravesical therapy, if successful in this trial, may eventually be able to satisfy the unmet medical need of this group of bladder cancer patients.”
About Special Protocol Assessments
A Special Protocol Assessment is a binding declaration between a sponsor and the FDA indicating that a proposed Phase 3 or Phase 2/3 study design, endpoints, and statistical analyses are acceptable to support regulatory approval of the product. The process is intended to increase the likelihood that -- if the specified clinical trial protocols from the SPA are followed, the clinical trial endpoints are achieved and there is a favorable risk-benefit profile -- trial data may serve as the primary basis of an efficacy claim in support of a New Drug Application (NDA) or a Biologics License Application (BLA). Final marketing approval in the U.S. will depend on study results, safety issues and an overall evaluation. More information on Special Protocol Assessments can be viewed at:
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm080571.pdf
CG0070 in Bladder Cancer
CG0070 was previously tested in a Phase 1/2 trial (V0046) of 35 patients with non-muscle invasive bladder cancer from 2005 to 2008. This previous study consisted of two phases, with the first phase as a dose escalation study of administering CG0070 by a single intravesical instillation while in the second phase CG0070 was administered in a weekly times six or a 4-weekly times three to six (depending on response at the third month) schedules. In this study, CG0070 was generally well-tolerated, with only a few Grade 3 adverse events, and which quickly resolved within a few days. Good response rate and durable response in a small group of non-muscle invasive bladder cancer patients, especially those associated with Cis, were noted. More information on the V0046 trial can be found in the ASCO GU session meeting at San Francisco (Feb 2-4, 2012) abstract #271 and ASCO annual meeting 2012 at Chicago (June 1-5, 2012) abstract #4593.
About Cold Genesys
Cold Genesys Incorporated is a privately held biopharmaceutical company with a product portfolio that is focused on delivering innovative conditionally replicative (Rb pathway defective molecule E2F as the viral E1A gene promoter) competent oncolytic adenovirus vector expressing GM-CSF for the treatment of patients with bladder cancer. Currently, the Company is developing CG0070 for non-muscle invasive bladder cancer patients with Cis and Cis with Ta and/or T1 disease after BCG failure. Cold Genesys, Inc. received the intellectual property rights and products related to CG0070 previously held by Cell Genesys, Inc. and BioSante Pharmaceuticals in a transfer and royalty agreement on November 15, 2010. Cold Genesys was launched in August 2010. The company’s shareholders include BioSante Pharmaceuticals (Nasdaq: BPAX).
For more information on the Company, please visit our website at www.coldgenesys.net
Safe Harbor
This press release may contain forward-looking statements with respect to business conducted by Cold Genesys, Inc. By their nature, forward-looking statements and forecasts involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Such forward-looking statements include those that express plan, anticipation, intent, contingency, goals, targets, or future developments and/or otherwise are not statements of historical fact. The words “potentially”, “anticipates”, “projects”, “expects”, “could”, “calls for”, and similar expressions also identify forward-looking statements. The Company does not undertake to update any forward-looking statements. Factors that could affect actual results include, without limitation, risks associated with:
- the Company’s ability to obtain necessary regulatory approval for its product candidates from regulatory agencies, such as the U.S. Food and Drug Administration and the European Medicines Agency;
- the safety and efficacy of the Company’s products or product candidates;
- the commencement and completion of any clinical trials;
- the Company’s assessment of its clinical trials;
- the Company’s ability to develop, manufacture, license, or sell its products or product candidates;
- the Company’s ability to enter into and successfully execute any license and collaborative agreements;
- the adequacy of the Company’s capital resources and cash flow projections, the Company’s ability to obtain sufficient financing to maintain the Company’s planned operations, or the risk of bankruptcy;
- the adequacy of the Company’s patents and proprietary rights; and
- the impact of litigation that has been brought against the Company.
SOURCE: Cold Genesys, Inc.
CONTACT:?Cold Genesys at www.coldgenesys.net
Address: 19800 MacArthur Blvd., Suite 690, Irvine, CA 92612
Tel: 949.200.7680
Fax: 949.288.6298
