BETHESDA, Md., Dec. 16, 2013 /PRNewswire/ -- Northwest Biotherapeutics, Inc. (NASDAQ: NWBO) ("NW Bio"), a biotechnology company developing non-toxic DCVax® personalized immune therapies for solid tumor cancers, today responded to inquiries from shareholders and investors about differences between NW Bio's technology, products and clinical trials and those of a Competitor who recently announced the results of a Phase II clinical trial with a dendritic cell vaccine for Glioblastoma multiforme (GBM). NW Bio described several areas of significant differences.
Technology Differences. A dendritic cell vaccine involves two main ingredients: the dendritic cells themselves (master cells of the immune system), and antigens (i.e., biomarker targets) associated with the particular cancer being treated. The antigens are the targets on the cancer that the dendritic cell vaccine mobilizes the immune system to attack. The antigen component of NW Bio's DCVax technology and products is different from the Competitor's in two ways which the Company believes are quite important.
Experience Differences. NW Bio's DCVax platform technology has been applied in two prior Phase I/II clinical trials for GBM, a small Phase I trial in metastatic ovarian cancer, a Phase I/II trial in late stage prostate cancer, a large number (over 100) prostate cancer patients in an academic setting, and a number of diverse compassionate use cases, over the course of more than ten years of clinical development. The Competitor's technology had only previously been applied in one Phase I trial with 16 patients.
Clinical Trial Differences. NW Bio is well under way with a 312-patient Phase III clinical trial. The trial has been approved by three separate, highly rigorous regulators: in the US, UK and Germany. The DCVax technology and the Phase III trial have been evaluated by the National Health System (NIHR) in the UK and "adopted" as a nationwide priority in the UK. NW Bio's Phase III trial is robustly designed. If the primary endpoint is met, it is anticipated that this may result in a p value of 0.01 one-sided (0.02 two-sided), with a power of 82%. A two-sided p value of 0.02 is well below (and hence stronger than) the p value of 0.05 which is generally needed for statistical significance. Such a p value would provide a substantial cushion in case the difference in PFS turns out to be less than was projected in determining the statistical powering in the design of the trial.
NW Bio's Phase III trial is also potentially powered for the secondary endpoint of overall survival (OS), and it has an interim analysis devoted solely to reviewing the size of the trial, which can enable increasing the size of the trial if desired. In addition to all these measures, the trial has built-in analyses of specified subsets of the patients in the trial.
In contrast, the Competitor's trial was a 124-patient Phase II trial. It was approved by only one regulator (FDA) and conducted only in the US. According to the Competitor's description of its trial, the trial was designed with a target of 9 months' difference in overall survival between the treated arm and the control arm in the trial. It is unclear to what extent a cushion was built into the statistical powering in this trial design.
Company Positioning Differences. NW Bio has undertaken years of work and devoted extensive resources to building many chances for success. The Company now has two potential major value-driver programs under way in parallel: its lead Phase III trial with DCVax-L for GBM brain cancer, and its large (60 patient) Phase I/II trial with DCVax-Direct for all types of inoperable solid tumors with a particularly significant efficacy endpoint (regression of existing inoperable tumors).
Each of these parallel programs are expected to have ongoing catalysts throughout 2014. Each of these product lines has the possibility for broad market potential: DCVax-L potentially for all or most types of operable solid tumors (i.e., tumors in any tissue of the body, which can be surgically removed) and DCVax-Direct potentially for all or most inoperable solid tumors (i.e., tumors in any tissues, which cannot be surgically removed). NW Bio has also painstakingly built its operations on two continents, in both the US and Europe – including manufacturing as well as clinical trials. Accordingly, today NW Bio has many potential catalysts and chances for success, including in the near term.
In contrast, the Competitor had only one main program under way (the Phase II trial for which the results were just reported), and only in the US. The Competitor has not yet built anything in Europe, and it is typically a multi-year process to do so.
Market Timeline and Access Differences. Since NW Bio is well under way with a Phase III trial now, and the Competitor will need to conduct a Phase III trial, and the Competitor's Phase III trial will need to be quite large if it is powered for results similar to those just announced from the Phase II trial, NW Bio believes it is now potentially at least 3 to 4 years ahead of the Competitor on the clinical development pathway.
NW Bio also has orphan drug status granted in both the US and Europe for DCVax-L for GBM and other glioma brain cancers. The orphan drug laws grant 7 years of market exclusivity in the US and 10 years of market exclusivity in Europe for products that are the first of their type to reach the market. Such market exclusivity could apply to NW Bio's DCVax-L. If so, the scope could be broad: for treatment with DCVax-L, there are no limitations based on either tumor characteristics or patient characteristics. Generally, any GBM patient can be treated with DCVax-L if their immune cells are adequate and just 2-3 grams of their tumor tissue (roughly similar in size to one sugar cube) can be obtained, to provide the antigens for the product.
"At NW Bio we have worked long and hard, and without major fanfare, to develop our technology, our robust clinical trial designs, and our many chances for success through multiple programs on two continents," commented Linda Powers, CEO of NW Bio. "Although there are never any guarantees of successful results in clinical trials, we hope to deliver strong results for both patients and investors through one or more of our multiple programs."
About Northwest Biotherapeutics
Northwest Biotherapeutics is developing cancer vaccines designed to treat a broad range of solid tumor cancers more effectively than current treatments, and without the side effects of chemotherapy drugs. NW Bio's proprietary manufacturing technology enables the Company to produce its personalized vaccine in an efficient, cost-effective manner. The Company has a broad platform technology for DCVax dendritic cell-based vaccines. The Company's lead product, DCVax-L, is currently in a 312-patient Phase III trial for patients with newly diagnosed Glioblastoma multiforme (GBM), the most aggressive and lethal brain cancer. The Company's second product, DCVax-Direct, is currently in a 60-patient Phase I/II trial for direct injection into all types of inoperable solid tumor cancers. The Company has also conducted a Phase I/II trial with DCVax for metastatic ovarian cancer together with the University of Pennsylvania. The Company previously received clearance from the FDA for a 612-patient Phase III trial with its third product, DCVax-Prostate, for late stage prostate cancer.
Statements made in this news release that are not historical facts, including statements concerning future treatment of patients using DCVax and future clinical trials, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "expect," "believe," "intend," "plan," "continue," "may," "will," "anticipate," and similar expressions are intended to identify forward-looking statements. Actual results may differ materially from those projected in any forward-looking statement. Specifically, there are a number of important factors that could cause actual results to differ materially from those anticipated, such as the Company's ability to raise additional capital, risks related to the Company's ability to enroll patient s in its clinical trials and complete the trials on a timely basis, the uncertainty of the clinical trials process, uncertainties about the timely performance of third parties, and whether the Company's products will demonstrate safety and efficacy. Additional information on these and other factors, including Risk Factors, which could affect the Company's results, is included in its Securities and Exchange Commission ("SEC") filings. Finally, there may be other factors not mentioned above or included in the Company's SEC filings that may cause actual results to differ materially from those projected in any forward-looking statement. You should not place undue reliance on any forward-looking statements. The Company assumes no obligation to update any forward-looking statements as a result of new information, future events or developments, except as required by securities laws.
|SOURCE Northwest Biotherapeutics|
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