News Releases

BETHESDA, Md., June 4, 2019 /PRNewswire/ -- Northwest Biotherapeutics (OTCQB: NWBO) (NW Bio), a biotechnology company developing DCVax® personalized immune therapies for solid tumor cancers, announced today highlights from Dr. Marnix Bosch's Program Update in the Industry Expert Theater presentation at the 55^th annual ASCO Conference, which was publicly webcast and was posted on the home page of the Company's website at www.nwbio.com.

Dr. Bosch provided an update about the Company's DCVax®-Direct Program for inoperable tumors, including new follow-up data about patient survival in the Phase I trial and new information about the next DCVax-Direct clinical trials for new disease applications. Dr. Bosch also provided an update about the Company's DCVax®-L Program for operable tumors, including the Company's progress on the road to unblinding the data from the Phase III trial in Glioblastoma, and new follow-up data from the Information Arm of patients who were not eligible for the Phase III trial because they had actual or apparent rapid tumor progression (recurrence) before they could even be enrolled in the trial.

Highlights included the following:

DCVax-Direct

• Follow-up data reveal that in the Phase I trial, 76% of the evaluable patients in the Phase I trial exceeded their expected survival time by an average of about 14 months.
• DCVax-Direct produces very large quantities of cytokines, and this correlates with patient survival.  The cytokines cause rapid influx of T cells into the tumors, massive tumor cell death and mobilization of systemic anti-tumor T cell responses.  
• Importantly, there has been no cytokine release syndrome or similar toxicity with DCVax-Direct, unlike with certain other types of cell therapies.
• The Company has been preparing for multiple new DCVax-Direct clinical trials, building on the encouraging results of the Phase I trial.  The first 2 of these trials will address severe unmet medical needs:  brain metastases of lung and breast cancer, and DIPG (cancer involving the brain stem) in children. 
• These 2 trials are anticipated to start in the coming months at leading cancer centers.  For example, the brain metastases trial has been designed together with the Mayo Clinic and will be conducted there.  The brain metastases trial has already been cleared by FDA, and the DIPG trial is in the process of FDA review and clearance.

DCVax-L Phase III Trial for Glioblastoma

• The Company is progressing through each of the stages of work needed to reach unblinding of the Phase III trial for Glioblastoma, in accordance with the roadmap provided by the Company in its November 19, 2018 announcement following the Society for Neuro-Oncology meeting. 
• After 5 months of work with independent statisticians, the draft Statistical Analysis Plan (SAP) is nearly complete at this point.  (The SAP contains all of the planned analyses for the data that will emerge from the Phase III trial.)  This has been the most labor intensive stage for the Company on the road to unblinding of the Phase III trial data.   
• To save time to the finish line, the independent CRO that manages the Phase III trial has been checking and validating all of the trial data, in parallel with the Company's work on the SAP.  This involves checking all of the data points in hundreds of pages of case report forms for each of the 331 patients.  The process has generated thousands of "queries" relating to any missing data or data questions, as is typical in a large trial.  This task is also nearly complete, too, with only a few hundred queries remaining to be resolved.
• As soon as the SAP is finished (including review by advisers), it will be submitted to the regulatory agencies for each of the 4 countries where the trial was conducted, for their comment and buy-in.  As soon as the SAP process is completed with the 4 regulatory agencies, and the simultaneous data checking is finished, the trial will be unblinded and the data analysis stage will take place and result in top line data.
• Follow-up survival data from the Information Arm patients who did not qualify for the Phase III trial are encouraging and appear consistent with the blinded interim data from the trial.  In the group of 25 Information Arm patients who had actual or apparent early tumor recurrence, the follow-up data showed that 40% of the patients lived for 3 years or more, 20% of the patients lived for 5 years or more, and 12% of the patients are still alive at 7 years. 
• This Information Arm survival data is especially encouraging since this group included patients who already had actual tumor recurrence as well as others who had the appearance of tumor recurrence but who could not be definitely determined.

A video of the webcast of Dr. Bosch's presentation, a copy of his presentation slides and this press release can be found on the home page of NW Bio's website at www.nwbio.com 

ASCO is the largest annual gathering of oncologists and related service and research providers, held every year at the beginning of June in Chicago. Some 35,000 oncologists from around the world travel to Chicago to exchange information and make presentations on the latest potential breakthroughs in cancer research and clinical trial results.

About Northwest Biotherapeutics

Northwest Biotherapeutics is a biotechnology company focused on developing personalized immunotherapy products designed to treat cancers more effectively than current treatments, without toxicities of the kind associated with chemotherapies, and on a cost-effective basis, in both the United States and Europe.  The Company has a broad platform technology for DCVax dendritic cell-based vaccines.  The Company's lead program is a 331-patient Phase III trial in newly diagnosed Glioblastoma multiforme (GBM).  GBM is the most aggressive and lethal form of brain cancer, and is an "orphan disease."  The Company is also pursuing a Phase I/II trial with DCVax-Direct for all types of inoperable solid tumor cancers.  It has completed the 40-patient Phase I portion of the trial, and is preparing for the Phase II portion.  The Company previously conducted a Phase I/II trial with DCVax-L for metastatic ovarian cancer together with the University of Pennsylvania.

Disclaimer

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," "design," "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 risks related to the Company's ongoing ability to raise additional capital, risks related to the Company's ability to enroll patients in its clinical trials and complete the trials on a timely basis, uncertainties about the clinical trials process, uncertainties about the timely performance of third parties, risks related to whether the Company's products will demonstrate safety and efficacy, risks related to the Company's and Cognate's abilities to carry out the intended manufacturing and expansions contemplated in the Cognate Agreements, risks related to the Company's ability to carry out the Hospital Exemption program and risks related to possible reimbursement and pricing.  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