Company develops personalized immune therapies
Minimizing Side Effects
Northwest Biotherapeutics, Inc., a Bethesda, Maryland-based clinical stage biotechnology company, focuses on the development of personalized 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. The company claims that its proprietary manufacturing technology enables the production of the personalized vaccine in an efficient, cost-effective manner.
Northwest Biotherapeutics has a broad platform technology for DCVax dendritic cell-based vaccines. Its lead product, DCVax-L, is currently in a 348-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. Northwest Biotherapeutics has also conducted a Phase I/II trial with DCVax for late-stage 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.
In May Northwest Biotherapeutics reported that an application for certification of the manufacturing facility in Sawston, UK, was submitted to the Medicines and Healthcare Products Regulatory Agency (MHRA), requesting the certification of the Sawston facility to produce Good Manufacturing Practice (GMP) clinical grade medical products for patients. This application represents the culmination of more than 2 years of preparations, including more than a year conducting these efforts under challenging COVID-19 restrictions and conditions.
Preparations have included the physical buildout of Phase I of the facility, development of more than 500 regulatory documents (including Standard Operating Procedures (SOPs) for all aspects of operations, batch manufacturing records and other formal documents), development of a Sawston team of nearly 40 persons with all of the required types of expertise for production of Advanced Therapy Medicinal Products (ATMPs) and training in regard to the DCVax® technology and processes.
The next step will be an on-site inspection of the facility by MHRA. Following the inspection, a report of the inspection results and any findings needing any corrective action will be issued by MHRA. Following completion of any actions, the company hopes that an initial license will be issued and production of GMP DCVax-L products in the Sawston facility may begin by around the end of Q3.
In addition to the MHRA submission activities, a prototype of the Flaskworks system has been delivered to the Sawston facility. Northwest anticipates that initial practice runs with the system may begin at the Sawston facility during the summer. Meanwhile, manufacturing of vaccine products for compassionate use patients (“Specials”) has been and is continuing at the GMP facility in London.
The process outlined in the company’s October 5, 2020 announcement relating to the Phase III trial of DCVax-L is continuing to move forward. The process includes review and analysis of the raw data by independent statisticians and experts, and preparation of summaries of the trial results for review by the company, the principal investigator, the steering committee of the trial, the scientific advisory board and a panel of independent brain cancer experts in preparation for public announcement and scientific publication. Northwest continues to be in a quiet period while this process is under way.
The database for the Phase III trial of DCVax®-L for Gliobastoma was locked in October. The independent service firms managing the Clinical Trial arranged for the independent statisticians to have access to the unblinded raw data from the trial.
Neither the company nor any party other than the independent statisticians had access to any unblinded data at that stage. The statisticians proceeded as quickly as possible with analyses of the raw data and prepared summaries of the trial results for review by the company, the principal investigator, the steering committee of the trial, the scientific advisory board and a panel of independent brain cancer experts who had the responsibility to analyze the data with the statisticians in preparation for public announcement and scientific publication.
Linda Powers, the company’s CEO, said, “We are excited to be so close to the finish line now, after such a long road. We are hopeful that DCVax®-L can become an important new treatment option for patients who urgently need more and better treatments for Glioblastoma brain cancer.”
She added, “We are grateful to the independent service firms and the clinical trial sites who have worked so hard to complete the data collection and confirmation during many months of COVID restrictions and challenges. We are also very grateful to our shareholders for their patience and support, which has made all this possible.”
Is Northwest Biotherapeutics a good investment? There are various viewpoints.
Zhiyuan Sun of The Motley Fool, commented, “The company's flagship candidate is an immunotherapy known as DCVax-L, which is under investigation for the treatment of glioblastoma multiforme, the most deadly form of brain cancer. The phase 3 study has been going on since December 2006 (almost 15 years now)… Northwest was supposed to conduct two interim analyses of the trial data starting in December 2013. Yes, you read that right, December of 2013. By August 2014, however, the company had released a statement claiming that ‘no interim analysis of efficacy in the Phase III trial [had] been done.’ What's more, the FDA halted the DCVax-L clinical trial in August 2015 before lifting it two years later.”
He added, “The company did eventually perform the interim analyses in 2017 and 2018, but released the results unblinded. When it comes to deadly diseases with significant unmet needs, clinical trials can be stopped early if the experimental drug meets the primary endpoint for ethical reasons. That did not happen with DCVax-L on both occasions … Last October, Northwest finally announced that the trial was coming to its end and that data was to be sent for statistical analysis. It's been more than nine months since the announcement, and no news. But, again, from an ethical endpoint, if DCVax-L was working, it doesn't make sense that Northwest wouldn't release top-line data and rush the drug to approval to save lives.”
However, Amsden Lindsay of Investors Hub reported that Northwest Biotherapeutics had an 8.59 percent increase on July 12, creating the largest rise in the past two months. As he explained, “DCVax-L is the company's lead product for Glioblastoma multiforme, the most lethal form of primary brain cancer. The company has completed two phase I/II trials and is now well under way with a large phase III trial. Glioblastoma multiforme represents a potential market that it is expected to reach $1.4 billion by 2025. There have been indications that the top-line data for DCVax-L will be overwhelmingly positive. If the phase III trial of DCVax-L reached the primary endpoint, there is no limit to how high the company can go.”
He concluded, “The company is also pursuing development of DCVax-Direct for inoperable solid tumor cancers. It is currently conducting a 60-patient Phase I/II trial of DCVax-Direct for all types of inoperable solid tumors. The trial is under way at MD Anderson in Houston, TX and MD Anderson in Orlando, FL, with additional sites in varying stages of preparation. With all of this in mind, NWBO is a penny stock that investors should consider watching closely. With all of this in mind, NWBO is a penny stock that investors should consider watching closely.”