October 9, 2017
By Mark Terry, BioSpace.com Breaking News Staff
VBI Vaccines (VBIV), which has headquarters in Cambridge, Mass., has a lot going on right now. Focused on vaccine development for infectious disease with at least one foot in the immuno-oncology space, VBI Vaccines has a vaccine for hepatitis B, Sci-B-Vac approved for use in Israel and 14 other countries, and clinical programs for cytomegalovirus (CMV) vaccines and glioblastoma multiforme (GBM).
Sci-B-Vac has recently started Phase III trials in the U.S., Canada and Europe. Since the vaccine has been effectively and safely used by 300,000 people, primarily in Israel and Hong Kong, VBI Vaccines is reasonably confident it will be approved in additional countries without too many problems. Its Investigational New Drug (IND) application was accepted on August 30.
On July 27, the company announced positive data from an interim data readout from its Phase I trial of its preventative vaccine for CMV. And on Aug. 15, the company announced that the U.S. Food and Drug Administration (FDA) had accepted its IND for VBI-1901, a novel immunotherapy that targets glioblastoma multiforme (GBM), one of the most common and aggressive types of brain tumors. It plans to start a multi-center Phase I/IIa clinical trial this year.
The company is clearly busy, but its president and chief executive officer, Jeff Baxter, took time out to talk to BioSpace about the company and where it’s headed.
The company’s technical platform is eVLP, which allows it to design enveloped virus-like particle vaccines. “It’s a third-generation VBL platform. I know you probably hear a lot of people saying they have a third- or fourth-generation product, but what does that really mean?” Baxter says.
In the case of VBI’s eVLP, it goes back to the traditional way licensed vaccines are developed. About 90 percent of viral vaccines, Baxter says, “take the non-harmful part of the virus, and through vaccination, elicit a natural immune response, generating neutralizing antibodies and B cells and in some cases T-cell responses against the foreign virus. We call ours a third-generation eVLP because it’s an envelope-like particle.”
The company creates an “envelope” made up of a lipid bilayer, which allows the construction of a virus-like particle that closely mimics the type of viruses found in nature. “Through that virus presentation, (it) allows us a much more immunogenic response by the human immune system. Also, the presence of this liquid bilayer allows us to either entrap antigens or adjuvants inside the particle, or to bind the adjuvant or antigens to the surface of the particle, depending on what type of response you’re looking at,” Baxter says.
VBI picked up the Sci-B-Vac vaccine when it acquired the Israeli company SciVac about a year ago.
VBI employs about 150 people. There are about six people at the Kendall Square, Cambridge, Mass. headquarters, about 60 or so in Israel at the company’s manufacturing facility, and about 25 in Ottawa, Canada, which is primarily the company’s research-and-development center. “The fact we have the bulk of our operations outside of Kendall Square makes us an extremely capital-efficient operation. The unit operating costs in Canada and Israel are probably half of what they would be in Kendall Square,” Baxter says.
The company’s two other lead products are intriguing. Cytomegalovirus infection is the leading cause of prenatal developmental delays, but in the U.S. is largely overlooked in early screening. The virus is common and in most children, adolescents and adults, is harmless. But in people who are immunocompromised or immunosuppressed, it can be deadly. It attacks the central nervous system and the brain. And one of the targets is the developing fetus in the first trimester, when the fetus gets its immunity from the mother. If the mother is not already resistant to the virus and is exposed to CMV, the fetus can develop very severe complications including visual, hearing and brain development defects.
The only country in the world that screens pre-pregnant women for CMV is Israel. In the U.S., it’s not common, although Brigham and Women’s Hospital in Boston screens women.
“Congenital CMV is the biggest cause of birth defects in the seven major markets. Fortunately, in those markets, through vaccination, we’ve eliminated most other significant risk to the developing fetus. And, in fact, there are only two outstanding vaccine targets in this space (CMV and rotavirus), and consequently, because of the significance of the unmet medical need, they are both the highest priority vaccine targets for the organizations that maintain the vaccines schedules in the U.S,” Baxter says.
The interim data on that trial published about a month ago demonstrated 100 percent seroprotection in the highest-dose cohort. Final results from the study are expected in the first half of 2018. “There’s a little bit of excitement about this data. We’re the first people to actually publish seroprotection and seroconversion in epithelial cells. So, there’s a lot of anticipation around this vaccine,” Baxter says.
According to the U.S. Centers for Disease Control and Prevention (CDC), about one in every 200 babies are born with congenital CMV infection. Most won’t have symptoms, but about one in five with the infection will have hearing loss, vision loss, intellectual disability, small head size, lack of coordination, weakness, and seizures.
The company’s other lead program is for glioblastoma multiforme (GBM). GMB is a very common and malignant brain tumor that is difficult to treat. Treatment usually involves surgery, radiation and chemotherapy. The median survival rate is about 14.6 months with less than 10 percent of patients surviving five years or longer. CMV is often found in glioblastoma cancer cells, but not generally in normal brain tissue.
So VBI added an antigen for GBM into its CMV vaccine. In a very early study several years ago, four out of six patients survived, which Baxter notes, “is quite remarkable.”
So VBI developed a vaccine and through the FDA’s Fast Track status, it had an IND approved recently for a Phase I/IIa study in recurrent brain cancer. “That study will begin enrollment for the first Phase I 18-subject cohort in the U.S., in the fourth quarter of this year. We’ve designed an extensive panel of biomarkers and clinical tests that will be conducted every six weeks and with brain scans to understand what happens using the vaccine post-surgical care and chemo and radiation therapy. We will know by this time next year, assuming the study does start with patients in the fourth quarter, and we’ll know if this vaccine will extend overall survival,” Baxter says.
Which is all pretty exciting. The company also has early studies going in medulloblastoma, Zika virus, and respiratory syncytial virus, as well as some other indications it hasn’t announced yet.
“Why isn’t there more private investment in the infectious disease market? There’s really only one company working on rotavirus, and we’re about the only one working on congenital CMV. Vaccines tend to be capital intensive in their early development programs, but it’s often possible to get to proof of concept quicker. Of course, with early development of vaccines, the safety hurdle is very, very high and the low-hanging fruit has already been picked and it takes very brave investors to invest in them,” Baxter says.
On the other hand, after the initial launch, vaccines don’t typically face generic competition, or, in many cases, much competition at all. Still, in an era that talks a lot about preventive medicine, you’d think there would be more interest in one of the most effective ways to prevent diseases—vaccines.
Mark Terry, a regular writer for BioSpace, is a full-time freelance writer, editor, novelist and ghostwriter specializing in biopharma, clinical diagnostics, medical practice management and resume writing. He has written 1000+ articles and more than 20 books, including the award-winning THE FALLEN. When not writing, he can be found practicing Sanchin-Ryu karate, riding his bicycle or reading.
