Biotech Company Focuses on Developing Needle-Free Vaccines

Bioscience Technology (BST) caught up with Dr. Kees Leenhouts (KL), the chief scientific officer of Mucosis B.V. His company, stationed in The Netherlands, is working on a needle-free vaccine candidate for treating human respiratory syncytial virus (RSV).

Dr. Leenhouts discussed how Mucosis’s vaccine is produced and operates, along with where he sees the market for needle-free injections heading.

BST: Talk about your background. Where are you from? Where did you go to school? What did you study?

KL: “I conducted my graduate studies at the University of Groningen in the Netherlands, ultimately receiving a Ph.D. in biology. Following my doctorate program, I spent approximately two years as a research fellow at the International Centre for Genetic Engineering and Biotechnology in Trieste, Italy. Prior to that, I was program manager at Biomade Technology Foundation where I managed the intranasal vaccine technology development team.”

BST: Describe in your own words what Mucosis does and what your role is at the company.

KL: “Mucosis is a clinical stage biotechnology company that focuses on developing vaccines for infectious diseases that can be delivered needle-free through the nose or mouth.
In addition to common infectious diseases such as respiratory syncytial virus (RSV) and pneumococcal related illness, Mucosis is researching oral vaccines for the prevention of rare conditions such as diarrhea caused by Shigella and ETEC (enterotoxic Escherichia coli), with the support in the past from the Gates Foundation.

In my role as chief scientific officer, I am primarily responsible for leading the company’s research and development activities and managing the advancement of our pipeline vaccine candidates.”

BST: Can you elaborate on the differences between needle-free vaccines and the regular variety?

KL: “In addition to having the benefit of a painless delivery system, our needle-free vaccine technology efficiently evokes immune responses that engage disease-causing pathogens at their port of entry, the mucosal layers of the body. Uniquely, our technology is built on particles that not only elicit a strong and robust systemic immune response, but also a strong local immune response in mucosal layers in the nose, airways, gut and other places in the body.”

BST: Please provide some background on the company’s lead product SynGEM. What does it treat? How is administered? What does it cost? Which markets does it have regulatory approval?

KL: “SynGEM, is a stabilized, recombinant, vaccine against RSV, administered intranasally. SynGEM is based on the company’s core Mimopath technology that uses a bacterium-like particle (BLP) derived from the food-grade bacterium Lactococcus lactis as an immunostimulant and/or as an antigen carrier.

There is increasing evidence that mucosal immunity is key for protection against RSV infection, which is a global health challenge. Recent data have demonstrated that SynGEM effectively elicits robust and durable mucosal immunity in addition to providing systemic immunity. Our unique and stable prefusion F form of the antigen can raise more potent neutralizing antibodies against RSV compared with a postfusion F antigen. These data have shown the vaccine's immunogenicity, efficacy and safety in animal models, and support the prophylactic potential of SynGEM across target age groups, further validating the potential clinical value of our unique intranasal approach and the utility of the proprietary prefusion-like F form of the antigen we have developed.

SynGEM is still in the preclinical development stage and we have not yet determined pricing. “

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BST: The company website says SynGEM is intended for RSV. Why was that the company’s first target for this vaccine candidate?

KL: “The company’s first vaccine candidate was against influenza. A human trial in 2011 was conducted which provided proof of concept for the platform by way of clean safety results along with potent immune responses. With this in mind, the company decided to pursue a much needed vaccine target in RSV. The annual global burden of RSV illness is significant, with 33.8 million estimated new episodes of RSV-associated acute lower respiratory infection (ALRI) worldwide in children under age 5, and over 3.4 million hospital admissions associated with severe RSV disease. Global mortality was estimated at 253,500 deaths in 2010. There is currently no available treatment for RSV.

Since RSV enters the body through the respiratory tract, our technology platform was a good fit for a first line of defense against the virus. The mucosal surfaces of the respiratory, and digestive tracts and reproductive organs represent an enormous surface area (> 400 m2 in an adult) that are quite vulnerable to infections by pathogenic bacteria and viruses. The mucosal membranes, however, have the potential to secrete antibodies (IgA) to block or inactivate pathogens.”

BST: How do these vaccines get made? How does your proprietary technology, Mimopath, create these vaccine candidates?

KL: “Our Mimopath technology is built using bacterium-like particles (BLP) that are derived from food-grade inactivated bacteria. The BLP has immune stimulating properties that are used, either by mixing or binding with a vaccine, to target the disease-causing pathogens in the nasal pathway.

Mimopath technology is a plug- and play vaccine platform that addresses the innate immune system through Toll-like receptor 2 (TLR2) to activate the adaptive immune system. This mechanism efficiently evokes immune responses that engage disease-causing pathogens at their port of entry, the mucosal layers of the body. Uniquely, our technology is built on particles that not only elicit a strong and robust systemic immune response, but also a strong local immune response in mucosal layers in the nose, airways, gut and other places in the body. “

BST: What other areas do you see as potential opportunities for making needle-free vaccines of this variety?

KL: “Needle-free vaccines have significant potential to be used for many types of diseases. While our current focus is primarily on RSV, we are continually exploring unmet needs in across multiple disease categories where our core technology may have applications. We’ve even recently investigated potential applications in allergies and oncology.”

BST: What else is Mucosis working on this year?

KL: “Mucosis has several other pipeline products. Our influenza vaccine, FluGEM, is in phase 1 clinical trials. We are hoping to continue those studies with partners and are working to advance our SynGEM vaccine from the preclinical stage into early clinical studies.”