Many of the biotech firms we have focused on so far in this series have, as their primary goal, alleviating the symptoms of, or finding cures for, certain diseases and conditions. This week’s biotech focus, however, takes a different perspective: the idea of disease prevention through specific vaccination. The idea of vaccination is, of course, well-known and not particularly novel—the extent to which Novavax (NVAX) implements it, however, may prove to be.

Most of us can remember the time when our pediatrician first placed a needle in us. First the cotton swab, then the irritable prickling sensation, then the small plastic toy or candy that followed up the doctor’s visit. Those vaccines, however, have saved lives by helping human immune systems acquire a “taste” for diseases since Louis Pasteur developed the technique in the 19thcentury. The purpose was simple: to prevent maladies before they occur. This is what Novavax hopes to do with their products.

Novavax

Novavax Incorporated is a “clinical-stage vaccine company committed to delivering novel products to prevent a broad range of infectious diseases,” according to their self-description.   The first step in studying such a company is to ask the question: what diseases?

Respiratory Syncytial Virus (RSV)

RSV attacks the lower respiratory tract in individuals of all ages, especially at the extreme ranges of the age spectrum. Infants and older adults especially, in light of their undeveloped or aging immune systems. According to the US National Library of Medicine, RSV is the “most common germ that causes lung and airway infections in infants and young children. Most infants have this infection by age 2.” This is an area of medicine that appears gravely neglected.

RSV is easy to catch. If someone in your vicinity with RSV sneezes, you may be contaminated. If you touch your nose, eyes, or mouth after touching something touched by a person with the virus, you may contract the virus.

So what are the remedies to RSV?

  • Antibiotics do not treat RSV.
  • Hospitalization can only provide oxygen, humidified air, and IV.
  • In some cases, a breathing machine may be required.

RSV also is responsible for being the number one cause of pneumonia in children under 1 in the United States. The Novavax website cites a research report that between 150,000 and 200,000 deaths in children under 5 are due to RSV. That includes, again, only children.  As mentioned above, older adults are also liable to acquire the disease.

Novavax is targeting a medicinal market that has been hitherto little touched by medicine, and with a very real potential to improve the lives of those vulnerable to RSV. The vaccine for RSV is currently in Phase 3 clinical trials for older adults and infants, and entering Phase 2 for pediatric use.

Using Insect Cells and Genetic Engineering to Combat RSV

What makes the Novavax vaccine production process unique is its use of genetic engineering, biotechnological advances, and insect cells to create its vaccines. Using the cell system called Sf9 from insect cells, the company is able to create nanoparticles (remember that nano signifies something extremely small) that act as the foundation of the vaccines in development. The company utilizes two types of nanoparticles.

First are recombinant protein nanoparticles, which are nanoparticles explicitly designed to combat a single protein type and deliver the necessary antigen(something that activates the body’s immune response). The goal is to have the body itself be prepared to have an immune system response to RSV, which can sometimes fail to trigger a full response from the body.

The second nanoparticle type is a VLP, or virus-like particle. While this might sound unusual at first glance (why would you use a virus to combat another virus?), the explanation is not as complex as you might expect. Using a combination of genetic engineering and the insect cells, the scientists at Novavax create particles that look, to the body’s immune system, like viruses. Remember that a virus is basically a shell with some type of DNA or RNA inside—the dangerous part of the virus is not the shell, which does nothing on its own, but the DNA or RNA that it injects into the body’s cells. Since the virus-like particles that Novavax produces have no DNA or RNA, there is nothing they can do to infect the body; however, because they appear to the body as viruses, when the body has an actual influenza virus or RSV, these “fake viruses” can produce a stronger immune response to the actual threat. In particular, the way these virus-like particles are created gives them a stronger capacity to induce response in the two most vulnerable parts of the market we discussed above: young children and older adults.

Other opportunities

Novavax is not just focusing on one market, however. They also have, in development, a proposed vaccination for influenza (the flu), a combination vaccine for the flu and RSV, and two “emerging threats” as they have listed on their sites, which are Ebola and Middle East Respiratory Syndrome (MERS). Novavax’s diverse pipeline is spread across all phases of clinical trial.

In summary, Novavax’s use of insect cells and genetic engineering to produce different types of nanoparticles to act as vaccines against traditionally difficult-to-treat markets shows promise, and its pipeline suggests that it has more than one product at its disposal, which is an important component of probability of success.

That’s it for this week’s biotech digest analysis. Remember that, apart from probability of success and marketplace analysis, a key component of any biotech study is an examination of the chance that a company such as Novavax has enough funds to survive through FDA testing without having to significantly dilute its stock (meaning create more stock to raise money, which makes current stock less valuable). For examples of this type of additional analysis, which we at BTA do for our Monthly Biotech Focus subscription newsletter, you can visit our sample page by visiting http://belltowerassoc.com/biotech-monthly/.

 

 

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