Indroduction
Welcome to The Niche Podcast – Your weekly rundown of the biotech, clinical research, and applied science industries. I’m your host, Dr. Noah Goodson. This week, Pfizer submits to the FDA, how mRNA vaccines work, and Novartis drops a billion, but really only $50 million.
Pfizer/BioNTech Submit for EAU
Pfizer and BioNTech have submitted their COVID19 vaccine to the FDA for emergency use authorization (EUA). This is the first preventative treatment for COVID19 to be submitted to the FDA and represents a milestone in scientific advancement. EUA is not a full approval but allows the drug or in this case vaccine to be used in a subset of the population that may be specifically vulnerable. Based on the available safety data it is likely the FDA will approve this EUA and full authorization can be expected as early as the end of the year. Moderna will likely follow Pfizer shortly.
How mRNA Vaccines Work
We have gone from a novel disease- causing virus to a vaccine in just 8 months, something previously unimaginable. How is it possible? It’s all thanks to the underlying technology driving both Pfizer/BioNTech’s and Moderna’s platform. I wanted to take a minute and re-educate our listeners on why these vaccines could be developed so quickly and why they are so surprisingly safe and effective. While we won’t go into all the details, I want you to understand how these vaccines work in concept. I’ve done my best to use simple terms and I am glossing over the details to give you a big picture understanding. So, if you’re an immunologist, please don’t @ me.
In general terms, most vaccines work because our immune system has a mechanism for memory. When infected by a virus, the body has the ability to generate specific antibodies to the molecular signals from that specific virus. Think of this like finger-print recognition of intruders. Once an intruder has been identified and their fingerprints logged, the body will then be able to stop infections in future if they have the same fingerprints. Many vaccines in recent history work by taking dead version of the virus and injecting that into your body. Think, fingerprints attached to the intruder, but the intruder can do no harm. The body analyzes the fingerprints and catalogues them in a cellular database. Then if a “live” intruder shows up, the body recognizes the fingerprints and defeats them.
The new mRNA vaccines by Pfizer and Moderna work differently. They utilize mRNA. Messenger RNA is an intermediary in the production of proteins. Recall that basic sequence everyone learned in high school biology, DNA to RNA to Protein. The problem with mRNA in general is that it is very unstable. It is rapidly broken down in cells. If you’ve ever worked with RNA in a lab you know how easily you can contaminate and destroy your samples. Biology does this on purpose. If mRNA is in a cell, in general the cells machinery will make whatever protein that mRNA gives the blueprint for until the mRNA breaks down.
mRNA vaccines coopt this machinery in a very clever way. Here is how it works. Scientists take a virus like COVID19, and instead of using a dead version of it, they just identify the fingerprints. Once they know what the fingerprints look like, they reverse engineer these into mRNA. They then take this mRNA and package it inside something called a liposome. A small nano-sized carrier vessel that cells use to transport materials. The liposomes both protect the mRNA and ensure it enters body’s cells. In the vaccine liposomes filled with blue-prints for viral fingerprints are injected into your body. Cells in your body absorb the liposomes and for a short time turn the mRNA into proteins. These proteins (viral fingerprints) are then dumped out of the cell and the immune system catalogs the fingerprints. Then if a real COVID19 virus ever shows up, they are already recognized, without ever having been in your body.
The mRNA will be naturally degraded in your cells and the proteins will be broken down over time, leaving your body free of the signs of the vaccine. However, your immune cells will have catalogued a memory of the viral fingerprint. How safe and effective is this? It turns out to be incredibly safe, and wildly effective based on the data we have seen. I know for many people around the world, a rapidly delivered novel vaccine technology can seem scary, but it’s actually doing something similar to what is done with many vaccines: It’s teaching your body to recognize the fingerprints of an intruder so it can prevent you getting sick.
Special Notes
Hi folks, Dr. Noah Goodson here. I want to let you know about a special bonus episode coming in a couple of weeks. I’m sitting down with a young COVID survivor who also works in healthcare. Hearing from those who have survived this disease, the dramatic personal consequences, even for the young and healthy, and getting an inside look at the experience of just one of the millions of front-line workers. I think you’re really going to enjoy this, so keep your eyes on your podcast feed for this upcoming Niche Podcast Bonus.
Novartis Drops A Billion... or Less
Novartis signed a $1.35 billion deal with Mesoblast for Remestemcel-L, a treatment for acute respiratory distress syndrome (ARDS). Remestemecel is slotted to treat ARDS in a variety of conditions that include COVID19. The drug is current in a Phase III clinical trial.
While the Novartis deal appears a huge one on the surface, it is remarkably short on cash up front. Mesoblast gets $25 million for the license and another $25 million for development. The remaining $1.30 billion is tied up in milestone achievements at a 40/60 pre/post market split. While the agreement has a sizeable final payout potential, the initial payout of just 3.7% of the deal suggests to me that Novartis has limited faith in the success of the product, or suspects that vaccinations may decrease short-term ARDS cases due to COVID and make it more reasonable for them to hold on for long-term incremental gains. Ongoing phase III studies for COVID and non-COVID related ARDS will be completed in the first half of 2021.
Sponsors
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Closing Credits
Thanks for joining me on The Niche Podcast; your weekly summary of top news in the biotech, clinical trials, and applied science industries. You can find us on your favorite podcast app. Like, comment, subscribe, and most of all share with your friends. If you like what you hear, please rate and review, it really helps us. Once again, I’m Dr. Noah Goodson, I’ll see you next week.