- In the sprint to develop vaccines for the coronavirus outbreak, the world’s largest drugmakers have been lapped by a startup biotech.
- It took 42 days for Moderna to turn the genomic sequence of the virus into a vaccine candidate and ship it to US health officials.
- The National Institutes of Health will now start the first human trial in a matter of weeks, focused on safety. It will require months of additional study to know if the vaccine is safe and effective in protecting people against this novel coronavirus.
- Moderna showed how its novel genetic technology can speed up vaccine development. The Cambridge, Massachusetts-based biotech is also testing vaccines for infectious diseases like the Zika virus, respiratory syncytial virus, and cytomegalovirus, or CMV.
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Scrambling to respond to the growing coronavirus outbreak, some of the world’s largest drugmakers have begun researching vaccines to prevent the deadly virus.
But a small, startup biotech has sped past them, becoming the first company to produce a potential vaccine ready to be tested in humans.
That biotech is Moderna, which earlier this week shipped the first batch of its coronavirus vaccine to begin clinical trials in people. It’s still a long ways away from determining if it is safe and effective at stopping the virus.
The first study will focus on safety, with the National Institutes of Health (NIH) planning to administer it to 45 healthy adults in the US. Future tests would focus on whether the vaccine works, and would involve hundreds to thousands of people.
Even with a long road ahead, Moderna has beaten history in the race to construct a potential vaccine. Moderna needed 42 days to translate the DNA of the virus into a vaccine and ship it. For comparison, it took about 20 months to start testing a vaccine candidate for the 2002-2003 SARS outbreak after that virus was sequenced.
Moderna designed and manufactured the vaccine even more quickly: in 25 days. The company had the vaccine ready on February 7, according to a regulatory filing. The next couple weeks were spent on analytical testing before sending the first batch to the NIH.
The urgency has been driven by the rapid spread of the virus, which causes the disease known as COVID-19. The death toll has surpassed 2,800 people with more than 82,000 infected. While the vast majority of cases have been in China, the virus has spread to at least 47 countries on six continents.
The Cambridge, Massachusetts-based company is dwarfed by the size of drugmaking giants also hunting for vaccines. At $9.5 billion, Moderna is less than 3% the size of Johnson & Johnson by market value. The French pharma giant Sanofi has more than 100 times as many employees — 100,000 compared to fewer than 1,000 at Moderna.
The Boston biotech venture capital group Flagship Pioneering founded Moderna in 2010. It’s led by Stephane Bancel, previously the CEO of a French diagnostics company.
Moderna’s unique advantage is its novel technological platform, which also drove its record-breaking IPO for the biotech industry in 2018, in which it raised $604 million. Through this point, the company’s value has been driven by the potential of its genetic platform, as the company has no approved drugs or vaccines on the market.
That speedy progress to get to the brink of starting clinical trials is “unquestionably the world indoor record,” Anthony Fauci, the longtime leader of the NIH’s infectious disease division, recently told The Wall Street Journal. “Nothing has ever gone that fast,” he added.
How Moderna went from DNA to vaccine in 42 days
Moderna’s speed is built on the decades of progress in vaccine development and genetics.
At the basic level, vaccines work by giving cells a taste of a virus — just enough for our bodies to recognize it as an intruder and pump out antibodies to fight a similar invasion in the future.
For decades, vaccines have traditionally contained a dead or weakened version of the virus itself. Early advances in genetics allowed a vaccine to use just proteins made by the virus. That was first used in the 1980s to develop a vaccine for hepatitis B.
Moderna has gone a step further back: instead of using viruses or proteins, the platforms works with just the genetic material. The biotech is focused on messenger RNA (mRNA) — the genetic material from DNA that makes proteins.
In the case of the coronavirus, Moderna’s strategy was to load its vaccine with mRNA designed to produce the coronavirus protein. The body’s immune cells can recognize that protein and create antibodies against it, prepping to fight a future infection.
Moderna’s speed wasn’t a solo effort. Scientists from Fudan University in Shanghai posted the genomic sequence of the coronavirus on January 10. NIH investigators then worked with Moderna’s scientists to study that data and find potential targets.
Just three days later, NIH investigators and Moderna’s infectious disease team finalized the sequence for the vaccine, moving into production mode. The Coalition for Epidemic Preparedness Innovations also supported Moderna’s work, announcing a funding agreement later in January. By February 7, the company had completed a first batch of the vaccine.
If Moderna was using traditional vaccine technologies, Moderna would “still be working at it as we speak, and we most probably would not even have started to make the product,” CEO Bancel said a couple days after shipping off the vaccine to the NIH.
Instead, Moderna had to do “a tiny bit of optimization” to an mRNA molecule “and then go right into production,” he said — the promise of its platform, realized.
The challenges ahead include a lengthy clinical timeline, uncertain production capability
Even with the speed in getting to this point, determining the safety and efficacy of Moderna’s drug will likely take more than a year, at a minimum.
Vaccine research experts have told Business Insider that it typically requires several years of testing and hundreds of millions in funding to bring a vaccine for an infectious disease to approval. In past cases, like SARS, when the virus began to go away, so did the money for research. There’s still no approved vaccine to prevent SARS.
The NIH’s Fauci laid out the timeframe for vaccine development in a February 26 press conference at the White House.
This first study is based out of Seattle, Washington, at the Kaiser Permanente Washington Health Research Institute. The trial is looking to enroll 45 healthy volunteers who are men or non-pregnant women ranging from ages 18 to 55.
They will be split into three groups of 15, with each group receiving a different dose strength — two injections about a month apart. The strongest dose is 10 times as potent as the low dose. Currently, the trial is expected to begin in March, according to a clinicaltrials.gov listing.
While the study will follow participants for a year, early safety results should be available about three months after the trial starts, according to Fauci.
If those show the vaccine is safe, it can advance to mid-stage testing that enrolls hundreds or potentially thousands of people to begin testing its effectiveness at stopping the virus. Those trials would take at least six to eight months to run, Fauci said.
While the NIH takes the lead on development, Moderna is still in charge of producing the vaccine. If the demand persists and the mRNA-1273 shows promise, that will be a formidable challenge for a biotech with no history and limited capability to mass-produce a product.
“We’re looking at everything that it would take and how to actually get it done,” Moderna’s Chief Medical Officer Tal Zaks said about scaling up manufacturing on a February 26 earnings call. This could include building out the company’s own manufacturing capiability or partnering with contract manufacturers.
The Norwood, Massachusetts plant that produced mRNA-1273 is focused on developing new treatments, not pumping out lots and lots of a vaccine. That means it prioritizes flexibility, not scalability, Bancel said.
Moderna’s bold vision for the future of vaccines
Wall Street analysts have expressed skepticism over whether the coronavirus vaccine would drive commercial value for Moderna, but also acknowledge the speediness demosntrates the power of its technology.
“The rapid turnaround of a COVID-19 vaccine candidate is impressive and illustrates the potential power of this platform,” JPMorgan analyst Cory Kasimov said in a Wednesday note.
“However, the ultimate commercial opportunity is TBD but unlikely to be substantial, in our view,” he wrote.
But Moderna’s value stems from the ability to potentially crank out vaccines for a wide range of diseases in rapid time with essentially a few tweaks to the genetic code.
Investors have also noticed — the biotech’s stock has jumped up 33% in the first couple months of 2020. Shares kept climbing even when the company sold $550 million of additional stock in early February.
Beyond coronavirus, Moderna is testing 12 mRNA programs in clinical trials, a majority of which are vaccines. The compaany is going after some major diseases with no approved vaccines, like respiratory syncytial virus, Zika, and cytomegalovirus (CMV).
If successful, mRNA could become a new template for speedy vaccine development.
“I have never been more optimistic about Moderna’s future and potential since joining as employee number two in 2011,” Bancel said on a February 26 earnings call. “I believe mRNA is going to be a new class of medicines, and I believe Moderna is the leading company in that field.”
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