With seven months of data in hand, Boston-area biotech Moderna is more confident than ever that it has an effective vaccine for one of the world's more common viruses.
On the heels of an initial, positive look at the data in September, Moderna announced Thursday afternoon more results from a small study showing patients given its vaccine expressed high enough antibody levels to protect against CMV, short for cytomegalovirus.
Transmitted from infected mothers to babies, the virus can sometimes impair growth of vital organs such as the brain and liver. It's also quite prevalent, infecting about one in three children by age five and more than half of adults by age 40, according to the Centers for Disease Control and Prevention. Regulatory agencies have yet to approve a vaccine for the virus.
"We have a chance, with a vaccine with this level of performance, to have a big public health impact and to eradicate this virus," Moderna CEO Stéphane Bancel said in an interview with BioPharma Dive.
Bancel's company, founded in 2010, wanted to show its vaccine could boost antibody levels higher than where they typically are for people infected with the virus. The latest data support this aim, as antibody concentrations in already-infected patients were 22- to 40-times greater one month after they received a third dose of the vaccine.
Patients not previously infected, meanwhile, had antibody levels that were 10 times above what infected patients showed at baseline.
Notably, these results pertain to the two middle doses of Moderna's vaccine, set at 90 and 180 micrograms. The company is testing 30 and 300 microgram doses as well, but didn't disclose much data on either in Thursday's release. That highest dose has three months of available data versus seven months for the others, yet Moderna said its safety has been similar to the 180 microgram dose and its effect on antibodies has followed a dose-dependent pattern.
Across the trial, Moderna reports there have been no vaccine-related serious adverse reactions. The most common reactions thus far are headache, fever, chills, and muscle and injection site pain.
As data from this early-stage study mature, Moderna is already planning next steps. The company also said Thursday a Phase 2 study has dosed its first patient. The study is designed to test three doses — 50, 100 and 150 micrograms — in about 250 healthy volunteers, with initial data expected in the second half of the year.
The best-performing dose would then advance into a Phase 3 trial, something Moderna is already talking about with regulators. This trial would begin in 2021, by the company's estimates, and enroll no more than 8,000 patients at sites across Europe and North America.
A successful Phase 3 trial could clear the path to market, though that in turn presents certain obstacles. Vaccines require large-scale manufacturing, especially when they target common viruses like CMV. They're also generally sold at a lower price than drugs in areas like cancer or immunology, and can yield lower margins. Because of these challenges, most of the vaccine market is cornered by four drug giants: Pfizer, Sanofi, GlaxoSmithKline, and Merck & Co.
Bancel expects Moderna to independently commercialize its CMV vaccine, known as mRNA-1647, though he said the company is open to partnerships for other vaccines — and in particular geographic partnerships that help develop a global reach more quickly.
In the meantime, Bancel said manufacturing shouldn't be as much of a burden as it is for other vaccines because of the small amount of mRNA needed for production. Moderna has also started getting ready for a potential rollout by hiring its first marketing associates. Commercialization plans are likely to include physician and patient education as well.
Investors have shown great interest in Moderna's approach. At the end of 2018, the company delivered the biggest initial public offering in biotech history, raising just north of $600 million.
Bancel acknowledges that some investors are less than thrilled that his company's first commercial product might be a vaccine. He points to Merck's Gardasil, GSK's Shingrix and Pfizer's Prevnar franchise, each of which are blockbusters, as examples of what mRNA-1647 could ultimately become.
"People sometimes do not appreciate [the potential] because all of the vaccines are buried into the business of four big, successful pharmaceutical companies, so the biotech world is not as exposed to vaccines," he said. "If you look at the facts and numbers, vaccines are an amazing business."
Unlike those other products, Moderna's vaccine uses a type of biological instruction kit called messenger RNA to create proteins that alert the body's immune system to recognize CMV. Moderna sees mRNA therapies as useful for many other diseases too, evidenced by the company's more than 20 experimental programs that go after heart disease, metabolic disorders and various cancers, among other targets.
When it went public, Moderna sold shares at $23 apiece. Shares have mostly stayed below that value, however, since going public. They rose 3% in mid-September, when the company revealed positive early findings for both mRNA-1647 and a different experimental vaccine. Shares were trading at $18.30 at market's close Thursday, reflecting an 8% increase from the same time a year prior.
Whether a CMV vaccine can raise investor confidence has yet to be seen.
"All the successful biotech companies that have come before us, they all have been built around one key commercial product," Bancel said. "We really believe that CMV will be that for Moderna."