Moderna Gets 3% Stock Boost From Early Clinical Data

Stephane Bancel, chief executive officer of Moderna Therapeutics Inc.
© 2017 Bloomberg Finance LP

Moderna (MRNA) stock rose on Wednesday after the company released news of positive clinical data to coincide with its R&D day in New York. The company, which focuses on therapeutics and vaccines that involve messenger RNA (mRNA) shared interim data from two phase one clinical trials: a vaccine to prevent cytomegalovirus (CMV), a common cause of birth defects, and monoclonal antibody therapy to prevent chikungunya virus. The stock peaked midday increasing 9% to $18.38, and then closed at a 3% increase to end at $16.40. 

Moderna had a rocky few months after its relatively large IPO last December, in which it raised over $600 million, a biotech record. At the time, it had a valuation of $7.5 billion. Yet after the 6-month lock up period, some investors took advantage of their ability to sell the stock, and Moderna’s market capitalization is now at $5.4 billion. 

But this recent data is early proof that Moderna’s mRNA technology may work. "It's quite frankly reassuring to see that kind of outcome," says Alan Carr, an analyst at Needham. The sets of data released today, “were two important milestones for the company this year,” he says. Both showed that the therapeutics were generally well tolerated and stimulated an immune response.  

Messenger RNA may sound familiar to those who learned about “transcription and translation” in biology class. In the body, mRNA allows code from DNA to be transcribed into proteins. Moderna’s therapeutics inject mRNA into the body, where it  instructs cells to create proteins that help fight against disease. In the case of the CMV vaccine, the mRNA helps create a segment of the virus that can activate the immune system. For the chikungunya therapeutic, mRNA tells cells to create antibodies that protect against the disease.

"In one case you're mimicking the virus,” says Tal Zaks, Moderna’s Chief Medical Officer, “ in the other case you're mimicking the immune system's response to the virus." 

There is no vaccine for CMV on the market, despite the fact that it is the most common virus passed from pregnant women to their babies. With the release of this data, Carr says, “"we're getting a clearer picture of a vaccine that has the potential to generate a lot of revenue for Moderna."

Zaks says preparations for a phase 2 study that will focus on the efficacy of the CMV vaccine are “well underway” and the trial will likely start soon.

The chikungunya treatment, which consists of an intravenous infusion of mRNA, is a more fundamental proof-of-concept study for the company, Zaks says. “For the first time in history,” he says, “we've actually taught a human body to make its own medicine." 

Zaks and Moderna CEO Stéphane Bancel say that they plan to expand the use of their mRNA technology to treat everything from rare genetic diseases to cancer. The idea is that once there is a reliable way to deliver mRNA into the body, it can encode any number of proteins. “It's like ‘copy and paste,’" Bancel says. 

 In the case of rare genetic diseases, Zaks says, mRNA could create proteins missing in children’s bodies and allow them to produce the proteins internally. They are hoping that this is a possible treatment for methylmalonic acidemia (MMA), one of the rare diseases they are currently studying.

Academics and researchers are excited about the promise of mRNA vaccines and therapeutics, since they have the potential to be less expensive and more rapidly produced than traditional vaccines. Moderna certainly isn’t the only company in this cutting-edge field, but they are one of the largest and most well funded. German company BioNTech, which works on mRNA therapeutics for cancer and other diseases, filed for a lower IPO this week than originally anticipated. Translate Bio, another competitor, recently announced it was dropping one of its big drug-development programs, causing its stock to fall. 

But Moderna is different, Zaks says. “There is no company out there right now that has our breadth of application nor frankly our depth of clinical data."

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Moderna (MRNA) stock rose on Wednesday after the company released news of positive clinical data to coincide with its R&D day in New York. The company, which focuses on therapeutics and vaccines that involve messenger RNA (mRNA) shared interim data from two phase one clinical trials: a vaccine to prevent cytomegalovirus (CMV), a common cause of birth defects, and monoclonal antibody therapy to prevent chikungunya virus. The stock peaked midday increasing 9% to $18.38, and then closed at a 3% increase to end at $16.40. 

Moderna had a rocky few months after its relatively large IPO last December, in which it raised over $600 million, a biotech record. At the time, it had a valuation of $7.5 billion. Yet after the 6-month lock up period, some investors took advantage of their ability to sell the stock, and Moderna’s market capitalization is now at $5.4 billion. 

But this recent data is early proof that Moderna’s mRNA technology may work. "It's quite frankly reassuring to see that kind of outcome," says Alan Carr, an analyst at Needham. The sets of data released today, “were two important milestones for the company this year,” he says. Both showed that the therapeutics were generally well tolerated and stimulated an immune response.  

Messenger RNA may sound familiar to those who learned about “transcription and translation” in biology class. In the body, mRNA allows code from DNA to be transcribed into proteins. Moderna’s therapeutics inject mRNA into the body, where it  instructs cells to create proteins that help fight against disease. In the case of the CMV vaccine, the mRNA helps create a segment of the virus that can activate the immune system. For the chikungunya therapeutic, mRNA tells cells to create antibodies that protect against the disease.

"In one case you're mimicking the virus,” says Tal Zaks, Moderna’s Chief Medical Officer, “ in the other case you're mimicking the immune system's response to the virus." 

There is no vaccine for CMV on the market, despite the fact that it is the most common virus passed from pregnant women to their babies. With the release of this data, Carr says, “"we're getting a clearer picture of a vaccine that has the potential to generate a lot of revenue for Moderna."

Zaks says preparations for a phase 2 study that will focus on the efficacy of the CMV vaccine are “well underway” and the trial will likely start soon.

The chikungunya treatment, which consists of an intravenous infusion of mRNA, is a more fundamental proof-of-concept study for the company, Zaks says. “For the first time in history,” he says, “we've actually taught a human body to make its own medicine." 

Zaks and Moderna CEO Stéphane Bancel say that they plan to expand the use of their mRNA technology to treat everything from rare genetic diseases to cancer. The idea is that once there is a reliable way to deliver mRNA into the body, it can encode any number of proteins. “It's like ‘copy and paste,’" Bancel says. 

 In the case of rare genetic diseases, Zaks says, mRNA could create proteins missing in children’s bodies and allow them to produce the proteins internally. They are hoping that this is a possible treatment for methylmalonic acidemia (MMA), one of the rare diseases they are currently studying.

Academics and researchers are excited about the promise of mRNA vaccines and therapeutics, since they have the potential to be less expensive and more rapidly produced than traditional vaccines. Moderna certainly isn’t the only company in this cutting-edge field, but they are one of the largest and most well funded. German company BioNTech, which works on mRNA therapeutics for cancer and other diseases, filed for a lower IPO this week than originally anticipated. Translate Bio, another competitor, recently announced it was dropping one of its big drug-development programs, causing its stock to fall. 

But Moderna is different, Zaks says. “There is no company out there right now that has our breadth of application nor frankly our depth of clinical data."

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I'm the assistant editor for healthcare and science at Forbes, focused mainly on biotech and the business of healthcare. I have a Master's in Journalism and a Master's

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