Not so crazy now: Biotech’s most closely watched unicorn just produced its first clear snapshot of human efficacy data

The executive team at Moderna raised a cheer today after publishing their first early snapshot of human efficacy data that demonstrate their messenger RNA tech works — at least on the first try.

Stéphane Bancel

The biotech tested their H10N8 flu vaccine on a small group of 31 subjects, looking at their response in two different measures. All demonstrated a sufficient immune response to fight off the virus in the first measure, and all but 3 in the second, for a total of 23 who received the vaccine.  None of the 8 subjects who received a placebo responded.

Typically, evidence of Phase I success in a vaccine study — which followed a number of animal studies — wouldn’t attract much attention for your average biotech. But there’s nothing average about Moderna. CEO Stéphane Bancel and his team have attracted $1.9 billion to create a company that’s building a pipeline around messenger RNA, one of a handful of players looking to coax cells to produce proteins to fight disease. And that’s a massive amount for a company that just showed human proof of concept data.

This vaccine is not pointed toward commercialization, Bancel told me Thursday afternoon. It’s a demonstration project. The goal was to start out with “low biology risk where we knew where the goal post was before we started the trial.”

Moderna’s board has already decided not to seek an IPO this year, and Bancel adds that there’s no definitive plans set for 2018 either. What he would like to do, he says, is add new pieces to the pipeline — new verticals to the three he’s already outlined — adding more data on the potential mRNA has to create a new pathway on drug development for a wide array of diseases. Then, have something substantial that proves what they can do, he’ll set out to convince investors.

The huge amount of cash raised to date and Moderna’s plans to prove the principles behind its science — focusing on vaccines as the most likely initial demonstration program that could prove that mRNA can safely work — has also triggered more than a little schadenfreude for a company that is often billed as mysterious and lavishly funded. STAT picked up on that, reporting a battery of mostly anonymous criticism about its tough work environment and lofty claims, laying into the biotech at every turn.

It is still early days at Moderna, which has to prove that it can develop mRNA drugs for some of the more lucrative therapies than the frontrunning vaccines. That will take some time. But Moderna’s been doing much more to outline what programs it has in the pipeline, and when it can discuss more about the data.

“I’m cautiously optimistic this could be a really important addition to our vaccine arsenal,” Barney Graham, deputy director of the Vaccine Research Center at the National Institute of Allergy and Infectious Diseases, told the Wall Street Journal. He added that RNA is unstable, leaving him eager to see more mature data on how this works.

Bancel and his fellow execs at Moderna may have a long way to go, but they’re confident that they took a big step along the journey today. From now on, it’s all about clinical data at Moderna.

H10 mRNA Immunogenicity in Humans | Molecular Therapy

Said Tal Zaks, Chief Medical Officer of Moderna:

The data “provide important validation of our core mRNA platform, as we continue to advance our development pipeline, tackling more complex vaccines including personalized cancer vaccines, and moving our mRNA therapeutics into clinical development.”

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