stimulate production of dozens of different proteins through this method, out of the roughly 22,000 different protein molecules that make up the tissues of an individual human being, Bancel says. Moderna says it has shown it can make its mRNA molecules to be given as intravenous infusions, intramuscular injections, or in the more convenient shots just under the skin, known as subcutaneous injections. The company says it has demonstrated safety and effectiveness of its drug candidates in mice, rats, and primates.
Moderna remained stealthy in its first couple years, as my colleague Greg Huang wrote in December, because it was afraid a Big Pharma company would come along with a $50 million budget and squash it. It has one competitor of note, Germany-based Ethris, Bancel says. Moderna believes it is on course to start its first clinical trial with this new kind of therapy in 2014, Bancel says.
The company has some big names on its list of co-founders. Bob Langer, the MIT professor; Tim Springer, the professor at Children’s Hospital Boston and founder of LeukoSite; Ken Chien, a cardiologist at Massachusetts General Hospital; Derrick Rossi of the Harvard Stem Cell Institute; and Noubar Afeyan, the managing partner at Flagship Ventures.
Afeyan, Moderna’s chairman, was clearly excited about the AstraZeneca partnership when we spoke yesterday. But he also didn’t want to get carried away.
“I’ve been around 30 years in this industry, long enough to know that deeming something the next big thing is not a very useful activity,” Afeyan says. “We didn’t even talk about company publicly until December because we weren’t sure how broadly applicable it might be. We still don’t know, but we have some hints. We wanted to be sure we understood the critical challenges.”
One of the big challenges is finding ways to make sure the mRNA molecules don’t get degraded by various enzymes or destroyed by the body’s immune defenses, which might see them as foreign invaders. The company hasn’t seen that happen so far, and it has used proprietary chemistry to modify its drugs to avoid sparking that kind of immune reaction, Bancel says. The company has also been keeping an eye out for whether its drugs are toxic to the liver or the heart—where toxic side effects can be a show-stopper.
But if Moderna can clear those hurdles, it could have a huge opportunity with a cheap, efficient R&D platform. Moderna doesn’t need to go through long chemical screening processes to find a drug that hits a molecular target. It doesn’t need to use expensive, and time-consuming protein manufacturing processes in cell culture to make large molecules. Moderna can essentially start with a snippet of genetic code of interest, synthesize an mRNA candidate based on that code within days, do some basic purification, and dose the molecule to animals in days. This process can shave months, if not years, off the discovery processes that must come before a drug even enters animal tests.
“We went from nothing to having two drugs in primates in nine months,” Bancel says. He adds: “On Oct.1, 2011 we had nothing. On June 5, 2012, we had two drugs tested in non-human primates. I don’t think a company has ever done that in biotech.”
Moderna could have chosen to license its technology widely, like other technologies thought to have broad application in the pharmaceutical business—like RNA interference. It chose not to do that, Afeyan says, preferring to become a product developer in partnership with AstraZeneca, and maybe one more partner. By going this route, Moderna will get the money and development expertise of a bigger company and still get the resources it needs to grow as an independent company. Moderna, he says, will not be forced to tap into its new $240 million cash horde to carry out further development of drugs for AstraZeneca—that expense will be shouldered by AstraZeneca, he says.
Financially, Moderna is now sitting in about as good a position as a biotech startup can ever expect to be. It still has $20 million left in the bank from its original venture financing, Bancel says. The company now has $240 million from AstraZeneca which it can use to build up its own pipeline of mRNA drugs for rare diseases, and certain forms of cancer. It plans to hire another 20 to 30 people in short order to pursue some of its own opportunities, beyond just serving its big new collaborator.
“This is a very transformative moment for us,” Bancel says. “It will enable us to accelerate the company, start many new programs in the clinic, to build a very special company.”
