MODERNA:
HARNESSING A BOLD, NEW MODALITY

Moderna, a disruptive biotech startup was determined to move beyond traditional drug development practices and do something radically new: help the human body manufacture what it needs to fight a disease in a novel way. The company, based in Cambridge, MA, aimed to harness the power of messenger RNA (mRNA) to "re-program" a body's cells to make the proteins or antibodies it needs to repair what's wrong.

CREATING NEW THERAPIES

Although many had tried to create mRNA-based therapies before, Moderna believed it had the right combination of talent, technology and resources to turn its promise into reality. Key to this was a new way of formulating mRNA that did not trigger the body's response to recognize and subsequently degrade it, that was a common problem in the past.

"Still, we knew the road would be bumpy," says Moderna CEO Stéphane Bancel. "Nobody has done mRNA medicine before," he says. "When we first set out, we didn't know what should work or what shouldn't work. You need a special mindset. You have to be both humble enough to question everything and at the same time very ambitious—like wanting your technology to become a cure for a lot of patients around the world."

This is about learning faster than anyone else in the world how to make mRNA medicines, because patients are waiting for new and effective treatments.

- Stéphane Bancel, CEO, Moderna

Merck's Business Development & Licensing Boston Innovation Hub is focused on engaging with academia, biotech and venture capitalists in this global epicenter of scientific advancement and biomedical entrepreneurism. The team has full search, evaluation and transaction capabilities (up to POC) to expedite deal execution. As a world leader in vaccine discovery and development, Merck took notice of the strong platform Moderna had built around mRNA for developing vaccines. As discussions began, they found that they shared an important philosophy: "You have two companies who both believe that you cannot make great medicines without doing great science," says Bancel. "Merck is very committed to doing the science right. I think that's always been part of Merck's reputation, but it was good for us to witness firsthand," Bancel says. "We saw that Merck is fact-driven, science-based and calm. When things don't go as expected, the attitude is 'let's figure out what happened, let's make a plan in a very thoughtful way.'"

AN EVOLVING PARTNERSHIP BUILT ON TRUST

In January 2015, Moderna and Merck entered into a license and collaboration agreement to develop mRNA-based vaccines and therapeutics to treat infectious diseases. Merck's investment would help Moderna design and create multiple mRNA drug candidates (with an additional candidate added after the first year). In turn, Merck would lead the discovery and development of candidates and commercialization of any products resulting from the agreement.

What we have in our partner is the combination of an incredible R&D engine and the capabilities of a group of folks who are passionate about this very disruptive technology.

- Stephen Hoge, President, Moderna

The partnerships' impact was immediate, as it enabled Moderna to create multiple mRNA vaccine candidates for preclinical testing on a time scale they could not have achieved on their own. "What we have in our partner is the combination of an incredible R&D engine and the capabilities of a group of folks who are passionate about this very disruptive technology," says Moderna President, Stephen Hoge, MD. And although speed is important—"patients are waiting for new and effective treatments," notes Bancel—Merck also demonstrated that it shared Moderna's long-term view of scientific discovery and a tolerance for the uncertainty and setbacks that are an inevitable part of doing something completely new.

Moderna believes it has found the right partner in Merck, and Merck in turn has demonstrated a deepening trust in Moderna. "The first deal for infectious disease was supposed to be only for discovery with Merck handling development. Ten months into the collaboration, when the first vaccine candidate was nominated to move into development, Merck said, 'would you mind doing the clinical trials?' and I remember I was pinching myself!" Bancel marvels.

COMBINING THE POWER OF MODERNA'S mRNA THERAPY WITH MERCK'S ANTI-PD1 THERAPY

The close ongoing collaboration between the two companies led to another exciting strategic collaboration just 18 months later, this one focused on developing novel mRNA-based personalized cancer vaccines. Merck's anti-PD1-therapy blocks a protein that prevents the body's disease-fighting immune system from attacking cancer cells. Now, through a June 2016 agreement with Moderna, it is exploring the potential for combining this immunotherapy with mRNA vaccine technology to create personalized cancer treatments. The vaccine will be based on neoantigens from the surface of the patient's own tumor—mutations likely to be recognized as foreign by the immune system—and aim to trigger the body's immune response against the cancer. The hope is that combining the two drugs can help improve the outcome for cancer patients.

Moderna's rapid-cycle time, small-batch GMP manufacturing process will allow it to supply personalized cancer vaccines tailored to patients within weeks.

The personalized cancer vaccines will leverage Moderna's impressive rapid-cycle time, small-batch GMP manufacturing capabilities, which will allow vaccines tailored to patients to be supplied within weeks.

"There is a great, great quality of science at Merck. For them to come back 18 months after the first collaboration and want to do more together, that said something," says Bancel. "This is about learning faster than anyone else in the world how to make mRNA–based therapeutics, because patients are waiting for them."