Moderna Therapeutics still hasn’t produced data in humans supporting its technology, which is meant to turn our bodies into little drugmaking factories. But the Cambridge, MA-based company nonetheless keeps grabbing the attention, and cash, of other drugmakers.
Moderna said today that it will work with Merck (NYSE: [[ticker:MRK]]) to develop “personalized” cancer vaccines based on its messenger RNA work. Moderna will get $200 million to build a manufacturing facility in suburban Boston to make the vaccines, and lead the early work on a development program consisting of “multiple” studies in several types of cancer. Those studies will combine a Moderna vaccine with Merck’s cancer immunotherapy drug pembrolizumab (Keytruda). Moderna CEO Stephane Bancel (pictured) said in a statement that the goal is to begin the trials next year.
After human proof of concept studies, Merck will have the option to share the rights to those vaccines, along with the development costs and profits down the road, for an unspecified payment.
The deal is the industry’s latest vote of confidence in Moderna, the high-flying yet mysterious maker of mRNA drugs. For those unfamiliar with the company, the idea behind Moderna is to inject synthetic mRNA strands into the body so patients’ cells can produce their own therapeutic proteins. It’s a novel way of making drugs that is also completely unproven in humans. Moderna began its first clinical trial in 2015, and now has a second study underway.
Yet the allure of mRNA drugmaking has enabled Moderna to land a string of substantial deals and financings. Merck, for instance, was already a Moderna equity holder and partner before today’s deal. In early 2015, Merck paid Moderna $50 million up front, and made an additional $50 million equity investment, as part of a three-year research deal to develop mRNA-based drugs and vaccines. That agreement was one of three lucrative partnerships for Moderna, adding to deals with AstraZeneca ($240 million up front) and Alexion Pharmaceuticals ($100 million up front). Combined with the preponderance of cash Moderna has raised—its last round in January 2015 clocked in at $450 million, a record biotech financing—the company has been able to amass a prohibitive war chest and turn itself into something of a startup incubator, even though it has yet to produce human clinical data and is still privately held.
Through a growing web of startup subsidiaries and partnerships, Moderna has been developing mRNA therapies for a range of diseases—cancer, rare diseases, cardiovascular disorders, and more. The core concept is that the parent company does the early stage work, while its startup subsidiaries do more advanced development. But one of its startups, Caperna, was specifically formed around the idea of personalized cancer vaccines, following in the footsteps of a few other new companies, Neon Therapeutics and Gritstone Oncology. All are trying to identify what are known as “neoantigens,” small protein fragments that are unique to tumor cells and can tip the immune system off that a cancer is growing. The presence or lack of neoantigens is believed to be one reason folks respond, or don’t, to checkpoint inhibitors, the name for cancer immunotherapy drugs like Merck’s pembrolizumab.
The idea behind these companies is to analyze peoples’ tumors, find the key neoantigens, engineer synthetic versions of them, and load them into a vaccine—which hopefully trains the immune system to spot the cancer. The concept has gained a lot of traction, but there are significant challenges ahead as well, as Neon co-founder Nir Hacohen explained at an Xconomy event in April.
Bancel previously told Xconomy that Moderna aims to do something similar, just faster and in a way that could lead to a more significant immune response. Merck has bought in.
“Combining immunotherapy with vaccine technology may be a new path toward improving outcomes for patients,” said Merck’s R&D chief, Roger Perlmutter, in a statement. “While the area of personalized cancer vaccine research has faced challenges in the past, there have been many recent advances, and we believe that working with Moderna to combine an immuno-oncology approach, using [pembrolizumab], with mRNA-based personalized cancer vaccines may have the potential to transform the treatment of cancer.”
