Vertex and Moderna Hammer Out $315 Million+ Deal to Treat Cystic Fibrosis Using mRNA Technology

July 6, 2016
By Mark Terry, BioSpace.com Breaking News Staff

Vertex Pharmaceuticals , headquartered in Boston, and Moderna Therapeutics, based across the river in Cambridge, have signed a research and licensing deal to develop messenger RNA Therapeutics to treat cystic fibrosis (CF).

The collaboration is scheduled for three years and will focus on using mRNA therapies to help cells in the lung manufacture functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which does not work properly in CF patients. The two companies will also attempt to develop pulmonary mRNA delivery approaches.

“We are excited to begin this collaboration with Moderna to further broaden our pipeline and support our goal of treating the underlying cause of CF for all people with this disease,” said David Altshuler, Vertex’s executive vice president, global research, and chief scientific officer, in a statement. “The collaboration with Moderna underscores our commitment to invest in and pioneer multiple innovative approaches to expand and enhance the future treatment of CF. Moderna’s messenger RNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF.”

Moderna will receive $20 million in an upfront cash payment from Vertex. In addition, Vertex will invest $20 million in a Moderna convertible note that will convert to equity. This will give Vertex an ownership stake in Moderna. Developmental and regulatory milestones to Vertex could also hit $275 million, including $220 million in approval and reimbursement milestone payments. There are also tiered royalty payments on any future sales.

Moderna’s approach has been to use mRNA to force the body to manufacture its own drugs. Vertex successfully develops and commercializes CF drugs. Without the normally functioning protein in CF patients, sticky mucus accumulates in the lungs and other organs, which is often fatal. Moderna’s approach is to introduce into the lungs pieces of mRNA that code for the normal gene, where the cells will integrate that code and churn out the normal protein.

Moderna’s technology has definitely gotten the interest of the pharmaceutical industry. Last week, the company inked a $200 million deal with Merck to develop personalized cancer vaccines. It also has deals with AstraZeneca for cardiovascular diseases and cancer, and Alexion Pharmaceuticals for rare diseases. AstraZeneca coughed up $240 million for its deal, and Alexion paid $125 million.

Vertex received approval for its CF drug Kalydeco (ivacaftor) in 2012, for a specific gene mutation that affects about 4 percent of CF patients. Last year, its combination drug Orkambi (ivacaftor/lumacaftor) was approved by the U.S. Food and Drug Administration (FDA) for a different mutation.

At this time, the two companies are looking at introducing the mRNA by way of a spray in the mouth, rather than as an injectable drug. So far they’ve managed to get mRNA into lung cells in animals, although not in anything resembling CF.

“If this works in humans in clinical trials, that will make other [CF] drugs not relevant,” Stephane Bancel, Moderna’s chief executive officer, told Xconomy. “You will have addressed the root cause and fixed what is missing.”

Frank Vinluan, writing for Xconomy, said, “That means that Vertex has its hands on technology that could render its own CF drugs and pipeline obsolete. But Moderna’s mRNA approach is far from a sure thing. Of the many programs across several disease types that Moderna has disclosed, not one has yet produced human data.”

But as a type of gene therapy, mRNA is intriguing.

“We’re playing a very long game,” Bancel told the Boston Globe. “Our goals is to bring the best medicines to patients, and we always like to start with a partner who can bring expertise to a new area we’re looking at. It’s about whatever organ system we can get messenger RNA into. This is important because it brings it into the lungs.”