UPDATED: Vertex, Moderna pair to create mRNA cystic fibrosis treatment

A researcher in a white lab coat

Vertex Pharmaceuticals ($VRTX) and Moderna Therapeutics have partnered in a deal potentially worth more than $300 million to discover and develop messenger ribonucleic acid (mRNA) therapeutics for cystic fibrosis (CF). This is the second major partnership for Moderna in the last week.

In late June, the Cambridge, MA-based startup also partnered with Merck ($MRK) for $200 million upfront plus an additional undisclosed payment on human proof-of-concept data in a deal to create mRNA-based personalized cancer vaccines.

Between these two latest deals, Moderna racked up $1 billion in cash at June 30, giving it several years of runway. With all that money in hand, it's under no pressure to go public any time soon. The startup already has a pair of ongoing Phase I clinical trials and expects to move more programs into the clinic by year end. 

The latest deal with Vertex entails a $20 million cash payment upfront, with an additional $20 million investment in the form of a convertible note that converts to equity. In addition, it will pay Moderna up to $275 million in development and regulatory milestones--of which $220 million are specifically tied to approval and reimbursement--as well as undisclosed, tiered royalties on future sales.

And, unlike all its prior deals, including ones with the likes of AstraZeneca ($AZN) and Alexion Pharmaceuticals ($ALXN), this time the Vertex deal is focused on a single asset rather than an expansive portfolio of assets.

“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,” said Vertex EVP of Global Research and CSO Dr. David Altshuler in a statement.

The partners will conduct exclusive research, development and commercialization work for mRNA treatments designed to enable cells of people with CF to produce functional CFTR proteins in the lungs. Moderna will lead the discovery portion, enlisting Vertex’s assay platform based on human bronchial epithelial cells of multiple different CF gene mutations.

Vertex is to lead all preclinical, development and commercialization activities for any resulting mRNA therapeutics--and it is slated to fund all expenses from the collaboration. CF is a core indication for Vertex, whose Orkambi combo treatment for CF patients with the F508del mutation in their CFTR genes was approved by the FDA last year. It includes Vertex’s Kalydeco for CF patients with different CFTR mutations, which has an approval dating back to 2012.

“This collaboration is illustrative of our consistent strategy to partner with world class therapeutic experts in order to maximize the potential of leveraging mRNA as a novel therapeutic approach across a spectrum of serious diseases,” said Moderna CEO Stéphane Bancel in the release. “We’re particularly excited to explore the potential of pulmonary delivery as a new modality to bring transformative mRNA medicines to patients and address unmet needs.”

- here is the release

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