An Update on Our Cystic Fibrosis Collaboration with Vertex

Today, Vertex announced that it has decided to discontinue the Phase 1/2 study of the investigational inhaled CFTR mRNA candidate for cystic fibrosis (CF), VX-522. Despite extensive efforts by both teams, we were not able to achieve a profile that supported continued development given tolerability issues. The early termination precludes assessment of efficacy and full safety and prevents further development of the VX-522 program.

The program brought together the strengths of Vertex and Moderna with a shared goal: to help people living with CF who do not benefit from existing therapies. Together our teams worked with urgency to explore the potential of delivering mRNA directly to the lungs to restore CFTR protein function.

This is a disappointing outcome, particularly for patients and families in the CF community who continue to wait for new options, and for the many colleagues across Vertex and Moderna who have dedicated years to this program. I want to personally thank our teams for their commitment, innovation and resilience. We are especially grateful to the trial participants, their families, investigators and site staff, trial networks and our partners for their essential contributions. We are thankful for the collaboration with Vertex and the shared commitment behind it.

This program represented our only inhaled therapeutic approach and utilized a unique formulation and delivery system, with no broader implications for our portfolio. That said, the learnings are important and will inform our broader scientific efforts as we continue to advance our mRNA pipeline across multiple modalities.

At Moderna, our Mission remains unchanged. We will keep pursuing bold science in service of patients, including those with rare and serious diseases where the need is greatest.

We are proud of the work done and we remain focused on the opportunities ahead.