Vertex has stopped a Phase 1/2 study of VX-522 inhaled RNA therapy for the treatment of cystic fibrosis and has ceased development of the product, the company said in its most recent report of financial results. According to Vertex, the trial was stopped “after observing persistent tolerability issues in the study.” Vertex was partnered with Moderna on development of the therapy for patients who do not produce cystic fibrosis transmembrane conductance regulator (CFTR).
Moderna CEO Stéphane Bancel responded to the discontinuation in a blog post on the company’s web site, saying, “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”
Bancel noted, “This program represented our only inhaled therapeutic approach and utilized a unique formulation and delivery system, with no broader implications for our portfolio.”
