Vertex Seeks Trials of Therapies for Cystic Fibrosis, Diabetes

(Bloomberg) -- Vertex Pharmaceuticals Inc. plans to seek permission from U.S. regulators next year to advance two experimental therapies for cystic fibrosis and Type 1 diabetes into human trials. 

The Boston-based biotech is conducting studies to support investigational new drug applications, which drugmakers are required to submit before studying experimental therapies in humans, Vertex Chief Operating Officer Stuart Arbuckle said in an interview Tuesday. 

Vertex and partner Moderna Inc. are developing an mRNA treatment for the 10% of cystic fibrosis patients Vertex’s current drugs can’t help. Vertex is also working on transplanting insulin-producing pancreatic cells into Type 1 diabetes patients using a protective medical device. If successful, the approach would eliminate the need for treating transplant patients with immune-suppressing drugs. 

Vertex is on track to file IND applications for both programs next year, Arbuckle said. He declined to provide a more specific timeline. 

Investors are searching for growth beyond cystic fibrosis, which causes severe damage to the lungs, digestive system and other organs. Vertex is pursuing a number of new disease areas, including Type 1 diabetes, sickle cell disease and severe kidney disease. 

“We know it’s a show-me world and people are looking for evidence our strategy’s right,” Arbuckle said. Vertex plans to share data from trials in other programs such as severe kidney disease in the coming months. 

“Hopefully if they’re positive, they’ll allow people to see the strategy we’re pursuing does have a disproportionately high chance for success,” Arbuckle said. “That’s what we believe, and that’s what we’re expecting to see.”

For now, cystic fibrosis drugs remain Vertex’s profit engine. Growth in Vertex’s triple-combination therapy caused the drugmaker to raise its full-year sales outlook. Vertex forecast product revenue between $7.4 billion to $7.5 billion, up from the previously guided range of $7.2 billion to $7.4 billion.

Shares of Vertex rose 1.5% after hours on Tuesday. They had fallen 23% this year as of Tuesday’s close. 

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