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Vertex to test new class of cystic fibrosis drugs in patients

Vertex chief executive Jeffrey Leiden said the Boston-based company’s long-term goal “is to treat all patients with CF.” The company’s shares rose 6 percent in after-hours trading.Craig F. Walker/Globe staff/Globe Staff

Vertex Pharmaceuticals Inc. said Tuesday it will begin clinical trials for a new class of “triple combination” drug candidates that could nearly double the number of cystic fibrosis patients its medicines can treat.

Patient advocates and investors have been awaiting the move because it puts the Boston-based biotech company on a path to marketing drugs for about 24,000 additional cystic fibrosis patients worldwide with multiple types of genetic mutations. Together, they represent the largest remaining population of untreated patients with the obstructive lung disease.

Vertex announced its plans for the clinical trials after financial markets closed, as part of its quarterly earnings report. Its shares were up 6 percent in after-hours trading.


“This is the type of news the entire CF community has been waiting for,” said Bob Coughlin, president of the Massachusetts Biotechnology Council and the father of a 14-year-old boy with cystic fibrosis. “Any time they can unlock the mysteries of these additional mutations is music to the ears of the whole community.”

Rival drug maker Galapagos NV, based in Belgium, is also developing treatments for some of the same sets of cystic fibrosis patients. But by advancing its triple combination treatments into mid-stage clinical studies, Vertex is “comfortably ahead of next-generation competitors,” John Scotti, biopharma analyst at New York research firm Evercore ISI, wrote in a note to investors.

Vertex already sells the drugs Orkambi and Kalydeco, which together have the potential to treat about 29,000 patients worldwide who have two more common CF mutations. But only about 9,000 of those patients in the United States and several other countries are currently using the drugs.

“Our long-term goal is to treat all patients with CF,” Vertex chief executive Jeffrey Leiden told stock analysts in a conference call Tuesday afternoon.


Vertex said it would begin enrolling patients in a pair of clinical studies to test a drug combination that includes ivacaftor — which already is sold as Kalydeco — and a so-called corrector known as VX-661 because it corrects a part of a malfunctioning protein in patients.

The third compound in the combination will be one of two other correctors that will be used separately in each of the trials. All of the drugs will be taken orally.

Scientists at Vertex’s research center in San Diego have been developing and testing the compounds in their labs since 2000, said Paul Negulescu, the company’s San Diego site head. “We’ve been working on this approach for a long time, and this is the next big step that could allow us to treat the vast majority of cystic fibrosis patients,” Negulescu said in an interview.

Vertex has asked the Food and Drug Administration for permission to begin enrolling patients in both studies by the end of 2016. The company is expected to post its first data from them in the second half of 2017.

For the three months ending Sept. 30, Vertex reported that its sales of Orkambi climbed 79 percent to $234 million from the same quarter last year, while Kalydeco sales rose 6 percent to $176 million. The company narrowed its quarterly loss to $42 million from $95 million a year ago. It posted overall CF revenue of $410 million, up from $297 million last year. The numbers were in line with Wall Street expectations.


Robert Weisman can be reached at robert.weisman@globe.com. Follow him on Twitter @GlobeRobW.