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Vertex Nails Pivotal Study for Cystic Fibrosis, Racing Toward Market With Second Drug

Xconomy Boston — 

Vertex Pharmaceuticals has been moving in this direction for a long time, but it can now say with confidence that it is on its way to shaking up the standard of care for not just one disease this year, but two.

The Cambridge, MA-based biotech company (NASDAQ: VRTX), which has operations in San Diego, is announcing today it has passed a pivotal clinical trial of 161 patients with cystic fibrosis who were randomly assigned to get the company’s VX-770 pill or a placebo. The study found that patients on the Vertex drug had a 17 percent relative improvement in their ability to force out air from their lungs in one second—a common measure of success in treating lung disease—compared with those on a placebo. The effect lasted over the entire 48-week course of the study. Side effects included headache, upper respiratory tract infections, nasal congestion, rash, and dizziness, although more patients on the placebo group dropped out of the study early because of side effects, Vertex said.

While 17 percent relative improvement in breathing capacity for people with a deadly lung disease might not sound like much, this result represents a watershed for a portion of the 30,000 people in the U.S. with cystic fibrosis. Vertex is valued primarily for a drug (telaprevir) that has shown an ability to double the cure rate for patients with hepatitis C liver infections, but the company could have a similar life-changing effect for many patients with this genetic disorder. Cystic fibrosis, the result of a genetic mutation, causes the buildup of thick, sticky mucus in the lungs, which suffocates people over time, allowing a typical life expectancy of about 37 years. Doctors currently treat the symptoms of the disease, but Vertex’s drug could become the first FDA-approved therapy that works by altering an underlying faulty protein at work in patients with cystic fibrosis.

This drug isn’t for everybody—it was tested in a subpopulation of just 4 percent of cystic fibrosis patients with an abnormality known as G551D—but it’s the kind of result that scientists hope they will be able to build on to expand treatment to more patients with the deadly lung disease.

The results Vertex is releasing today from a study called Strive, which will be discussed later this year at a medical meeting, are “far beyond our expectations,” Vertex said in a statement. The firm plans to put together an application before the end of this year to seek regulatory clearance in the U.S., and Europe, to start marketing VX-770.

“The results from Strive are highly encouraging for the CF community and provide scientific evidence supporting our long-standing belief that targeting the underlying defect of CF may have a profound effect on the disease,” said Robert Beall, the president and CEO of the Cystic Fibrosis Foundation, in a company statement. “We have much more to do to eliminate this disease, but these data are extremely exciting.” (The CF Foundation helped develop VX-770.)

Vertex’s trial would have been deemed a success if it delivered just 4.5 percentage points of absolute improvement in patients’ breathing, as measured by the standard clinical trial goal (forced expiratory volume in one second, or FEV1). The actual result, in absolute terms from the beginning to the end of the study, was a 10.5 percentage point improvement in lung function.

The new Vertex drug was delivered as a twice-daily 150 milligram pill. Without going through this data set chapter and verse, there’s a lot here for scientists and physicians to comb through.

Besides reaching the main goal of the study, researchers saw several advantages for the Vertex drug on secondary measurements. Patients on the Vertex drug were 55 percent less likely to suffer what’s known as a pulmonary exacerbation, in which the signs of symptoms of disease get worse and force them onto antibiotics to kill nasty bugs building up in their lungs. Patients were able to gain an average of seven pounds, rather than waste away. And, like Vertex saw in earlier studies, patients had significantly less salt in their sweat, which is a key sign that the Vertex drug is delivering the lung function improvement by doing what it’s supposed to do at the molecular level.

Cystic fibrosis patients and their parents are a highly engaged and motivated group of people, so this is a result that will surely have the message boards buzzing today. Bonnie Ramsey, one of the research leaders who helped coordinate early VX-770 studies for the CF Foundation—which has put more than $75 million into supporting the Vertex R&D program over the years—has said this drug has the potential to be a “man on the moon” for CF.

Meanwhile, Wall Street has been fixated on the telaprevir story for a couple of years, although the tide of interest started to switch last month toward cystic fibrosis, Vertex CEO Matt Emmens said during an interview at the JP Morgan Healthcare Conference. Vertex has another drug in the works for CF, VX-809, that’s designed to be complementary to VX-770. More data on the combination is expected later this year, and that will have to be factored in to the bigger picture of what Vertex is doing. How much a drug like this, in a small market, might generate in sales is a big question analysts will continue to drill into over the weeks and months to come. But this result has the potential to change the storyline around the company, Emmens says.

Matt Emmens

To help stir more interest among investors in the CF story, Vertex passed around straws before an investor presentation last month, and asked them to try to breathe through straws so they could feel what it’s like to have lung impairment like a CF patient. I didn’t attend that presentation—I’m kicking myself for skipping it—but afterwards I heard this generated quite a few “aha” moments from people in the room who started to get the idea for the first time.

“We’ve talked about this, and watched people’s eyes glaze over in the two years I’ve been here.” Emmens says. “We used a little bit of showmanship, but it was to show them this is a severe disease, it’s an orphan [rare] indication, patients really know about our drug, and, ‘Oh boy, try breathing once through this straw.’ Here’s what it feels like.” He adds: “People are convinced we have another drug, and we have a good one.”

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4 responses to “Vertex Nails Pivotal Study for Cystic Fibrosis, Racing Toward Market With Second Drug”

  1. Brief comment from Bonnie Ramsey of Seattle Children’s Hospital, a principal investigator of the Phase 3 Strive study of Vertex’s VX-770.

    “I have only been able to see the results briefly today [2/22] because of the embargo. However, as noted in the press release, these are impressive findings. VX770 is the first therapy to treat the abnormal CFTR protein so it is a big step forward in this disease. The results of the previous Phase 2 study with this drug were published last year in the New England Journal of Medicine (Accurso is the first author). It is important to note, however, that it has been shown to work only in patients with the G551D mutation which is about 5% of the CF population worldwide. There are ongoing studies to see if it will have any effect in the most common mutation, delta F508.”

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  3. joanne poskey says:

    Are studies being done on children age 2 to 6 yrs.? If not, why not?