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

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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?