Cystic fibrosis drug trial success boosts Vertex

Markus MacGill

pharmaphorum

Vertex’s shares have soared on the news that its cystic fibrosis candidate improved patients’ lung function in a phase II trial.

The drug, known as VX-661, showed lung function improvement in people who have two copies of the most common mutation in cystic fibrosis, in the F508del gene.

The value of Vertex rose by more than $6 billion as shares surged by 54 per cent in after-hours trading. Vertex has another cystic fibrosis drug in development and the company’s R&amp,D chief says it is “making significant progress” in the field.

“Patients in the 100 and 150 mg combination dose groups showed statistically significant mean relative improvements in lung function, versus placebo, of 9.0 percent (p=0.01) and 7.5 percent (p=0.02), respectively, at Day 28. In contrast, patients who received placebo showed a 0.03 percent mean relative change in lung function at Day 28 (within-group).”

Vertex press release.

“With these data and the recent initiation of a phase III programme for a combination of our lead CFTR corrector VX-809 with ivacaftor, we are making significant progress toward our goal to help many more people with CF.”

Peter Mueller, chief scientific officer and executive vice president of global R&amp,D, Vertex.

Vertex says it now intends to talk to regulators about plans towards future marketing approval for VX-661, which was also well tolerated by patients in the mid-stage trial.

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