Good news about the combination VX-770 & VX-809

Question

Hello,

I noticed news about the VX-770 and VX-809 combination for patient with F508del mutation (deltaF508). Unfortunately I am not very strong in English and the automatic translators are not always clear.
Here are the sites where I could see the information:
http://newsblaze.com/story/2011060904034600002.bw/topstory.html

http://www.canada.com/health/Interim+Phase+Data+Showed+Combination+Improved+Function+Defective+Protein+that/4917737/story.html

I understand that the news were good
Could you enlighten us on this?

Thank you

A mother

Answer

Hello

Indeed Vertex, which markets these 2 molecules, has published the results of the first part of the clinical trial VX-809 in combination with VX-770. These results are positive; news is good about this first phase.
The objectives of this part were:
check the tolerability and safety of the combination
measure the effect of VX-809 combined to VX-770 on CFTR function by measuring the rate of chloride (a reduction rate means improving of CFTR function).
62 patients (> 18 years with two copies of the mutation DF508) were included and treated with three different types of treatment:
group 1: 14 days of VX-809 (200 mg) then 7 days of VX-809 (200 mg) + VX-770 (150 mg)
group 2: 14 days of VX-809 (200 mg) then 7 days of VX-809 (200 mg) + VX-770 (250 mg)
group 3: 14 days of VX-809 placebo then 7 days of VX-809 placebo + VX-770 placebo.
The rate of chloride was measured using the sweat test:
before treatment (day 0)
before the combination-dosing portion (day 14)
at the end of treatment (day 21).
As a reminder the sweat chloride of cystic fibrosis patient is > 60 mmol / L (millimoles per liter). The standard is 21 days)
and study their effect on the chloride

I hope that answers your question
Anna Ronayette

12.07.2011