Your donation will make a difference:
Cystic Fibrosis Trust

Triple Therapy Trial - what's it all about?

This morning the drug manufacturer Vertex announced some encouraging preliminary results for their next generation of precision medicine. But what does it all mean? We asked Dr Janet Allen, Director of Strategic Innovation at the Trust, to pull out the key findings from the press release.

First things first - what is the triple combination therapy?

Put simply, the drug uses a combination of ivacaftor (which you may know as Kalydeco), Vertex’s new product tezacaftor, and a third compound, to tackle the underlying cause of cystic fibrosis – the mutation of the CF gene.

In these trials, Vertex has been trialing three variations of the triple combination drug, with one of three new compounds (VX-152, VX-440 and VX-659) as that third element, to see how they improve lung function.

The data we are looking at was first trailed by Fred van Goor from Vertex when he presented pre-clinical data for the triple combination therapy at the UK Cystic Fibrosis Conference last year. (Visit our conference page to find out what's in store at this year's conference!)

Ok, so who could benefit?

The trial tested the effects of the triple combination in people with one copy of the F508del mutation and one copy of what is known as a ‘minimal function mutation’ – these are Class I–III mutations where little or no working CFTR protein is produced. These people do not respond to Orkambi (which works with people who have two copies of the F508del mutation). In short, this triple combination could benefit around 90% of the CF population, with a severe and difficult-to-treat form of cystic fibrosis.

And what have they found?

The three versions of the triple combination all appear to improve lung function (FEV1) significantly, between 9.6–12%, depending on the compound and the trial (two were tested in a Phase II trial and one in Phase I).

These are very encouraging results, but it is still early days. The press release only announces very top-line data, and so far they have only conducted Phase I and Phase II trials, which make sure the treatment is safe and works in a small test group. If comparable results were found in a Phase III trial, that could be very good news.

So what’s next?

According to Vertex’s statement, they hope to develop one or more of these variations of the triple combination therapy in a Phase III trial, beginning in the first half of 2018. Phase III trials tend to involve hundreds of people with CF across many countries, and can last between 6–12 months.

Where can I find out more?

Watch our video on the different mutations of the CF gene, and how they affect the production of CFTR protein.

Find out more about the stages of clinical trials in this short animation.

Lear more about clinical trials and how to get involved.