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Rebjane
05-29-2012, 01:16 PM
http://www.businesswire.com/news/home/20120529005664/en/Vertex-Corrects-Additional-Data-Interim-Analysis-Phase

Anyone care to explain what this means?

Aboveallislove
05-29-2012, 01:19 PM
Press release: See http://investors.vrtx.com/releasedetail.cfm?ReleaseID=677520

BEFORE YOU FREAK--NOTE: The mean absolute improvement was 8.5%. Vertex had not released the mean absolute before, only # of individuals with absolute improvement 5% or 10%. The revised numbers are substantially worse for that. BUT the 8.5% mean absolute is GREAT NEWS. Remember Kalydeco was a 10% mean absolute improvement and that Pulmozyme and Hyper Tonic saline were around 5%. Vertex is still planing on moving to Phase 3 and with 8.5% I am still confident of success.

The revised data is not nearly as good as the original. The third party who did the data crunching screwed up. BUT the mean absolute is the key and it is still good.

rainsmom
05-29-2012, 01:46 PM
give me an example of 8.5 mean% if your fev1 is 80 then what would that put it to? may be such a silly question but b/c im not a wiz at %s i need it spelled out lol

Aboveallislove
05-29-2012, 01:55 PM
Rainsmom,
The 8.5% is mean absolute, meaning the average improvement of all people. More later. DS Screaming.

GenH
05-29-2012, 02:30 PM
I've just read the press release several times trying to make sure I'm understanding the figures correctly, but my interpretation is that on average, the 37 participants on Kalydeco/VX809 improved 4% from their starting FEV1 (8.5% compared to placebo, but placebo went down 4.6%, its likely at least 1/11 people in the placebo group became sick, hence the 4.6% drop). <span class="commentBody" data-jsid="text">The final Kalydeco trial with G551D had a much larger sample size, and in that trial the placebo group dropped about 1.5% over the 48 weeks, so when they say that Kalydeco had a 10.5% improvement compared to placebo, the actual improvement in the Kalydeco group from their starting point was more like 9%. I'm reading these figures off the graph so they are not 100% accurate. A 4% overall improvement is still positive, as is 35% improving by at least 5% and 19% improving by 10%. A larger sample size (37 is not that large) & a longer trial will tell us more.

stranger
05-29-2012, 04:25 PM
Good news / bad news:
Good news: Vertex is trying for a Phase III trial of this combo which would have appeared unlikely a few months back because the side chat was how 'disappointing' the initial data was.
Bad news: Vertex' credibility has taken a hit which *may* impact their ability to fast-track this with the FDA. Up till now, they have been on auto-pilot with the FDA. Thus time to market may have changed assuming the Phase III results pan out.

I still prefer having these types of discussions rather than ones we were having ... five years ago...

GenH
05-29-2012, 04:32 PM
rainsmom, an absolute improvement of 8.5% would take you from 80 to 88.5% (but the trial had a mean 4% absolute improvement with the Kalydeco group from their starting pfts). I just posted this on the VX809/770 facebook group, I figured I would repost it here incase there is anyone else wondering about relative v absolute improvement. Absolute improvement- a 5% increase means I increase from 50 to 55%. Relative improvement means that my 5% increase from 50 to 55 is a 10% increase from my starting 50% (5/50 * 100), so they call that a 10% relative improvement. At 50% it works out that the relative compared to the absolute is exactly double, at 100% they are equal. Thats why in the results that were just released there are less people with a 5% and 10% improvement now (as the starting point was probably 60-80%, the relative % will always be higher than the absolute%).

trw144
05-29-2012, 07:50 PM
Also bare in mind that dosing was split between 3 groups (200,400,600mg) and in the conference call they said the 600mg dose had "a more consistent and more robust effect as compared to other doses". This is where I see the main hope in the results.