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View Full Version : VX 809/770 Phase 2 interim results - 5-10% improvement in lung function



jamesB
05-07-2012, 10:38 AM
<a href="http://investors.vrtx.com/releasedetail.cfm?ReleaseID=670755">http://investors.vrtx.com/releasedetail.cfm?ReleaseID=670755</a>

<h2>Interim analysis showed 46% of patients experienced at least 5% absolute improvement in lung function (FEV1) from baseline; 30% of patients experienced at least 10% absolute improvement.</h2>

I did notice that sweat chloride levels didn't show a statistically significant change.
Still encouraging results.
Vertex up 54% premarket.

jamesB
05-07-2012, 10:38 AM
<a href="http://investors.vrtx.com/releasedetail.cfm?ReleaseID=670755">http://investors.vrtx.com/releasedetail.cfm?ReleaseID=670755</a>

<h2>Interim analysis showed 46% of patients experienced at least 5% absolute improvement in lung function (FEV1) from baseline; 30% of patients experienced at least 10% absolute improvement.</h2>

I did notice that sweat chloride levels didn't show a statistically significant change.
Still encouraging results.
Vertex up 54% premarket.

Rebjane
05-07-2012, 10:49 AM
What mutation is this for?

Rebjane
05-07-2012, 10:49 AM
What mutation is this for?

jamesB
05-07-2012, 10:54 AM
From the release:
<h1>'People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation'</h1>

jamesB
05-07-2012, 10:54 AM
From the release:
<h1>'People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation'</h1>

saveferris2009
05-07-2012, 11:01 AM
Thanks for posting!

saveferris2009
05-07-2012, 11:01 AM
Thanks for posting!

Aboveallislove
05-07-2012, 11:05 AM
Live conference call on this on going.

Aboveallislove
05-07-2012, 11:05 AM
Live conference call on this on going.

jamesB
05-07-2012, 12:10 PM
From listening to the conference call, they said the results exceeded expectations.

Didn't release results of the single F508 del as not enough patients have completed enough of the study.

jamesB
05-07-2012, 12:10 PM
From listening to the conference call, they said the results exceeded expectations.

Didn't release results of the single F508 del as not enough patients have completed enough of the study.

Rebjane
05-07-2012, 12:11 PM
I was wondering about single Delta F508...Thanks for the info

Rebjane
05-07-2012, 12:11 PM
I was wondering about single Delta F508...Thanks for the info

jamesB
05-07-2012, 12:24 PM
They also said, no reason the current results shouldn't be maintained over the duration of the trial. A 10% increase in FEV was about what they saw in phase 3 trials of 770 for G511D. Moving forward as quickly as possible with further studies to get drug combos to market.

jamesB
05-07-2012, 12:24 PM
They also said, no reason the current results shouldn't be maintained over the duration of the trial. A 10% increase in FEV was about what they saw in phase 3 trials of 770 for G511D. Moving forward as quickly as possible with further studies to get drug combos to market.

jamesB
05-07-2012, 12:47 PM
Finally from the call,

Similar level of sweat chloride reductions compared to the earlier studies, but there is no correlation between FEV1 and sweat chloride. FEV1 is what they are focused on.

Accelerated approval is something they'll be discussing with FDA.

Can't comment on what's more important - higher dose or longer duration - results are interim.

Compassionate use - too early to say.

Full results released in a couple of months - probably July.

jamesB
05-07-2012, 12:47 PM
Finally from the call,

Similar level of sweat chloride reductions compared to the earlier studies, but there is no correlation between FEV1 and sweat chloride. FEV1 is what they are focused on.

Accelerated approval is something they'll be discussing with FDA.

Can't comment on what's more important - higher dose or longer duration - results are interim.

Compassionate use - too early to say.

Full results released in a couple of months - probably July.

Rebjane
05-07-2012, 01:03 PM
Full results from Phase 2 jamesB?

Rebjane
05-07-2012, 01:03 PM
Full results from Phase 2 jamesB?

jamesB
05-07-2012, 01:19 PM
Yeah I think that's what they are saying Rebjane.

Will include all the different breakdowns for dosage and time and results for both single DF508 and the double delta's.

jamesB
05-07-2012, 01:19 PM
Yeah I think that's what they are saying Rebjane.

Will include all the different breakdowns for dosage and time and results for both single DF508 and the double delta's.

bigstar
05-07-2012, 01:22 PM
I think this is very very promising. I mean a 5-10% improvement of FEV1 in a month seems very nice to me. Lets keep our fingers crossed.

bigstar
05-07-2012, 01:22 PM
I think this is very very promising. I mean a 5-10% improvement of FEV1 in a month seems very nice to me. Lets keep our fingers crossed.

Hardak
05-07-2012, 02:04 PM
Yeah 5-10% is a big chunk, frankly I would just like to see a decrease in my cough.

Hardak
05-07-2012, 02:04 PM
Yeah 5-10% is a big chunk, frankly I would just like to see a decrease in my cough.

jamesB
05-07-2012, 02:05 PM
I agree bigstar. The only negative is the sweat chloride levels compared to the significant changes we saw with 770 and G511D. Is certainly good news that FEV1 levels were comparable though.

Will be interesting to see the sweat chloride results from the 2a 661 trial. They shouldn't be far away at all given how quickly these interim results were released.

jamesB
05-07-2012, 02:05 PM
I agree bigstar. The only negative is the sweat chloride levels compared to the significant changes we saw with 770 and G511D. Is certainly good news that FEV1 levels were comparable though.

Will be interesting to see the sweat chloride results from the 2a 661 trial. They shouldn't be far away at all given how quickly these interim results were released.

byrne819
05-07-2012, 02:19 PM
At the risk of getting too far ahead of myself, does anyone know how these drugs would affect other drug regimens, e.g., enzymes, physical therapy, nebs? Will people continue these treatments? What is the experience of people on Kalydeco? Have they been continuing treatments? Thanks!

byrne819
05-07-2012, 02:19 PM
At the risk of getting too far ahead of myself, does anyone know how these drugs would affect other drug regimens, e.g., enzymes, physical therapy, nebs? Will people continue these treatments? What is the experience of people on Kalydeco? Have they been continuing treatments? Thanks!

JENNYC
05-07-2012, 02:20 PM
Thanks so much for keeping us updated jamesB!!!

JENNYC
05-07-2012, 02:20 PM
Thanks so much for keeping us updated jamesB!!!

cfsucks
05-07-2012, 02:40 PM
good news- i want to see the impact on people with only one df508 gene!

cfsucks
05-07-2012, 02:40 PM
good news- i want to see the impact on people with only one df508 gene!

rmotion
05-07-2012, 02:41 PM
Well thats good maybe they'll keep going with it and get it out there sooner and continue with the vx661
I know they want a grand slam home run but this is a double and we'll take that, huh!

rmotion
05-07-2012, 02:41 PM
Well thats good maybe they'll keep going with it and get it out there sooner and continue with the vx661
I know they want a grand slam home run but this is a double and we'll take that, huh!

sdelorenzo
05-07-2012, 03:14 PM
I need someone to explain this to me.
17/37 FEV1 was greater than 5%.
11/37 FEV1 was greater than 10%.

Does that mean 11 of the 17 were greater than 10%, so only 46% saw a change greater than 5%? So 54% did not see a significant change?
or does it mean a total of 85% saw a change greater than 5%?
Thank you,
Sharon

sdelorenzo
05-07-2012, 03:14 PM
I need someone to explain this to me.
17/37 FEV1 was greater than 5%.
11/37 FEV1 was greater than 10%.

Does that mean 11 of the 17 were greater than 10%, so only 46% saw a change greater than 5%? So 54% did not see a significant change?
or does it mean a total of 85% saw a change greater than 5%?
Thank you,
Sharon

jamesB
05-07-2012, 03:42 PM
That's a good question. My interpretation was that those that saw the 10% increase were a part of the 17. So only 46% saw an improvement. The other 54% increased by less than 5%. I certainly could be wrong though - and hope I am. Analysts were pressing for an average FEV1 increase (mean) which Vertex said wouldn't be available until the final results were released.

jamesB
05-07-2012, 03:42 PM
That's a good question. My interpretation was that those that saw the 10% increase were a part of the 17. So only 46% saw an improvement. The other 54% increased by less than 5%. I certainly could be wrong though - and hope I am. Analysts were pressing for an average FEV1 increase (mean) which Vertex said wouldn't be available until the final results were released.

trw144
05-07-2012, 06:21 PM
Yes, the 46% over 5% FEV increase also includes the 30% that saw a 10% increase. It must be noted though that the study was split into three groups - all had the same dosage of kalydeco (vx-770) but the VX-809 dosage was varied between the three groups (200mg,400mg,600mg). They would nt say in the conference call but I was wondering whether their was a correlation between the FEV increase and the dosage - hence not everyone in the study achieved the same levels of increase (maybe this is just wishful thinking).

Anyway, Vertex have seen enough that they are pushing ahead with the next stage and are not going to wait for the results of VX-661 (which it had been suggested was the better potentiator).

trw144
05-07-2012, 06:21 PM
Yes, the 46% over 5% FEV increase also includes the 30% that saw a 10% increase. It must be noted though that the study was split into three groups - all had the same dosage of kalydeco (vx-770) but the VX-809 dosage was varied between the three groups (200mg,400mg,600mg). They would nt say in the conference call but I was wondering whether their was a correlation between the FEV increase and the dosage - hence not everyone in the study achieved the same levels of increase (maybe this is just wishful thinking).

Anyway, Vertex have seen enough that they are pushing ahead with the next stage and are not going to wait for the results of VX-661 (which it had been suggested was the better potentiator).

saveferris2009
05-07-2012, 08:30 PM
5% increase in FEV1 isn't too impressive, in my book. So for example, if you have an 80% FEV1, you would then be at 84%. Cool, but not amazing. The 20% FEV1 from Kalydeco with G551D is more impressive.

But I'll take whatever I can get. I'm just not surprised that they'll still be studying 661

saveferris2009
05-07-2012, 08:30 PM
5% increase in FEV1 isn't too impressive, in my book. So for example, if you have an 80% FEV1, you would then be at 84%. Cool, but not amazing. The 20% FEV1 from Kalydeco with G551D is more impressive.

But I'll take whatever I can get. I'm just not surprised that they'll still be studying 661

hbollotte
05-07-2012, 09:40 PM
as someone asked in an earlier post, while taking this drug does the patient stay on their breathing treatment regimen????

hbollotte
05-07-2012, 09:40 PM
as someone asked in an earlier post, while taking this drug does the patient stay on their breathing treatment regimen????

saveferris2009
05-07-2012, 10:09 PM
yes. this isn't a cure

saveferris2009
05-07-2012, 10:09 PM
yes. this isn't a cure

musclemania70
05-07-2012, 11:05 PM
I think it depends on the patient whether or not he or she would continue with treatments. As with Brady, he is young enough that he has no damage and is slowly decreasing his treatments as time goes on (g551d).

Each patient would probably have to use their judgment with their doctor's guidance about how to proceed with treatments in the future if their lung function improves with the new drugs.

Each patient will have to be handled case to case. Young ones with the least lung disease will have the best chances for staying treatment free......

musclemania70
05-07-2012, 11:05 PM
I think it depends on the patient whether or not he or she would continue with treatments. As with Brady, he is young enough that he has no damage and is slowly decreasing his treatments as time goes on (g551d).

Each patient would probably have to use their judgment with their doctor's guidance about how to proceed with treatments in the future if their lung function improves with the new drugs.

Each patient will have to be handled case to case. Young ones with the least lung disease will have the best chances for staying treatment free......

bkc3
05-07-2012, 11:29 PM
It is exciting to see progress. Looking forward to more information as the trial moves forward.

bkc3
05-07-2012, 11:29 PM
It is exciting to see progress. Looking forward to more information as the trial moves forward.

hammerpocket
05-08-2012, 03:00 AM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> 5% increase in FEV1 isn't too impressive, in my book. So for example, if you have an 80% FEV1, you would then be at 84%. Cool, but not amazing.</end quote>
I made this point in the other thread, but I'll repeat it here:
I think we may be reading this wrong. Notice the change is written as "an absolute improvement ... of 5 percentage points" rather than "a 5% improvement." If I understand it coreectly, an "absolute improvement" is the improved percentage<em>minus</em>the original precentage. In your example, the FEV1 of 80% with an absolute improvement of 5 percentage points would be 85%. (A <em>relative improvement</em>of 6.25%.) That may not seem impressive, but if the FEV1 started at 50%, the absolute improvement would result in FEV1=55% (or a 10% relative improvement).
And remember, 30% had an absolute improvement of <em>10</em>precentage points.

hammerpocket
05-08-2012, 03:00 AM
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>saveferris2009</b></i> 5% increase in FEV1 isn't too impressive, in my book. So for example, if you have an 80% FEV1, you would then be at 84%. Cool, but not amazing.</end quote>
I made this point in the other thread, but I'll repeat it here:
I think we may be reading this wrong. Notice the change is written as "an absolute improvement ... of 5 percentage points" rather than "a 5% improvement." If I understand it coreectly, an "absolute improvement" is the improved percentage<em>minus</em>the original precentage. In your example, the FEV1 of 80% with an absolute improvement of 5 percentage points would be 85%. (A <em>relative improvement</em>of 6.25%.) That may not seem impressive, but if the FEV1 started at 50%, the absolute improvement would result in FEV1=55% (or a 10% relative improvement).
And remember, 30% had an absolute improvement of <em>10</em>precentage points.

jamesB
05-08-2012, 05:42 AM
Interesting point Hammer. Thanks for posting.

jamesB
05-08-2012, 05:42 AM
Interesting point Hammer. Thanks for posting.

byrne819
05-08-2012, 11:20 AM
Can anyone speculate on the timing between now and potential FDA approval? I'm assuming final results will be released in July or August. When does Phase III begin? Then how long until FDA approval?

byrne819
05-08-2012, 11:20 AM
Can anyone speculate on the timing between now and potential FDA approval? I'm assuming final results will be released in July or August. When does Phase III begin? Then how long until FDA approval?

saveferris2009
05-08-2012, 11:56 AM
If I had to guess, it would be 3 years or so.

saveferris2009
05-08-2012, 11:56 AM
If I had to guess, it would be 3 years or so.

bigstar
05-08-2012, 12:22 PM
I would hope for 2 years saveferris but im too optimistic!

bigstar
05-08-2012, 12:22 PM
I would hope for 2 years saveferris but im too optimistic!

byrne819
05-08-2012, 12:55 PM
Just looked back. VX 770 started phase 3 in May 2009. If history is any lesson, that means a bit more than 2.5 years between the beginning of phase 3 and FDA approval.

byrne819
05-08-2012, 12:55 PM
Just looked back. VX 770 started phase 3 in May 2009. If history is any lesson, that means a bit more than 2.5 years between the beginning of phase 3 and FDA approval.

trw144
05-08-2012, 02:46 PM
James, Kalydeco (VX-770) achieved results of a 10.5% mean absolute improvement in its phase 3 trial, not 20% as mentioned above. This trial was also a far longer trial (48 weeks) compaerd to the recent 809/770 phase II trial. Unfortunately, the phase 2 study of VX-770 in G551 did nt use lung function increase as a marker and therefore it is not possible to compare the two trials.

Having listened to the conference call yesterday, the whole point of this announcement was that they could get the ball rolling with regulatory requirements. Vertex no have experience of this with Kalydeco and I would hope this could help reduce the time to approval (all being well). I would like to think the trial would take 18 months to complete from its start point (presumably the latter part of this year) and another 6 months for approval.

trw144
05-08-2012, 02:46 PM
James, Kalydeco (VX-770) achieved results of a 10.5% mean absolute improvement in its phase 3 trial, not 20% as mentioned above. This trial was also a far longer trial (48 weeks) compaerd to the recent 809/770 phase II trial. Unfortunately, the phase 2 study of VX-770 in G551 did nt use lung function increase as a marker and therefore it is not possible to compare the two trials.

Having listened to the conference call yesterday, the whole point of this announcement was that they could get the ball rolling with regulatory requirements. Vertex no have experience of this with Kalydeco and I would hope this could help reduce the time to approval (all being well). I would like to think the trial would take 18 months to complete from its start point (presumably the latter part of this year) and another 6 months for approval.

rainsmom
05-08-2012, 03:31 PM
the release said the sweat chloride.. not statistically significant on those using vx809 and kalydeco but then goes on to say there was a significant reduction in those that were treated with vx 809 alone. so what does that mean exactly? does the kalydeco change the way it works? that seemed confusing to me

rainsmom
05-08-2012, 03:31 PM
the release said the sweat chloride.. not statistically significant on those using vx809 and kalydeco but then goes on to say there was a significant reduction in those that were treated with vx 809 alone. so what does that mean exactly? does the kalydeco change the way it works? that seemed confusing to me

jamesB
05-09-2012, 04:48 AM
Rainsmom, on the call I think they said they were waiting for the full results to try and understand the inconsistency that you pointed out. trw144, you are spot on. We certainly can't make accurate comparisons at this stage, all we can take from this is that the results are promising enough to move forward. We are all essentially speculationg at this point in time.

jamesB
05-09-2012, 04:48 AM
Rainsmom, on the call I think they said they were waiting for the full results to try and understand the inconsistency that you pointed out. trw144, you are spot on. We certainly can't make accurate comparisons at this stage, all we can take from this is that the results are promising enough to move forward. We are all essentially speculationg at this point in time.

regina65
05-10-2012, 01:53 AM
I'll take 5 to 10 % improvement

regina65
05-10-2012, 01:53 AM
I'll take 5 to 10 % improvement

saveferris2009
05-25-2012, 02:47 AM
Interesting perspective:

<b>Vertex falls after analyst expresses doubt about cystic fibrosis drug </b>

Vertex (VRTX) is retreating after Cowen's analyst Dr. Phil Nadeau said he's not convinced that the company's VX-809+Kalydeco treatment for certain cystic fibrosis patients will be successful. The analyst believes that the improved interim lung function data from the Phase II trial of the treatment may not be duplicated in longer Phase III trials. Dr. Nadeau, - who believes that most investors now expect the treatment to be successfully developed - identifies two main reasons for his skepticism. First, each patient's interim lung function, or FEVI, can change without indicating a true change in lung function, he said. Dr. Nadeau is concerned that the study didn't adequately account for this factor. Secondly, the study didn't record a significant sweat chloride change in the patients, even though the treatment is supposed to work by improving CFTR function, which is correlated with sweat chloride, Dr. Nadeu contends. In mid-morning trading, Vertex declined 95c, or 1.49%, to $62.99. :theflyonthewall.com"

http://www.theflyonthewall.com/permalinks/entry.php/VRTXid1641918/VRTX-Vertex-falls-after-analyst-expresses-doubt-about-cystic-fibrosis-drug

saveferris2009
05-25-2012, 02:47 AM
Interesting perspective:

<b>Vertex falls after analyst expresses doubt about cystic fibrosis drug </b>

Vertex (VRTX) is retreating after Cowen's analyst Dr. Phil Nadeau said he's not convinced that the company's VX-809+Kalydeco treatment for certain cystic fibrosis patients will be successful. The analyst believes that the improved interim lung function data from the Phase II trial of the treatment may not be duplicated in longer Phase III trials. Dr. Nadeau, - who believes that most investors now expect the treatment to be successfully developed - identifies two main reasons for his skepticism. First, each patient's interim lung function, or FEVI, can change without indicating a true change in lung function, he said. Dr. Nadeau is concerned that the study didn't adequately account for this factor. Secondly, the study didn't record a significant sweat chloride change in the patients, even though the treatment is supposed to work by improving CFTR function, which is correlated with sweat chloride, Dr. Nadeu contends. In mid-morning trading, Vertex declined 95c, or 1.49%, to $62.99. :theflyonthewall.com"

http://www.theflyonthewall.com/permalinks/entry.php/VRTXid1641918/VRTX-Vertex-falls-after-analyst-expresses-doubt-about-cystic-fibrosis-drug

fly30
05-29-2012, 08:38 AM
We got a 10% increase just starting pulmozyme once daily. I thought the kalydeco treats the root of the problem ie no enzymes, no physio, a major improvement in overall health etc

I may be totally wrong......haven't read up on all that much info on kalydeco regarding ddf508

jamesB
05-29-2012, 10:01 AM
Vertex has released some corrected results from the trial, with the results not quite as good as previously thought. <a title="http://investors.vrtx.com/releasedetail.cfm?ReleaseID=677520 " href="http://investors.vrtx.com/releasedetail.cfm?ReleaseID=677520%20">http://investors.vrtx.com/releasedetail.cfm?ReleaseID=677520 </a> Vertex is down 20% premarket. - Corrected Data: Responder analysis showed 35% of patients experienced an absolute improvement in lung function (FEV1) of at least 5 percentage points and 19% had at least a 10 percentage-point improvement when treated with VX-809 and KALYDECO - - Additional Data: Patients treated with VX-809 and KALYDECO experienced an 8.5 percentage point mean absolute improvement in lung function compared to patients treated with placebo (p=0.002) - - Vertex plans to start a pivotal study of this combination to treat the underlying cause of CF in adults with two copies of the F508del mutation, pending final data and discussions with regulatory agencies -

saveferris2009
05-29-2012, 10:17 PM
@James, similar to what I posted on 5/25 above!

jamesB
05-30-2012, 04:28 AM
saveferris2009, this has definitely put the heat back on Vertex. I do find it interesting that some of the issues people have been highlighting are in this release. If it's possible, the final results are now even more hotly anticipated.