and it starts to decline from there and there are side effects. After the treatment stops the patient goes back to zero but the side effects such as liver enzymes start to improve.
you are right, the three drug combo will not be Kalydeco + 809 + 661, but a combo of Kal + 809 + X or Kal + 661 + X. Most likely it will be the 661 containing cocktail. X - referred to as second generation corrector -had been promised to be in the clinic by late 2014, but that has been postponed to sometime in 2015. I guess VRTX wants to see more results on 661 before making the final selection of X.
Have you been following UBIQ. Looks like it is about to breakout and make a move higher. Check out the chart Don’t miss out on UBIQ. From my research
As far back as I can recall Vertex or the Cystic Fibrosis Foundation has said that the problem isn't solvable with a single drug, at least for double deltas. More recently, though I think this has been known or promulgated for some time as well, Vertex or the CFF have shown data that suggests a three drug combination is likely the answer. Seems like there have been multiple slides over the years that point to this combination as having a synergistic impact. That a 4% improvement from 809 and a 4% improvement from 661 (obviously in combination with 771) won't amount to an 8% improvement in lung function, but something much more. That said, I don't know that I've ever seen anything that suggests that the three drug cocktail that I've heard so much about for so long is anticipated to consist of 771, 809 and 661. Has anyone seen this information published or discussed by someone in a position to know?
I should clarify though that the pool will be larger with 661 because it will with kalydeco get enough benefit to bring residual function onto the label....kalydeco shows improvement along for them, but widely rather than doing phase three for kalydeco with residual function vertex is doing 661 combo for them and that will definitely be enough for that new group.
No. To reach heteros the improvement for homozygotes would have to be enough to infer it will improve enough for those with only one copy of the gene. Half of six being three the market likely saw six as the only number that was likliy to get to heteros with 661 kalydeco combo. It might still....but it is definitely less likely and that means the pool won't increase until a three drug combo comes out....it is still good news because you need 661 for a three drug combo and the safety and timing means you'll have the 661 combo ready to add with next generatin corrector.
" I think the market is seeing the Phase 3 of 661 as only beneficial if it will be able to reach heterozygotes, "
Why 6% and not 4% improvement?
I thought the main reason for 661 was to increase the pool and if 661 showed a bit over 4% improvement doesn't that increase the pool nevertheless?
as i predicted. and again, this ist bad....661 is the gateway to three drug combo!
IF VX 661/770 shows a 6% improvement in absolute FEV-1 treating twenty CF 508 homozygotes for twelve weeks, the market is going to tell you what insurance companies are likely to pay for such an improvement, (along with the likely associated reduction in hospitalizations and pulmonary infections, improved body weight and nutritional status, lengthened life expectancy and improvement in quality of life in the majority of CF patients), with the movement up in VRTX share price after the data is released, We'll soon find out the answer in the next few days. Good luck to all longs and to the CF community!
same people who are willing to pay for 6" of improvement. Have you every watch " Cialis" ad before the nightly news? Something is good about the word " six".
For the record...I didn't use profanity...I used the word that is a ghost that starts with sp...I've learned already not to abbreviate homozygotes as the gets deleted too!
No, but I think the market correctly believes you need at least 6% with dd508f for it to get 3% for ddf508. But I think the market will wrongly think that it is bad if the improvement isn't enough to get to heteros now. It would be amazing if they couldn', but still good to have 661 fast tracked.
For 661 phase 2, did VRTX indicate what % FEV improvement it would consider a success? Did the study have an end point guideline? Did they mention the 6% as an expectation?