Thomson Reuters came out with a list of the top ten most promising drugs for 2015 in terms of potential sales and the Lumacaftor and Ivacaftor combo comes in at number 5.
Well the 809 combo is a start and to keep them healthy until more and better until they splice that nasty little mutation out and put in a good gene! gGodspeed
809 won't be used for the triple because it interacts with kalydeco; that's why the 661 results are key because they have 2 that work, work well and don't interact. Vertex is the farthest ahead with another corrector/modulator (think they use modulator because it doesn't correct the same thing but makes the protein more stable, i.e., it modulates it). But there are several other companies working on things (CFF doesn't list those it doesn't fund: Calista, Galapagos, Genzyme, N30, Pfizer, proteostasis, PTC are all working to fix different issues with CFTR function. And then there is the gene therapy, etc. Vertex, though, is the best hope in the short term. Is your DS ddf508 or do you have longer to wait? Prayers for all.
Got it. Well then maybe either of the 809 or 661 combos with one of the other "modulators" in development. Nothing outside the Vertex drugs on the CF website specifically uses the word "corrector" so I'm not sure if they are potentiators or correctors, though I do see that QBW151 is listed as a potentiator. Thanks for the information. It's much appreciated.
Don't forget the more than 3000 with residual function mutations that are not included in kalydeco or 809 kalydeco label but who will be under 661 trial. Not the large hetero group but still more progress.
Hey dad, no it won't be 661 and 809... They work the same way and do the same thing and you couldn't get them both because they'll be combined with kalydeco and I'm sure once 661 is on market 809 will be pulled. But this is great because 661 doesn't interact negatively with kalydeco so the three drug combo is more likely to succeed and this will speed 661 to fda approval which is needed before getting to the three drug.
Huh, a ways off I know, but I wonder if, assuming no adverse interaction between drugs, doctors might prescribe both 809 and 661 once these two drugs are approved, assuming approval. My hope as a dad is that is what happens and there is a synergistic effect.
Some of these idiots and their posts about costs. You have no idea about costs until the first time you walk out of the pharmacy with $7,000 worth of antibiotics or you leave the hospital after a 14 day stay. Between enzymes, hypertonic saline, pulmozyme, Tobi, Vanco, and all the various machines, people have no flippin clue. Thank god for insurance, the CF foundation and Vertex.
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!