Vertex Conference Live Now--triple combo gets up to 75% function

[Aboveallislove]

Just to clarify if I was confusing so I'm not spreading misinformation:
Michelle: The 661 study I link to above is only for the combo 661/Kalydeco. Vertex doesn't think it will work for heteros but is giving it a shot. It is called a "futility" study, where they are doing a smaller number to see if there is hope it will work and if so will then fully recruit. The near 50% improvement comes from 661/Kalydeco/+a new corrector. That will be in healthy people in November and in CFers with the 3 drug combo in second half of 2016. Right now Vertex has 2 new correctors it is testing but also said it will have more to next year. So there are many they are looking at.

Heather: The 3 drug combo doesn't work on stop mutations. But it works so well on df508 that it gets enough improvement alone.

Hugs dear moms. Big momma hugs and prayers.
Love

 

[Gammaw]

Here's the link to Vertex's presentation, including the oral presentation to go along with the slides. It is under Investor Event at the NACFC on October 9, 2015. They do see to say that the triple combo inludes all d508F heterozygotes. In other words, it includes anyone who has at least one D508F mutation. Of course it also includes those on Kalydeco and Orkambi. It appears to not yet include approximately 10% who have nonsense mutations on BOTH alleles. Thanks AboveAll for plugging us into this . . . .

http://investors.vrtx.com/events.cfm?nav=media

 

[Aboveallislove]

Hey Gammaw,
Just to clarify: What do you mean whey they say the triple combo includes all df508 heterozygotes? What I had understood was that the goal is for the triple to be used for all df508 heterozygotes and Kalydeco for those with 551 or gating or residual function but not df508 on the other allele. BUT the results they showed were for df508 heterygotes AND those with minimal/non-response mutations on the other allele. So the 50%ish improvement does not include those who benefit from Kalydeco and Orkambi. See slide 11 at the top right where it shows the two genes tissues tested. Am I missing something??

PS To explain what I'm saying further: The context of the triple for all with df508 came in the context of explaining Vertex now thinking a different approach is appropriate; previously, Vertex saw multiple drugs for different mutations and now sees the triple for all with at least 1 df508 and Kalydeco the gating/residual function but no df508.

 

[Gammaw]

Yes, AboveAll. It appears we're saying the same thing. You are a DF508 heterozygote if you have one copy of DF508. So, if you have one copy of DF508, regardless of your other mutation, you are apparently included in the target population for a triple combo.