The following are some excerpts I found on yahoo's Vertex message board by someone who posted after listening to the investor conferences. Includes some additional insights on Vertx's plans for moving forward:
Post 1
2) Basically asked, "what's wrong with 809, that you are also doing 661." Vertex noted that it makes sense to go forward with two drugs because of the value for the expanded market, but also that CFF is funding entire research of 661 so in a sense a "feebie" (my word.) When asked the difference, Vertex noted basically the same with the only difference being that 661 has better tissue distribution in the lungs. (I noted 3 differences--which all may be related--between 661/809 in earlier posts). But just not sure if that will translate to better clinical results.
3) Stressed safety and SC is again factor for moving to Phase 3 and if safe and significant SC reduction (wish someone would ask what that means--just statistically significant or something more?), they would go to Phase 3.
4) Asked whether there was a path forward under both these scenerios: a) Overall average benefit to all; or b) A sub-set have a significant benefit and others none. Vertex said yes, path forward to approval under either scernerio and other drugs have been approved when only a sub-set benefits from the drugs.
5) Today mentioned 6 or 12 months for phase 3 longer-term; yesterday spoke of 12 months.
6) Again stressed that FEV might no longer be the controlling clinical improvement needed for approval given these are first drugs that treat underlying defect--mentioned weight, excerbations, quality of life, sc, as other potential clinical outcomes.
Post 2
3. After this and the last conference, I am more and more optimistic that 809/770 will go to Phase 3: After listening to the conference I said to dh with happy tears, this is going to Phase 3. The tone was very different and Vertex stressed that the issue is safety and sc and not FEV1--which they think might take as long as a year to show improvement. Re SC, Vertex noted that in Phase 2a, several participants had substantial reduction in SC and Vertex hopes that with higher dosing and longer time more participants will show higher reductions. And if safe, they will move to Phase 3, whether a reduction in FEV1 or not--a reduction would be great, but not expecting one b/c it might take up to a year to see such an improvement. This conference was the first such affirmative comment re moving to Phase 3. Vertex also noted that it would be in discussion with the FDA concerning what is needed to show clinical improvement and the inference was that Vertex thinks SC should be enough because they are altering the modulation and that is what SC measures. (As an aside, interestingly, at least to me, Vertex noted that it knew the approval of 770 was coming early.) Vertex also noted that it expects Phase 3 to be a year-long study. The presenter pushed Vertex on FEV saying the FDA might require a reduction in FEV for approval and if so, what reduction would be needed for approval. Vertex noted that in the last year Europe approved a drug with less than a 5% reduction.
Post 3
Was only listening with half-an-ear, but did find one new tidbit of interest. Vertex noted that the most frequent question it was getting was what results were needed for combo to move forward. Vertex explained that primary end points were SC and safety and that it might take as much as 6 months for FEV improvement to show, but that if they could see improvement in SC that would show they were addressing the underlying defect and support further testing even if no significant improvement in FEV. Didn't give a specific SC level of improvement, but those comments lead me to be more optimistic re the 809/770 combo moving to Phase 3 because 770's FEV improvement was pretty immediate, yet Vertex seemed open to seeing if a longer time frame was needed to see FEV1 improvement with combo, so long as there is an improvement in SC. How much of an improvement is up for debate: I would predict if Vertex can get a 30 point improvement in SC, it will move forward to Phase 3. A 60 point improvement translated into 10-15% in FEV for 770, so maybe 30 will be a 5 -8 improvement if given time.
