If you haven't read the transcript, especially CFers and family, DO. It is amazingly promising. Following are some highlights of NEW info (albeit some tied to OLD info), but you really want to read the entire transcript.
1) The cohort 3 Part 2 of 809/770 testing of higher dosing of 809 (400 2x) is based on the fact that level safety/effectiveness is supported by "molecular modeling" And that Cohort 3 is "to support the data we'd already gathered from Phase I and Phase II with our combination therapy and into PK/PD modeling exercise and that data in Cohort 3 will supplement the data we've already collected." Vertex indicated that cohort 3 was not "powered" to give FEV or SC info and that it will not release results separately but that the goal is to use this info to support a higher dosing in Phase 3. And the design of Phase 3, when announced, will be the implicit answer to what Cohort 3 did. Vertex added that the "higher dose," might not be 400 2x a day.
2) Expect 809/770 to start in early 2013. Meeting with FDA in next quarter. Expect a 6 - 12 month study; FEV primary endpoint; 600 mg dosing (possibly also higher--see above); other endpoints including weight gain, reduction in pulmonary exacerbations and CFQ-R.
3) 661: Discussed study design which is dose escalation. They expect results in first half o 2013 and will not announce early b/c they want to complete "all of those ascending doses." Specifically said that they are moving with 809 SAYS NOTHING re belief about 661. Vertex wants to get 809 to CFers NOW and not wait two more years on 661. Also noted in looking at results "661 has a different biodistribution, and so you cannot basically collet data from 809 and basically sort of refer to it." Vertex added "you can come with substantially lower doses, and that's basically wat we are trying to do to have basically a low proof of burden, a low dose burden and that allows us to do better combination studies in the future, as manny different second-generation correctorws or KALYDECO or anything in the mix." Also stressed 661 is strategy for heterozygotes.
4) Second-generation correctors: Noted that i"n last several months made progress in identifying second gneration correcotrs that show significant additive or synergistic activity in vitro when combined with VX-809 and Kalydeco." Here what was intersring is that Vertex thinks it will be a "mix-and-match" with some needing 770 and 809 and 1 of these new correctors, and in other cases maybe 2 correctors. This depends on heterozygotes, homozygoes and pancreatic insufficient, etc.
5) Phase 2 "residual CFTR function" study which hasn't gotten much focus. I've written on this before and noted that this really seems to have alot of potential to capture "class of 1." Vertex was asked how to define residual exocrine pancreatic function (which is one--disjunctive of three--inclusion criteria) and Vertex said "those who can manageage without" "pancreatic enzyme replacement. . . . That's how we definite it." NOTE: Some ddf508 are actually pancreatic sufficient. FEW, RARE, but SOME.
In the end, there is amazing hope right around the corner.