Vertex Pharmaceuticals Earnings Call Nuggets: Cohort Recruitment and KALYDECO

Vertex Pharmaceuticals (NASDAQ:VRTX) recently reported its fourth quarter earnings and discussed the following topics in its earnings conference call.

Cohort Recruitment

Geoffrey Porges – Sanford Bernstein LLC: So, just a follow-up on Peter’s comments about the CF combination programs. Could you confirm that the 400 milligram BID information that’s been provided to the FDA and you are just waiting for them to come back before starting that Phase 3 and by implication that you’ve applied to add that to study? Then secondly on 661, you mentioned the cohorts Peter. Could you give us a little bit more color on where you are in the recruitment of the cohorts? Specifically is the first 120 patients being recruited and being studied out to the, I think, it’s 28 days of the end of treatment, so you are starting to get that PK/PD data in now?

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Robert Kauffman – SVP and Chief Medical Officer: So Jeff, this is Bob. I will take the first part of the question and if you remember the Cohort 3 was designed really to provide PK/PD data to fit into our PK/PD model as well as safety data at the dose level of 400 milligrams BID. Data have been provided to the FDA along with our plan for the Phase 3 program and yes, we’re in active discussions with them at this point to finalize their program and obviously we’ll provide further feedback once we get that information.

Geoffrey Porges – Sanford Bernstein LLC: And 661?

Robert Kauffman – SVP and Chief Medical Officer: With respect to 661, yes the study is well along and recruited and we’re waiting for the data to come in and as we said before, we’ll be providing that for that study in the first half of the year.


Geoffrey Meacham – JPMorgan: For the KALYDECO, the ongoing mild therapy trial, can you give us any updates you have on the enrollment status for the different studies and then maybe speak for the review timeline given your interpretation without the grade 3 designation? I had one follow-up?

Robert Kauffman – SVP and Chief Medical Officer: I’ll maybe take the last part first. Yes, obviously we’re – we have grade 2 designation, although we’re working with the FDA now to determine exactly what impact that will have on those programs and with respect to the review time, I can’t really give any further update at this point. Those discussions are ongoing. The studies are moving on very well, particularly the mutations, other gating mutations aside from G551D is fully enrolled and moving along R117H doing very well as well and the (NA1) study again also doing very well. So no issues with recruitment.

Geoffrey Meacham – JPMorgan: Then for the KALYDECO commercially in Europe I don’t know if it’s possible to get a little bit more granular with kind of what you think the pace of reimbursement discussions will be and that will be helpful, but maybe if you could also address other regions such as Australia that maybe offer similar to the NICE review cycle and may be any other sort of gaining factors that you feel like when it comes to European reimbursement that, if you could get some clarity on in the next quarter or so?

Stuart A. Arbuckle – EVP and CCO: I have to say we are pleased with the speed with which the discussions have gone and frankly I am delighted that we’ve already been able to come to satisfactory and positive conclusion with England and as we said we are expecting that to really pick up from Q2 as you’ll know in Scotland they created a special orphan drug fund and have said that old patients with G551D mutation in Scotland will be eligible for that fund which begins in March and all I can really tell you about the other discussions is that, they are very active, they are ongoing, they are very productive, nobody has any doubts about the clinical benefit that KALYDECO brings and we are working as quickly as we can to bring them to a successful conclusion across the major markets in Europe. In terms of Australia we filed and we are hopeful that we’ll see an approval, but the product this year as you know the process reimbursement in Australia is as rigorous and lengthy as anywhere else in the rest of the world and I wouldn’t necessarily be optimistic that we’ll be able to conclude that within 2013.

Ian F. Smith – EVP and CFO: Jeff, let me just end the Stuart’s comments with. In my prepared remarks how this translates to revenue expectations in 2013, I do want to reiterate that we are anticipating Q1 the KALYDECO revenues to be similar to that of Q4 2012, and based on Stuart’s outlining of the timing of approval for reimbursements in these markets and specifically those four major markets in Europe we would anticipate growth starting to be seen in the second quarter of 2013.

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