Vertex Update: New data for other gating mutations and new trial for G551D

[GenH]

I've summarised the latest Vertex investor conference and added some images from the conference here: http://sixtyfiverosesblog.wordpress.com/2013/07/31/july-vertex-update/

Main points:

G551D
A new study with VX661 & Kalydeco is planned for the second half of 2013 with patients who have G551D & F508del. Data is expected in late 2013 or early 2014.

2-5 year old Gating Mutations
Part 2 is enrolling, results expected in the middle of 2014

Other Gating Mutations
Phase 3 data: Mean absolute FEV1 improvement of 7.5% from baseline & 10.7% compared to placebo at 8 weeks (both p<0.0001). Vertex plan to apply to the FDA and EMA later this year

R117H
Results expected this year, Vertex expect to apply to the FDA early next year

Residual Function
Data from the Denver study is expected in the first half of 2014

F508del homozygotes (2 copies F508del)
Phase 3 trial of VX809/Kalydeco has started and recruitment is going well. Vertex plan to report the data and submit an application to the FDA in 2014

F508del heterozygotes (1 copy F508del)
Planning to start an 8 week phase 2 study with VX809/Kalydeco later this year

Worldwide Reimbursement
Kalydeco uptake in Europe has been very strong, nearly all eligible patients in the EU are taking Kalydeco.
Kalydeco was approved by the TGA in Australia earlier this month, this is the third largest market after the US and England, with 200 patients aged over 6 with G551D. Canada has 100 G551D patients aged over 6. Kalydeco is not currently reimbursed in Australia or Canada.

There is more info in my link above.

 

[GenH]

That comment is straight from the transcript of the conference, referring to G551D and Kalydeco.

 

[GenH]

"Over the first half of this year, we have successfully completed reimbursement discussions in most of the major EU markets, including England, Scotland, Ireland, Northern Ireland and Wales, and made KALYDECO available for the many patients who need it. Since then, we have seen a similar pattern of rapid uptake of KALYDECO as we saw in the U.S. last year. I'm proud of the incredibly effective efforts of our field organization, which, together with the dedicated health care providers who care for CF patients, have enabled us to begin treating nearly all the eligible G551D patients in the EU."

http://seekingalpha.com/article/158...-2013-results-earnings-call-transcript?page=1

 

[bigstar]

Im sorry GenH i totally misunderstood. Im deleting my comment!

 

[triples15]

Thanks as always Gen! I appreciate you keeping me up to speed.

I've said it before and I'll say it again, I'm SO anxious to get my hands on Kalydeco it's ridiculous... Can't wait for the results from the n of 1 trial in Denver. I'm just doing everything I possibly can to stay healthy for if/when the label gets expanded for those of us with residual function mutations!!