February 28, 2013

February Vertex Update

Update from the 26/2/2013 Vertex Investor Presentation:

Current Trials:
– Enrolment is complete in the other gating Kalydeco trial
– Enrolment is ongoing in the R117H and residual function Kalydeco trials
– Data from both is expected later this year
– Dosing has commenced with the G551D 2-5 year old Kalydeco trial
– Worldwide the above groups are 10-15% of the CF population (slightly higher in Aus as G551D is 8%, R117H is 2.5-3%, other gating is about 1% and residual function could be another 5-10%)
– Data expected for VX661 by June

Phase 3 VX809/Kalydeco trial for people with 2 F508del:
– There are more than 30,000 double F508del patients worldwide
– In terms of the phase 3 study design that was announced yesterday: The two 24 week trials have the same design, each with 500 patients and 3 groups (600mg of VX809 once a day plus Kalydeco, 400mg of VX809 twice a day plus Kalydeco and placebo). After the trial there is a 96 week open label trial where everyone gets VX809/Kalydeco. Patients need to be aged 12 years or older, have two copies of F508del and an FEV1 between 40-90%. The tablets will contain both VX809 and Kalydeco. The trial will be held at 200 sites in the US, Australia and Europe.
– The primary endpoint will be relative FEV1% improvement compared to placebo. They are hoping for at least a 5% relative improvement.
– Depending on the results, it is planned to submit to the EMA and FDA following these trials. Data is expected in 2014.

Other future trials:
– The 6-11 year old 24 week study with patients who have two copies of F508del will look at safety and dosage. No time frame was mentioned. This will also have an open label period following the trial.
– No time frame was mentioned for the 8 week phase 2 study for people with one copy of F508del. This is looking at safety and lung function. They are accelerating the second generation correctors to help the people with one copy of F508del.

Results from the phase 2 VX809/Kalydeco trial for people with 2 copies of F508del:

– There was a statistically significant improvement from baseline and placebo at day 56 with the 600mg group (cohort 2). The patients who had higher levels of VX809 in their blood stream (higher exposure) had a better clinical response. There was a 3.4% absolute FEV1 improvement compared to baseline and a 6.7% improvement compared to placebo at day 56.

– The 400mg twice a day group (cohort 3) had better levels of VX809 in their blood stream. 70% of these patients were in the highest 25% in terms of the levels of VX809 in their blood stream (see the second image). 40% of the 600mg group were in the highest 25%. The means for both groups was in the top 25%. This demonstrated a relationship between levels in the blood stream and an improvement in FEV1. There was a 1.9% absolute FEV1 improvement compared to baseline at day 56 (not statistically significant). This trial was very small with only 11 patients so it was not designed to look for FEV1 improvement (designed for safety and dosage).

Sources: http://investors.vrtx.com/releasedetail.cfm?ReleaseID=743425
Feb 2013 investor presentation: http://investors.vrtx.com/eventdetail.cfm?eventid=126065


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1. Class 2 Mutations & F508del, 1. Class 3 Gating Mutations & G551D, 1. Residual Function Mutations & R117H, 3. Vertex - Kalydeco, 3. Vertex - VX809 & VX661