September 11, 2013

September Vertex Update

10/9/13 Vertex Investor Presentation

Vertex are trying to treat as many patients as possible & enhance the benefit for those already on Kalydeco.

3 Main Strategies
1. Kalydeco Monotherapy
2. Combination Therapy (one corrector with Kalydeco)
3. Triple Combination Therapy (two correctors with Kalydeco)

A small number of patients are not reached with these three strategies, the scientists at Vertex are ‘trying to figure that out.’

1. Kalydeco Monotherapy
Currently treating about 2000 G551D patients: 1000 in Europe and 1000 in US
Another 500-700 have other gating mutations
This means 2500-3000 overall with G551D & other gating mutations

Residual Function: Currently running multiple studies (including R117H & N of 1)
R117H study data expected at the end of this year/beginning of next year
R117H is the most common residual function mutation, approx 4% of the CF population
N of 1 study involves people from a group of 1500-1800 rare mutations

Aim to treat 10-15% of worldwide patients (those who have gating/residual function mutations)
This is 7000-10000 of 70,000 patients worldwide

2. Combination Therapy
Two phase 3 VX809 / Kalydeco trials underway with F508del homozygotes (2 copies F508del)
Recruitment going well, expect to complete enrollment by end of this year
Data collection & review planned for 2014
Worldwide there are 25,000- 30,000 homozygous F508del patients

3. Triple Combination Therapy
Vertex hope to have a second generation corrector in trials by the end of 2014

Enhancing benefit for those on Kalydeco
Approx 80% of the 2000 patients on Kalydeco have F508del as their second gene
Vertex plan to commence a trial with VX661 & Kalydeco with these patients
Aim to enhance pulmonary function and decrease exacerbations

Vertex Correctors
VX809: lead corrector, currently in phase 3 trials
VX661: 1 year to 18 months behind VX809, has less interaction with other medications, could be used in triple combination therapy
VX983: Trial with healthy patients complete, 28 day study with Kalydeco planned, currently in discussions with regulatory authorities

Source: September Vertex Investor Presentation (no longer available online)

Trial Updates:

Several Vertex trials have been updated recently on

1. Cohort 4 added to the phase 2 VX809 / Kalydeco trial:
Involves F508del heterozygotes (1 copy F508del)
with 400mg of VX809 twice daily & 250mg of Kalydeco twice daily for 8 weeks.

2. Group 7 added to the phase 2 VX661/Kalydeco trial:
Involves F508del heterozygotes
. This trial involves 100mg of VX661 once daily for 28 days. I believe this may be the trial for those already on Kalydeco with G551D/F508del.

3. Rollover study announced for those in the two phase 3 F508del homozygote trials & also for heterozygotes in cohort 4 of the phase 2 VX809/Kalydeco trial. Collecting data until May 2017.


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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 - Second Generation Correctors, 3. Vertex - VX809 & VX661