Kalydeco: Recently received FDA approval for 8 gating mutations

VX809: Phase 3 results expected mid 2014

VX661: Phase 2 F508del homozygote trial will start enrolling soon

Next gen correctors: Vertex aim to have one in trials by end of 2014


Kalydeco & R117H Trial Results:
+5.0% absolute improvement in FEV1 seen in patients aged over 18
Vertex plans to meet with the FDA in early 2014 to discuss this data and a potential FDA application

VX809 / Kalydeco:
F508del homozygote trial has completed enrollment, data expected mid 2014

Other Gating: Application submitted to FDA and EMA

R117H: Results expected this year, potential FDA application early 2014

F508del homozygotes: Phase 3 trials underway, data expected mid 2014

F508del heterozygotes: Phase 2 VX809/Kalydeco trial has commenced

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

Main Strategies:

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

G551D: New study planned with VX661 & Kalydeco with F508del/G551D patients

Other Gating: 10.7% absolute FEV1 improvement seen during phase 3 trial

F508del homozygotes: Phase 3 trial has commenced & enrollment is going well, FDA application planned for 2014

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Latest Vertex Data:
1) Heterozygotes increase to about 33% of normal function and homozygotes reach about 45% with triple combination therapy.

2) G551D/F508del heterozygotes improve from 50% to 80% when VX809 is added to Kalydeco.

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Kalydeco Monotherapy Beyond G551D:
– Results expected from other gating and R117H trials this year
– Hoping for label expansion in 2014

Phase 3 VX809/Kalydeco Trial:
– Involves F508del homozygotes
– 50% of CFs worldwide
– Encouraging phase 2 results

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