Summary of the 1/5/14 Vertex Investor Press Release & Conference Kalydeco Growth “In 2014, further growth and achievement of the companys total 2014 net product revenue guidance for KALYDECO is dependent on completion of reimbursement discussions in Australia and Canada for eligible patients with the G551D mutation and on the potential further expansion of the […]

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

Galapagos are developing new potentiators that have a superior effect on CFTR opening than Kalydeco. The superior opening was seen with class 2, 3 and 4 mutations. It is planned to start a trial by the end of 2014.

New correctors are also being developed in colloboration with AbbVie. It is planned to have a corrector in trials by the end of 2015.

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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