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: 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

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

A recent study suggested that VX809 helps the F508del mutation by improving several interactions between regions in the CFTR protein.

This study suggested that VX809 interacts with membrane spanding domain one and can help several other CF mutations.

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ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. ProQR are targeting the Cystic Fibrosis defect at the RNA level

This is currently in the pre-clinical stage.

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