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

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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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)

Proteostasis have identified multiple compounds that improve the folding and trafficking of F508del.

These compounds have a similar level of activity to VX809, and when added to VX809, approx 40% of normal CFTR function is reached.

Trials are planned to start in 2015.