Cystic Fibrosis & Ataluren About 10% of CF patients have a nonsense mutation Current treatments do not address the underlying cause of nonsense mutation CF Previous phase 3 trial results Patients on Ataluren were stable after a year, this stability continued in the extension study (see graphs below) These previous results were recently published in […]

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 […]

Approx 20% of the worldwide G551D patients are still waiting for Kalydeco in at least 13 countries.

Some of these countries cannot afford basic CF medications like Pulmozyme, so how are they going to afford $300,000+ per year per patient?

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10% of CF patients have a nonsense mutation. Ataluren has been trialled with nonsense mutation CF.

Previous phase 3 trial results (no TOBI):
+ 5.7% relative improvement in FEV1 compared to placebo
41% reduction in pulmonary exacerbations

New phase 3 trial is planned to start in the first half of 2014. Data is expected mid 2016.

Kalydeco recently received FDA approval for eight gating mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. G970R was not included.

The phase 3 gating trial showed a mean decrease in sweat chloride of -49 mmol/L & a mean increase of 10.7% FEV1. Detailed results are included in the post.

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