The CF Foundation and Vertex have had tremendous success in moving the Vertex drugs, VX-809 and VX-661 forward. These drugs have been developed to correct the most common mutation, Delta F508. Fifty percent of our patients will have two copies of this mutation and 90% will have one copy of this mutation.

The combination trial of VX-809 and Kalydeco, for those with two copies of this mutation, has been submitted to the FDA for new drug approval. The trial data showed a 3%-5% increase in lung function and most significantly, showed a 30%-40% decrease in lung exacerbations. We anticipate a quick review process with approval in early 2015. This could make a difference to 20,000 CF patients!

In addition, the drug combination of VX-661 and Kalydeco has moved to phase 3 trials. This drug is considered more potent than the VX-809 and, with increased dosage levels, does not have any side effects that were apparent with the VX-809 drug at those levels. Numerous phase 3 trials will begin in the first quarter of 2015 for those with one or two copies of the most common mutation.

Vertex Pharmaceuticals believes that a triple combination will be needed to ultimately correct the Delta F508 mutation. Currently they have multiple next-generation correctors in the lead-optimization stage of research and expect to begin clinical development of a next-generation corrector in 2015. In vitro data showed that a triple combination of VX-661, Kalydeco and a next generation corrector resulted in increased chloride transport in human bronchial epithelial cells with one or two copies of the Delta F508 mutation, as compared to the use of a single corrector in combination with Kalydeco.