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These Mutated Cystic Fibrosis Cells are Worth Billions to Vertex Pharmaceuticals (MA) (VRTX)


6/14/2013 8:04:13 AM

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This is my favorite data slide from Vertex Pharmaceuticals' (VRTX_) presentation at the European Cystic Fibrosis Society meeting. The slide is important because it helps explain how Vertex will dominate cystic fibrosis therapy and one day generate $10 billion in annual sales. You don't need to be an expert in cystic fibrosis to understand this slide and why it's bullish for Vertex. Let's go through it. The data on the left (with the orange bars) represents "heterozygous" cells with two different cystic fibrosis mutations -- F508del and G542X. Think of these cells as a proxy for about 20,000 "heterozygous" cystic fibrosis patients worldwide who are among the most difficult to treat. Treatment with single Vertex drugs -- Kalydeco (ivacaftor) or VX-809 ( lumacaftor) -- has minimal activity in these heterozygous cells. Doublet therapy -- using Kalydeco and VX-809 together -- enhances activity a bit. But check out what happens when you add a third "corrector" drug into the mix: The orange bar gets really tall. A tall bar in this graph is good because it means cells are functioning properly. In cystic fibrosis patients, functioning cells translates into vastly improved lung function.

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