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Vertex Shares Soar on Renewed Hope for Cystic Fibrosis Patients

By Sean Williams - Apr 19, 2013 at 2:10PM

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A phase 2 trial delivers significant improvement in lung function for patients with a specific type of cystic fibrosis.

Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes -- just in case they're material to our investing thesis.

What: Shares of Vertex Pharmaceuticals (VRTX 0.88%) -- a biopharmaceutical company with a current focus on hepatitis-C and cystic fibrosis treatments -- skyrocketed as much as 62% after announcing positive mid-stage data for its combination of VX-661 and its FDA-approved Kalydeco for the treatment of cystic fibrosis in patients with the F508del mutation.

So what: This particular trial is being tested on CF patients who express two copies of the F508del mutation. The results, released after the bell yesterday, demonstrated that the two highest dose groups, out of four being tested, experienced an FEV-1 improvement (a measure of how much air a person can exhale out of their lungs in the first second) of 9% and 7.5%, respectively. The placebo improved this function by a mere 3%. That is a statistically huge difference! Vertex notes that many of the adverse events seen were mild to moderate in nature and the drug combination was well-tolerated.

Now what: With so few drugs currently designed to treat CF, this would mark a gigantic step in improving patient quality of life -- assuming these results continue to translate to a larger patient pool. Luckily for Vertex, almost half of all CF patients express two copies of the F508del mutation, so it will have a pretty broad treatment group to work with if eventually approved by the Food and Drug Administration. The pop might be a bit concerning to some -- 60% is a big gain on a mid-stage trial for a multibillion-dollar company -- but considering that its future revenue stream likely lies in CF research I can, as a long-term viewing investor, support today's move higher.

Craving more input? Start by adding Vertex Pharmaceuticals to your free and personalized watchlist so you can keep up on the latest news with the company.

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