Vertex Will Go Ahead with Promising New Cystic Fibrosis Drug Trials (VRTX)

Vertex Pharmaceuticals Inc (NASDAQ: VRTX) released new clinical findings that show how promising a new cystic fibrosis treatment could be.  The approach combats the condition, improving lung function by significant measure and improving efficacy of other treatments currently used by 68,000 people globally; nearly half of whom seem to have no response to existing medicines.

The promising results has encouraged the Boston-based drug company to announce plans to launch at least one—maybe two—clinical trials for the biotech firm’s three drug combination early next year.

Three early- and mid-stage studies showed that Vertex (NASDAQ: VRTX) has a “triple combo” of medicines which seemed to work in those patients who have been the hardest to treat.

Dr. Steve Rowe is the director of the cystic fibrosis research program at the University of Alabama Birmingham Medical Center. He contends, “The magnitude of improvement we’re seeing is marked.”  Dr. Rowe helped to run these initial trials for Vertex, at this hospital. He goes on to say, “This is really a great example of precision medicine coming into fruition.  The results were impressive and conclusive.”

As to be expected, investors also said, on Tuesday, they are very excited about the prospect of a new—more effective—treatment for cystic fibrosis. In fact, they contend that this could eventually help as many as 90 percent of all cystic fibrosis patients.  This is evident, of course, in the fact that shares boomed 25 percent in after market trading, late on Tuesday. The stock has already climbed more than 75 percent since the beginning of this year.

Vertex (NASDAQ: VRTX) employees roughly 1,400 people in the Massachusetts area and another 2,100 across the globe.  The company is the only drug maker to develop a medical treatment for the underlying genetic cause of cystic fibrosis.  These drugs are the widely successful Kalydeco and Orkambi, which treat the genetic defects that result in cystic fibrosis for roughly 30,000 patients around the world.  Still, that is only about 40 percent of all cystic fibrosis patients in the world.

Cystic fibrosis is a genetic disorder which eventually causes mucus to block air passages in the lungs.  Approximately half of all those who are born with this disease do not live past the age of 38, according to the Cystic Fibrosis Foundation.

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