Why Is Vertex Pharmaceuticals Up More Than 50 Percent?

Early Tuesday morning, Vertex Pharmaceuticals VRTX issued a press release announcing that its two phase 3 studies of Lumacaftor in combination with ivacaftor met the primary endpoint with "statistically significant improvement in lung function."

Shares of Vertex have skyrocketed over 53 percent in the pre-market following the press release and were trading over $101, at last check.

The combination treatment of Lumacaftor and Ivacaftor is designed to treat individuals suffering from the most common form of Cystic Fibrosis.

The studies showed that patients who received the combination treat saw reductions in pulmonary exacerbation of 30 and 39 percent, as well as an absolute improvement compared to the placebo in the range of 2.6 to 4.0 percentage points.

“These data showed consistent evidence of clinical benefit in lung function and other measures of the disease. The significant improvements in pulmonary exacerbations are particularly important given the potential for these events to result in hospitalizations, permanent lung damage and the need for additional treatment with antibiotics and other medicines,” said Bonnie Ramsey, M.D., Professor of Pediatrics, University of Washington School of Medicine, Director of the Center for Clinical and Translational Research at Seattle Children's Research Institute and a lead Principal Investigator for TRANSPORT.

Based on the results of the studies, Vertex will submits applications for approval in multiple countries including the U.S. and Europe.

“The combination of lumacaftor and ivacaftor is the first regimen designed to address the underlying cause of CF for people with the most common form of the disease, and based on these data, we plan to move as fast as possible to submit applications for approval of this combination regimen in countries around the world,” said Jeffrey Chodakewitz, M.D., Senior Vice President and Chief Medical Officer at Vertex.