Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX), a biotechnology company engaged in the discovery, development, manufacturing and commercialization of small molecule drugs for the treatment of serious diseases, on Monday, reported that the new cystic fibrosis drug Kalydeco when combined with its experimental treatment for the disease led to substantially improved breathing ability in a mid-stage study.
VRTX shares surged following the announcement of the encouraging clinical data. The stock rose to an intra-day high of $58.75 before finishing the day 55.36% higher at $58.12 on above average volume of 40.22 million.
Dr. Joseph Pilewski, who is an adult cystic fibrosis physician at the University of Pittsburg Medical Center, said that this is very exciting data. Dr. Pilewski said that if this were to bear out in a Phase 3 pivotal trial it will dramatically impact the treatment options out there for patients with cystic fibrosis.
The data indicates that Vertex could have a multibillion dollar franchise in cystic fibrosis.
The study looked at 37 patients, who underwent 56 days of treatment with the Kalydeco/VX-809 combination and 11 patients, who underwent a placebo. The study found a statistically significant lung function improvement for those on the study drugs.
Chris Wright, M.D., Ph. D., Senior Vice President of Global Medicines Development and Medical Affairs at Vertex Pharmaceuticals, said that for the past 14 years, VRTX has been focused on learning about the underlying cause of cystic fibrosis so it can develop new medicines to help as many patients as possible and today VRTX believes that it is one step closer to this goal. Wright said that VRTX looks forward to starting discussions with regulatory agencies later this year when the company full data from the study, with the goal of moving forward with a pivotal study as quickly as possible.
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