Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) announced positive results on Wednesday from a late-stage STRIVE study of its oral drug VX-770 for treatment of cystic fibrosis, a genetic disease that impairs the lung.
The positive results was seen in trading of Vertex stock that had reached a new high for third day in a row. The shares of the drugmaker touched a new 52-week high of $46.75 on Monday.
The Cambridge, Massachusetts-based Vertex said the study showed the drug to notably improve lung function, compared with placebo. The company intends to submit U.S. and European regulatory applications for approval in the second half of 2011.
The drugmaker said profound improvements in lung function were observed through week 24, and sustained through week 48, among those who received the drug compared to those treated with placebo. In all key secondary endpoints, significant improvements were also observed through week 48 among those who received VX-770.
Treating the underlying cause of cystic fibrosis with VX-770 led to clinical improvements that were far beyond our expectations, providing support for an entirely new approach to the treatment of this disease, said Peter Mueller, Vertex chief scientific officer.
Cystic fibrosis or CF is a life-threatening genetic disease affecting about 30,000 people in the U.S. and 70,000 people worldwide. It is caused by defective or missing CFTR (cystic fibrosis transmembrane conductance regulator) proteins that affect ion flow in cell membranes, and the accumulation of thick mucus that leads to chronic lung infections and damage.
The STRIVE study assessed people with a mutation in the CF gene known as G551D. The study enrolled 161 people, who received at least one dose of either VX-770 as a single 150 mg tablet or placebo twice daily.
Vertex said it is also conducting a Phase 2a clinical trial to evaluate multiple combination regimens of VX-770 and VX-809 in people with two copies of the F508del mutation, which prevents the CFTR protein from moving to its proper location at the cell surface. Vertex said it anticipates to obtain data from Part One of the trial in the first half of 2011.
Vertex stock gapped open sharply higher Feb. 23 at $43.60, compared to previous day close of $38.22. The stock touched a new 52-week high of $44.68 during the Wednesday's trading session on the highest average volume of the year of 16.57 million shares.
On Feb. 25, the stock surpassed Wednesday's high to reach a new 52-week high of $44.98. The stock closed Friday's regular trading up 2.89 percent at $44.84 with a volume of 2.38 million shares.
But on Feb. 28, the stock surpassed Friday's high to reach a new 52-week high of $46.75. The stock closed Monday's regular trading up 4.08 percent at $46.67 with a volume of 3.28 million shares on the NASDAQ stock market. The stock traded between $31.25 and $46.75 during the past 52-week.