Vertex Pharmaceuticals ($VRTX) has nailed down some promising mid-stage data for a combo treatment against the genetic lung disorder cystic fibrosis. An interim analysis of the Phase II study showed that patients on the company's drug Kalydeco and an experimental treatment had significantly improved lung function compared with those on placebo, Vertex announced.
The data indicate that Vertex might have a remedy for treating a large number of the 70,000 people worldwide with CF, and the company's share price had jumped nearly 46% to $54.69 as of 12:16 pm ET today.
There was a 5% improvement in lung function in 46% of patients on the recently approved Kalydeco and VX-809, and 30% of patients on the combo saw at least a 10% boost in lung function, according to the company. A 4%-6% improvement in lung function in the study would be a "home run," an analyst from RBC Capital Markets wrote last month, according to Bloomberg's report on the study. Yet Forbes' pharma editor Matthew Herper cautions that the study isn't complete and the combo hasn't met everyone's expectations.
Once the company wraps the Phase II study and talks to regulators, Vertex aims to race ahead with a pivotal study of the Kalydeco-VX-809 combo for treating the root cause of the genetic disease in patients with two copies of the F508del mutation. Almost half of all patients with the disease, which causes thick and sticky mucus to build up in the lungs, have two copies of the mutation, according to the company. Kalydeco, the first drug approved to combat the underlying cause of CF, has approval for treating patients with a mutation that signifies about 4% of patients with the disease, Bloomberg reported.
"People with two copies of the F508del mutation have one of the most severe forms of cystic fibrosis. In these patients, the combination of VX-809 and Kalydeco led to significant improvements in lung function that exceeded our expectations," Dr. Chris Wright, Vertex's senior vice president, global medicines development and medical affairs, stated. "We look forward to beginning discussions with regulatory agencies later this year when we have full data from the study, with the goal of moving forward with a pivotal study as quickly as possible."
The FDA has shown that the agency will take quick action on drugs that prove effective against life-threatening diseases such as CF, which leads to premature death. As Bloomberg noted, the agency stamped an approval on Kalydeco after a three-month review.
Editor's note: Updated stock price at 12:20 pm ET on 5/7/12.