Vertex Pharmaceuticals has some good news this morning on its experimental drug for cystic fibrosis. The treatment, taken an oral pill, helped patients breathe better for 28 days, at both a high and low dose, than did a placebo. The drug didn’t appear to cause any serious side effects.
The findings for VX-770 were from a mid-stage study of 19 cystic fibrosis patients with a specific, and rare, mutation called G551D. The results confirm an earlier look at the data after 14 days, which Cambridge, MA-based Vertex (NASDAQ: VRTX) first announced in March, and which we highlighted again in August. Now that researchers say VX-770’s effect appears to be holding up, Vertex said it plans to finalize work with regulators around the world on the design for a pivotal clinical trial to start next year. The data will be presented later this week at the North American Cystic Fibrosis Conference in Orlando, FL.
If it can be shown to work in a larger study, the Vertex drug will be the first to treat cystic fibrosis by directly targeting this underlying mechanism, stimulating the faulty protein that causes the disease, researchers say. In contrast, other cystic fibrosis drugs on the market treat symptoms of the disease (for instance, Novartis’ inhalable antibiotic Tobi, which kills bacteria that build up in the lungs). About 30,000 children and young adults in the U.S. have cystic fibrosis, which causes a buildup of thick, sticky mucus in the lungs, and kills patients at an average age of about 37.
The new drug has its roots in work done in San Diego at Aurora Biosciences, which Vertex acquired in 2001 for more than $500 million. The treatment has also gotten a lot of help from the Cystic Fibrosis Foundation, which has pumped about $79 million into the program since it began.
The results, while still preliminary, showed that patients had a 11.6 percent improvement in what’s called FEV1, a measurement of lung capacity, after 28 days of taking a low dose of the Vertex drug twice a day. That was actually a little better than the high dose, which showed an average improvement of 7.4 percent.
Before patients get too excited, the drug isn’t for everybody. Only about four percent of patients with cystic fibrosis have the specific gene mutation that might be helped by this treatment, Vertex says. Still, these results will certainly get some buzz from researchers in Orlando at its presentation on Thursday, and it paves the way for an important clinical trial for Vertex in the coming year.