With new data released this evening, Vertex Pharmaceuticals is closer to bringing its third cystic fibrosis treatment to market, which would mean the company’s product line could potentially treat upwards of 40 percent of the roughly 70,000 people with the disease worldwide.
The late-stage data for the combination of ivacaftor, already approved as Kalydeco, and the experimental tezacaftor, show that patients with the deadly lung disease had improved lung function compared to patients on placebo.
But for Boston-based Vertex (NASDAQ: [[ticker:VRTX]]), the data have significance beyond the next FDA approval, which the company says it will ask for later this year. The strong showing of the ivacaftor-tezacaftor combination means Vertex’s strategy to build three-drug cocktails with ivacaftor-tezacaftor as the backbone is on the right track. Two triple combinations have already reached mid-stage studies, and others could follow. If Vertex succeeds with even one of these combinations, it might one day have a single treatment for a majority of patients, says CEO Jeff Leiden. Leiden calls today’s data “a huge de-risking” of the triple program. “We have two legs of the stool solid and in place,” he says.
“It’s an exciting time for those of us taking care of CF patients,” says Dennis Nielson, a professor of clinical pediatrics at the University of California, San Francisco, referring in general to the prospect of triple-drug combinations.
In one Phase 3 study, dubbed EVOLVE, Vertex enrolled 504 CF patients 12 and older who have the most common form of cystic fibrosis, affecting about half of the population. The patients on ivacaftor-tezacaftor saw an average 3.4 percent increase in a measurement of lung function from the start of the study, compared to an average 0.6 percent decrease for the 256 placebo patients—an average absolute improvement of 4.0 percent.
Patients on the drug combo also had a total of 78 pulmonary exacerbations—acute flare-ups of significant lung problems that can lead to hospital stays—compared to 122 on placebo, a roughly 36 percent reduction.
In the second Phase 3 study, EXPAND, 250 patients with less common forms of CF got either ivacator/tezacaftor, ivacaftor alone, or a placebo. After eight weeks, patients on the combo had a 6.8 percent absolute improvement in lung function compared to placebo, whereas the figure was 4.7 percent better than placebo for ivacaftor alone.
The most common reported side effects were pulmonary exacerbations caused by infections, cough, headache, colds, and increased sputum. Vertex said that the rates of those with respiratory side effects and the rates of patients dropping out of the studies were low, with little difference between the ivacaftor-tezacaftor and placebo arms of the studies. In EVOLVE, 41 placebo patients had respiratory side effects and eight dropped out due to side effects, while 33 patients on ivacaftor/tezacaftor had such side effects and 7 dropped out. (No one on ivacaftor/tezacaftor dropped out of EXPAND).
“This is about as good a result as you could expect,” said Patrick Flume, an investigator in the Evolve study and the director of the Cystic Fibrosis Center at the University of South Carolina.
Shares of Vertex climbed more than 17 percent in post-market trading on Tuesday.
Others not involved in the two studies also were positive but warned that
