Vertex Pharmaceuticals is being chased by a couple of deep-pocketed competitors—Schering-Plough and Roche—in the race to develop the next big thing for patients with the hepatitis C virus. Now Vertex, the Cambridge, MA-based biotech company, thinks it has found a way to fend off the challengers. It intends to show its drug—telaprevir—can cure patients who have failed on previous treatments, as well as those who are just beginning therapy.
Vertex (NASDAQ: VRTX) unveiled this key element of its strategy last month in the design of a 650-patient clinical trial called Realize. The study will look at whether telaprevir can cure a broad swath of hepatitis patients who didn’t respond to a prior round of treatment, and thus are in danger of serious liver damage later in life. The patients include those who didn’t respond at all to a previous treatment, some who responded partially but not enough, and some who were helped temporarily, but eventually relapsed.
Regulators in the U.S. and Europe allowed Vertex to recruit the hardest cases, known as “null responders” into the clinical trial. Those patients aren’t being allowed into a large trial of Schering-Plough’s competitor, boceprevir. Vertex was able to get those patients included based partly on promising data from a mid-stage study called Prove 3, which has shown that 52 percent of patients on telaprevir had no evidence of the hepatitis C virus left in the blood for at least three months after completing therapy. Standard treatments given a second time do that well for about 15 percent of patients. That kind of difference in effectiveness means big bucks for telaprevir: an estimated 6 million people in the U.S. and Europe have chronic hepatitis C infections, and about 650,000 have failed on the standard treatment. Telaprevir alone could generate $2.6 billion in U.S. sales in 2013 when factoring in patients who failed treatment and those who are new to therapy, according to Rachel McMinn, an analyst with Cowen & Co. in San Francisco.
“Vertex’s telaprevir will show strong data in treatment-failure patients that significantly outshines prior data for Schering-Plough’s boceprevir,” said McMinn, in a note to clients last month, which looks ahead to presentations at the American Association for the Study of Liver Diseases annual meeting in November.
Showing a benefit among the toughest-to-treat patients is important to Vertex, partly because it would appeal to the group of patients who are most motivated to seek new treatment options, and because it could enable the company to make claims that Schering-Plough won’t be able to, says Kurt Graves, Vertex’s chief commercial officer. It also could strengthen the overall package of evidence supporting use of telaprevir, which is being tested in a pivotal study among patients new to treatment, called Advance.
There’s much buzz in the medical and investment communities about both new drugs, protease inhibitors, which have been shown to be about twice as effective at curing the disease as the standard treatments. Many patients can’t stand to take the two drugs used in those standard treatments, pegylated interferon and ribavirin, because they cause flu-like side effects, need to be taken almost a year, and only offer a cure about one-third of the time.
“We’re in a position to be first, our drug can be used for all hepatitis C treatment-failure patients, and we’re likely to be used in a 24-week regimen, not a 48-week regimen,” Graves says. “We think we’re in a good position.”
It’s hard to say precisely how many more patients might be eligible to receive telaprevir if the Realize trial is a success. Among treatment-failure patients, an estimated 30 percent are non-responders to standard therapy, while the rest generate some sort of response that isn’t good enough or doesn’t last, Graves says.
Roche’s contender is being co-developed with Brisbane, CA-based Intermune, and is called ITMN-191. It is still in the earlier stages of development, although Roche recently paid its partner a $15 million milestone payment, and Roche has said it plans to move into the middle stage of clinical trials. Those companies have experimented with a once-daily or twice-daily dose that could be more convenient than either the Vertex or Schering-Plough drugs, which are made to be taken three times a day. Vertex is also looking to protect its flank there, by running a test called C208 to see if a twice-daily form of telaprevir can be shown equivalent to the regular form.
Wall Street will tune in next to the American Association for the Study of Liver Diseases meeting in November, where Vertex is expected to present more data on telaprevir trials in front of an audience of leading physicians. If the data look good, then Vertex shouldn’t have much trouble enrolling patients in the Realize trial. It will still be at least a couple years before we know for sure whether today’s strategy will pay off with greater market share in what’s shaping up to be a feisty new pharmaceutical market.
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