Vertex Fending Off Competitors By Treating the Toughest Patients With Hepatitis C

9/9/08Follow @xconomy

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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  • http://blow H Blower

    Any treatment that has high % of cure without the dreadful side affects leaving one worse than before with or without the disease would be welcome that is a fact

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  • ejbSF

    For those of us in need – some desperate – for new HCV treatments, this song and dance of study after study for market positioning is just wrong. Telepravir works for a lot of people who haven’t responded to previous treatments. GET IT OUT THERE, VERTEX! LIVES ARE ON THE LINE!

  • Paul

    I guess the drug companies have a dilema! They are currently making billiions of dollars on some people being cured. They are also invested rescue drugs that are required by many doing tx due to reduced white and red blood cell indicators. Now many txers are exending to do 72 weeks which means more units sold. Makes you wonder how hard they’re trying, if a cure is found, that gravy train crashes immediately. My sense is that vertex is pushing like crazy because they’re not so heavily invested in current tx regimes like Roche and Schering-Plough.

  • Vicki DeWitt

    If Vertex new hcv drug has already shown so much potential, isn’t there a way to get it approved sooner?

  • steph

    Any free studies available to get into please?
    Any info. would be appreciated

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  • Vickie Soriano

    The FDA are the ones who need to fast track this to approval. I have been watching this for a couple of years now and it looks to me like Vertex is doing everything they can to get this out as quickly as possible. I also understand their caution. We do live in a sue happy society. They need to play by the rules and dot their i s and cross their t s. I am just grateful there is hope on the horizon.

  • barry senn

    If there is any way to be a guinea pig in these studies, please someone let me know. thanx Barry in Oregon

  • Janet A

    Stop playing politics with our lives! I didn’t respond as well as hoped with the 48 week treatment and am borderline cirrohis. I need this as well as thousands of patients like me. I would be more than willing to be a guinea pig. I live in fear every time I go to the doctor that my options are getting fewer, but with this treatment, I have tremendous hope for a cure…but the politics! Vertex…please push this thru!

  • http://XCONOMY/BOSTON JAMES T LAWSON

    HOW CAN I GET INTO ONE OF THESE CLINICAL TESTS,MY SON WHO LIVES IN THAILAND FAILED IN THE FIRST COURSE OF TREATMENTS AND REFUSES ANYTHING THAT MADE HIM FEEL AS MISERABLE AS HE DID FOR 48 WEEKS.

  • http://www.xconomy.com/author/ltimmerman/ Luke Timmerman

    Patients who want up-to-date information on Vertex clinical trials can check here.

    http://www.vrtx.com/current-projects/Clinical-Trials.html
    Clinical Trials & Medical Information:
    Phone:
    877-634-VRTX
    617-444-6777

    For information on other open clinical trials for Hepatitis C, check here:

    http://clinicaltrials.gov/

  • Maria Becker

    My husband tried to get into this clinical trial but he was already on Interferon for the 3rd time so they wouldn’t take him in… Please hurry and get it on the market, we would have been willing to pay if it meant he could have gotten into this study. We have heard all good things about it.

  • Jasper

    Dear Dr. Kauffman,
    I remember contacting Vertex 9 years ago and I inquired about one of your early protease inhibitor. I cannot remember the name but it was something with a V and I believe a 6 was in the sequential numbers.

    Please, my point is that I was a FF and exposed to HCV and developed an autoimmune disease where if affects my autonomic and sensory nervous systems.

    I have tried interferon by itself as well as Interferon with ribavirin and my viral loads went down some and then back up into the millions but I never did clear the virus. Since then I have an internal pain pump with dilaudid which caused a granduloma in my spine so now I am back on large doses of oral meds with all of the side effects. My digestive tract does not function without large amounts prescription laxatives as the nerves fail to dilate my intestines. Needless to say I have some debilitating problems which are progressing.

    Sir, I am asking you to PLEASE get this product approved by the FDA and inform all of the HCV patients in the USA, as well as the rest of the world, as how we can help you by lobbing our politicians at the federal level.

    Yes, you are well ahead of the bigger money pharms and they should not hinder the process so once again, PLEASE let us help as well!

    Thanks, be strong and HELP us,
    Jasper

  • Greg

    I have been fighting this for 20 plus years now and would love to see this be approved I feel its my last hope for all other treatments that Ive done have failed.

  • Jane

    Hi to all who are waiting…I have been watching the stats on this drug for over 5 years and its very exciting to think its getting closer. Fast track for us is to slow for most of us…I hope I can stay stable until we get the green light to go. Our insurance coverage will not cover drugs until they are approved by the FDA…I just saw 2011′ and I thought the last time I called to Vertex they said last quarter of 2010. Hurry up to wait…Keep looking up and pray that all out there will be able to be able to clear the virus. We have to hang in….