Vertex Maps Out Combo Drug Game Plan for Treating Hepatitis C

Xconomy Boston — 

HIV has taught the pharmaceutical industry that the best way to fight an infectious virus that resists a single drug is to make a cocktail that attacks the virus in more than one way. Vertex Pharmaceuticals and its competitors are now following a similar formula with new therapies for hepatitis C.

Vertex, the Cambridge, MA-based company with operations in San Diego, offered a glimpse last week into its strategy for a two-drug combo that could significantly change hepatitis C treatment. If the company has mapped this out correctly, it could rid people of the virus while letting them ditch the detested standard therapies that force them to endure months of flu-like symptoms. I followed up last Friday to learn more from a conversation with Vertex’s chief medical officer, Bob Kauffman.

The big story from the past couple years at Vertex (NASDAQ: VRTX) is the development of its first-in-class protease inhibitor drug called telaprevir. This oral pill, taken two or three times a day, must be combined with the pegylated interferon alpha and ribavirin. It has excited researchers because it has been able to double the cure rate while shortening the course of therapy by half. That means that many more of 170 million people worldwide with chronic hepatitis C liver infections will be likely to seek out treatment, and be able to stand up to the side effects of standard therapy over a shorter period of time. If the ongoing clinical trials to test this idea are successful this year, Vertex could bring telaprevir to the market in 2011. U.S. sales alone could amount to more than $2 billion after a couple years, researchers say.

Bob Kauffman

Bob Kauffman

That will be headline news if and when it happens. But researchers around the world, and Vertex’s competitors, see even bigger advantages if someone can get rid of pegylated interferon alpha and ribavirin altogether. The vision is to combine a protease inhibitor like telaprevir with one or more other antiviral drugs that work differently, essentially blocking some of the escape routes that enable the virus to develop resistance to a single drug.

There are three other main therapeutic classes being tested in clinical trials—nucleoside polymerase inhibitors, non-nucleoside polymerase inhibitors, and NS5a inhibitors, Kauffman says. Vertex paid $375 million last year to acquire a small Canadian company, ViroChem Pharma, mainly to obtain a non-nucleoside polymerase inhibitor that Vertex thought would complement its own telaprevir. That drug, called VX-222, has shown promising antiviral punch in some small studies on its own, and now it is entering its first serious test in tandem with telaprevir in a clinical trial.

“It really became clear early on that these hepatitis C agents couldn’t really be given [as single agents] because of the virus’s ability to develop resistance,” Kauffman says. Combinations have long been thought to be the best way forward, and to get rid of the standard treatments. “That was the goal from the beginning,” Kauffman says.

Vertex spent the last year figuring out the right doses, and balance between the two drugs, that it thought would be ideal for a teleprevir/VX-222 combination trial. Last week, it unveiled … Next Page »

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19 responses to “Vertex Maps Out Combo Drug Game Plan for Treating Hepatitis C”

  1. cheryll says:

    If this is true , i know i would seek out the treatment,and try it. i have cronic hep c,but wont endurethe 12 month long sickness with only 30% chance of it helping.
    i would like to find out more about this treatment and how i can get involved in its use.

  2. rOBERT says:


  3. i would be interested having relapsed once

  4. Vertex has a hotline for patients who want to check into whether they are eligible for clinical trials. Here’s how to find more.
    Clinical Trials & Medical Information:

  5. marvin lyons says:

    This is good news for the hep-c community. I hope and pray that it works. The interferon and peg cause me temporary blindness.

  6. Michele says:

    My husband did the pegintron/ribavirin shuffle three times. The first two times the side-effects were miserable but his viral load dropped to undetectable for several months until it gradually returned. The third and last time he developed interferon psychosis, which leaves him with no current treatment option. A new combo therapy without interferon would be a life-saving dream come true.

  7. Diane says:

    I hope and pray that a new drug therapy minus interferon will be developed. Was on the combo therapy and although everyone on treatment gets hemolytic anemia, I developed auto-immune hemolytic anemia, meaning my own white cells were targeting my red cells, caused by interferon. If anyone knows about non-interferon based trials, please post. I’ve got alot of living to do….

  8. Sallie Little says:

    My son has hepatitis c and has a family of 6 total, and has to work. He cannot take the interferon and work. Is there anything he can take with the midication and still work? At this time he is not taking the medication!!!
    Thank you for your reply.

  9. Patrick DeHart says:

    Hello folks, I am a Vietnam era veteran as well as a veteran of the year long Hepatitus C treatment in 2007-2008. I am so glad to hear about this good work that the research scientists are doing that I could cry. I went through the Ribavirin-Interferon treatment at the VA in Fayetteville, Arkansas. God bless their hearts because they are the best. However, I was labeled a non-responder which is just not true in my case. I was under 50 bugs per milliliter from the first viral load blood test all the way through the therapy. At the end, I was absolutely clean, no virus. I felt great and I even went back to college to finish my Degree which I’m still doing. Unfortunately, after six months, the Hepatitus C came back by a factor of Ten. The bottom line is that I am more than ready to try anything that has twice the cure rate and at only half the time of the miserable year that I spent on my couch. I have the best wife in the world and she helped me make the whole year. There is a staggering large vanguard of veterans from the late 1960’s to early 1970’s that have Hep C. Some believe that it was the poorly sanitized Jet Injectors (innoculation guns). In any case, I once again thank the researchers. Warm Regards, Patrick DeHart

  10. Steve Walls says:

    Great stuff, a stand alone protease inhibitor is the answer.I hope the drugs companies give up on interferon and ribavirin, but you & I both know that drug companies never give up on drugs they have heavily invested in. Hopefully they will develop a conscience and develop the new therapies asap, as most people would get treated.
    There is an antibody that has been discovered which hopefully the drug companies haven’t bought & shelved, which clears hep c.Steve

  11. cheryll says:

    great comments,i too wish “they” would wake up,and find a different answer, ihaven’t heard too many g ood stoires about the first type of treatment,alot of people areatillbeing used as guinie pigs it sounds like.
    I would love to help discover the cure of a med to help.
    I don’t understand how they ca nfind something for aides,and not hep c

  12. kimellen says:

    This is wonderful news…if I weren’t in school, and already looking at joint surgery, I would absolutely enroll in the trial for this. I am still asymptomatic, and feel my health is better off for not taking the interferon/ribo proticol…I’m in the medical field and have done a lot of reading..if you maintain a healthy lifestyle…no drinking and drugging…your chances are good. But all other comments are right..its time for a cure!

  13. Donna says:

    I am hoping and praying this is true. I have had Hep C for 27 years now and been through the trials of Pegalated Interferon and Ribavirin. It was hard! I was classified as a non-responder. I have been asymptomatic since then. I lost my dad and aunt this last year, both from liver Cancer due to Hep C. We all contracted it about the same time. My life style is good and has been for many many years but my test results keep getting worse. I am ready for that cure!

  14. wm. donner says:

    all i hear is about relapsing after treatment 6-18 mo after treatment. are there any people out there that are still clear of the virus 5+ years on? i never hear of any and am beginning to think it never goes away. great for the drug companies. am coming up on 24 wks of standard treatment plus first 12 wks were also with experimental drug. standard flu symptoms all the time, horrible eczema, and am feeling a little nuts and miserable to everyone. is it really worth it?

  15. Cheryll says:

    I have been to difffernt sights,and i have heard of very few who the first treatment helped. unless thier viral load was low,seems to me they are still experimenting.
    it would be cool if we could some how get the government tosee out comments and actual results of the other verses different aproches.

  16. Robert says:

    I was diagnosed in 2008 my viral was low at 400,000 after 12 weeks of treatment it spiked to 1 million, I have also compensated scerosis, hep-c virus only lives 6 hours outside the body. These companies are bound to find the cure. I have also decided to start taking livercell for my liver. My doctor has told me that we will start treatment again, I also considered stem cell therapy