Vertex, J&J Hepatitis C Drug Works After Others Fail

Vertex, J&J Hepatitis C Drug Works After Others Fail

By Ellen Gibson and Elizabeth Lopatto - Bloomberg, Sep 7, 2010 5:16 PM ET



Vertex Pharmaceuticals Inc.?s most advanced experimental drug, in development with Johnson & Johnson, worked in patients with hepatitis C who didn?t respond to other treatment, a study found.


Vertex?s medicine, telaprevir, added to standard treatments for the infection, cured 65 percent of patients overall compared with 17 percent on the other regimen alone, according to a company-funded study that Vertex reported today in a statement.


No proven medicines are available for hepatitis C patients who don?t respond to standard care. If approved by the U.S. Food and Drug Administration, Vertex?s drug may garner $4 billion in global annual sales by 2014, said Phil Nadeau, an analyst at Cowen & Co. Vertex, of Cambridge, Massachusetts, said it will seek U.S. approval of telaprevir in the fourth quarter.


“What most physicians have been doing up to now is warehousing those treatment-failed patients,” Nadeau said in a phone interview. “Now they have a real chance of being cured.”


Standard hepatitis C treatment -- a combination of the antiviral medicine ribavirin and a pegylated interferon such as Roche Holding AG?s Pegasys -- cures fewer than half of those who take them, and many patients develop serious side effects. With a second round of treatment using the same combination, the success rate drops as low as 10 percent.


Shares of Vertex rose 24 cents to $35.25 at 4:49 p.m. New York time in extended trading after closing down 2.9 percent on the Nasdaq Stock Market. Trial results were reported after the close of U.S. markets. The shares fell 3.6 percent in past 12 months.


Racing Merck


Telaprevir is racing boceprevir from Whitehouse Station, New Jersey-based Merck & Co. to become the first new hepatitis C treatment approved in a decade. Both are in a family of medicines known as protease inhibitors that work by blocking an enzyme that viruses use to copy themselves.


“Today?s results give people who haven?t achieved success with prior treatments some real hope for having a cure,” Robert Kauffman, the chief medical officer for Vertex, said today in a telephone interview.


About 3.6 million people in the U.S. and 170 million worldwide have hepatitis C, a chronic and potentially fatal infection, according to the World Health Organization, based in Geneva.


The virus, which typically spreads through contact with contaminated blood, damages the liver and can cause the organ to fail. While many people with hepatitis C have no symptoms and go undiagnosed, others experience stomach pain, jaundice, fevers and fatigue.


Revenue


In the U.S., there are 270,000 to 300,000 patients with hepatitis C who failed standard treatments, Vertex Chief Financial Officer Ian Smith said in a presentation to investors in Boston on Aug. 12. The company expects most of telaprevir?s revenue to come from this group during the first three years after the drug is cleared for sale, he said.


Today?s study, called Realize, was from the third and final stage of testing generally needed for FDA approval. It involved 662 people who had received prior treatment and weren?t cured, or who relapsed after the course of therapy ended.


The trial included three patient groups. One group received telaprevir for 12 weeks, added to ribavirin and interferon, followed by an added 36 weeks on the two older drugs alone. Among those people, 64 percent were cured, meaning the virus was reduced to undetectable levels in a blood test.


Study Results


A second group received that same 12-week cocktail. This group also got 4 weeks of prior treatment and 32 weeks of follow-up therapy with ribavirin and interferon. For them, the cure rate was 66 percent. (from Jules: see results I emailed yesterday - 31% SVR in null responders, 80+% in relapsers, 57% in prior partial responders)


A third set of patients had 48 weeks of treatment with the older drugs alone, and had a cure rate of 17 percent.


About 4 percent of patients enrolled in the trial discontinued treatment with telaprevir, interferon and ribaviron, compared with 3 percent in the control arm. With telaprevir, 0.6 percent stopped because of anemia, and 0.4 percent discontinued due to rashes.


“The safety profile is comparable to previous studies,” Kauffman said.


Previously reported results from two other late-stage trials of telaprevir showed the drug achieved a 75 percent cure rate in patients who had received no prior therapies and that treatment time can be cut to 24 weeks, from 48, in those patients without affecting the drug?s efficacy.


Johnson & Johnson, of New Brunswick, New Jersey, funded the study reported today. J&J agreed in 2006 to pay as much as $545 million to jointly develop the drug and sell it in Europe, South America, the Middle East, Africa and Australia. Vertex retained rights to North American sales. Mitsubishi Tanabe Pharma Corp. of Osaka, Japan, has rights for parts of Asia.


To contact the reporters on this story: Ellen Gibson in New York at egibson9@bloomberg.net; Elizabeth Lopatto in New York at elopatto@bloomberg.net