The Cambridge, MA-based biotech company, which has significant operations in San Diego, is announcing today that a combination of standard treatment and its novel drug for hepatitis C was able to cure two-thirds of patients who had failed to respond to a prior round of the standard drugs alone. That response rate for patients on Vertex's telaprevir (65%) far exceeded that of the comparison group, in which just 17% of patients were cured after getting the usual combination of pegylated interferon alpha and ribavirin. Vertex is making the announcement today, based on findings from more than 660 patients who enrolled in a study called "Realize."
"The Realize data represent a major milestone in the development of new treatments for hepatitis C," Stefan Zeuzem, a professor of medicine at the JW Goethe University Hospital in Frankfurt, Germany and principal investigator of the trial, said in a Vertex statement. "These results may provide hope to people who have not been cured and who are in need of new treatment options."
This is the third major batch of study results from Vertex this year, and part of its quest to shake up the standard of care for patients with hepatitis C, a chronic liver disease. The company showed back in May, in a study of more than 1,000 patients, that about three-fourths of people getting their first round of treatment were considered cured after getting telaprevir in combination with the standard meds. A second study, released last month, showed that the drug could cut the treatment time in half, which is important because it means patients don't have to endure the flu-like symptoms caused by the other drugs in the regimen for nearly as long. And today's announcement reinforces findings from small trials that say the telaprevir-based regimen has far greater ability to kill the virus in the toughest patients to treat.
The business opportunity, which we've written about a lot in these pages, is huge. An estimated 6 million people in the U.S. and Europe have chronic hepatitis C infections, and an estimated 650,000 of them have failed a prior round of the standard treatment. If the FDA clears the drug for sale based on the latest clinical trials, telaprevir could generate more than $2.6 billion in U.S. sales by 2013, according to analyst Rachel McMinn of Cowen & Co.
Based on the results of the three pivotal trials, Vertex plans to file an application for FDA approval by the end of this year.
The drug's side effect profile appeared to be similar to what researchers have already seen from telaprevir. Patients in the trial reported cases of fatigue, itching, headache, rash, flu-like symptoms and nausea. About 4% of patients in the telaprevir group dropped out of the study because of adverse events, while about 3% discontinued in the control group, Vertex said.
The latest results from the Realize study are not just important to patients, but also to Vertex from a competitive standpoint. The company is preparing to face off against Merck's boceprevir, another drug from the class of anti-viral drugs known as protease inhibitors. Merck released results from a pair of Phase III clinical trials last month, one in which patients were getting their first round of therapy, and another in which patients were getting re-treatment. The re-treated patients had a clinical cure rate of 59% to 66%, according to this recap from TheStreet.com.
Of course, the Merck and Vertex trials were designed differently, and didn't test the drugs head to head, so it's really an apples-to-oranges comparison. But Vertex offered some insight in this feature back in September 2008 about how it designed the Realize trial to hopefully provide a little extra advantage for its product in the minds of physicians.
Now that Vertex has cleared all three major trials that it needed to run to fill out its FDA application, the question of how telaprevir stacks up against the Merck drug and how it will be received by physicians and patients will take on even greater significance than before. You can be sure that patients are going to be hounding the company and the FDA about exactly when this drug might become commercially available in the U.S.
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Luke Timmerman is the National Biotech Editor of Xconomy, and the Editor of Xconomy Seattle. You can e-mail him at firstname.lastname@example.org, or follow him at twitter.com/ldtimmerman.