Vertex Pharmaceuticals has been in business for more than 20 years, and burned through more than $2.8 billion on a quest to develop drugs that shake up the medical standards of care. Now in the coming weeks and months, it will get the first definitive evidence that will say whether its lead drug candidate for hepatitis C has achieved its goal.
Investors, looking at data from preliminary and mid-stage clinical trials, have already pumped up the market value of Vertex (NASDAQ: [[ticker:VRTX]]) past $7.5 billion largely in anticipation that its drug, telaprevir, will transform the lives of patients with the chronic liver-damaging condition. But the operative word there is preliminary. Vertex, which is based in Cambridge, MA, and has significant operations in San Diego, is still waiting for its first proof from Phase III clinical trials, the final step of testing required by the FDA before a drug can go on sale in the U.S.
These pivotal trials began two years ago, and enrolled more than 2,000 patients combined in three studies. The results are completely blinded to doctors, patients, investors, and people at Vertex. To say all parties are in suspense for these results would be an understatement. They can’t wait to get their hands on the new data and start digging through it.
“This is a year of defining moments,” says Bob Kauffman, Vertex’s chief medical officer.
Vertex has generated the excitement around what could be a first-in-class protease inhibitor drug for hepatitis C. It has excited researchers because it has been able to double the cure rate while enabling patients to cut their standard course of treatment time in half. That means that many more of the 3 million Americans and 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 that causes flu-like symptoms. If Vertex can prove this idea once and for all in the three pivotal trials, Vertex could seek FDA approval later this year and bring telaprevir to the market in 2011. U.S. sales alone could amount to more than $2 billion after a couple years, analysts say.
To help our readers get ready for this data, I spoke to Kauffman for a refresher on what the three big Vertex trials were designed to ask and answer. The key point to watch for in all of these studies is what is called a “sustained viral response,” or SVR, which is recorded when researchers can’t find any sign of virus in a patient’s blood sample for a full 24 weeks after they completed their course of therapy. This is the gold standard measurement for all hepatitis C drugs, and is commonly known as a “clinical cure.”
The first study to watch for is called “Advance.” This trial, started in March 2008, enrolled 1,050 patients who had never been treated before for hepatitis C—a so-called “naïve” patient population. This study is essentially designed to confirm earlier trials called Prove 1 and Prove 2, Kauffman says. Patients either got the Vertex drug in combination with standard treatments for 24 weeks, or the standard treatments for the usual 48 weeks. The trial is designed to ask whether patients can stop treatment early with the Vertex drug, so they can avoid having to put up with the flu-like side effects of pegylated interferon alpha and ribavirin, Kauffman says. Results from that study should be available by the end of June, he says.
Not long after that data arrives, Vertex plans to hear results from two other key trials before the end of September. One of them, called “Illuminate,” is enrolling patients who have never before
