2 New HCV Drugs at AASLD

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2 New HCV Drugs at AASLD

2 Winning Drug Tests, One Expected and One a Surprise

NY Times

By ANDREW POLLACK

Published: November 2, 2007

An experimental drug from Vertex Pharmaceuticals helped cure more

than 60 percent of patients with a tough-to-treat form of hepatitis

C, according to data to be presented at a medical meeting that starts

today.

The results, eagerly awaited by Wall Street as well as by doctors,

represent the highest cure rate yet reported for the condition - and

the treatment was accomplished in half the usual time.

But Vertex might have to share a bit of the limelight with Romark

Laboratories, a tiny privately held company that already sells a drug

called Alinia that is used to treat diarrhea caused by certain

parasites but that suddenly is emerging as a potential treatment for

hepatitis C.

In a presentation that has escaped the attention of Wall Street,

though it is to be made at the same meeting, researchers are to

report that Alinia helped cure as many as 79 percent of hepatitis C

patients, although they had a form of the virus that might be

slightly easier to treat than the type Vertex took on.

“There will be skepticism because this has come out of right

field,†said Dr. Emmet B. Keeffe, chief of hepatology at Stanford,

who is a consultant to Romark. But Dr. Keeffe said that he had

overcome his own initial skepticism and that the results were

“pretty exciting.â€

Both developments, experts said, will need to be followed up by

larger trials. But the progress could be important for patients. At

least three million Americans are thought to be infected with the

hepatitis C virus, and the number of cases of liver cirrhosis and

liver cancer caused by the virus is rising.

The existing treatment - a combination of two drugs, alpha interferon

and ribavirin - can cause debilitating side effects like flulike

symptoms, anemia and depression. The treatment for type 1 hepatitis

C, the hard-to-treat form that accounts for 70 percent of the cases

in the United States, takes nearly a year.

So the hunt for new drugs has been intense, and Vertex, a publicly

traded company based in Cambridge, Mass., is widely acknowledged as

the front-runner. Its pill, called telaprevir or VX-950, interferes

with a viral enzyme. This is how most AIDS drugs work, but it is a

new approach for hepatitis C.

While Vertex has previously shown that telaprevir can sharply reduce

virus levels in patients' blood, the new data, to be presented at the

meeting, an annual gathering of the American Association for the

Study of Liver Diseases in Boston, is the first strong signal of how

many patients could be cured.

In a midstage trial in the United States of 250 previously untreated

patients, 61 percent of the people who received telaprevir in

addition to the two standard drugs had no detectable virus in their

blood 24 weeks after the end of the treatment, a Vertex executive

said. Doctors consider that an effective cure.

In a European trial of 323 patients, about 65 percent of those who

got the three-drug combination had no detectable virus 12 weeks after

the end of treatment.

Both rates are higher than the 40 to 50 percent cure rate now typical

for type 1 hepatitis C using the standard drugs alone. Perhaps as

significant, doctors said, the treatment took 24 weeks instead of the

usual 48. That means patients would have to endure the side effects

of interferon and ribavirin for a shorter time.

“These are exciting findings,†said Dr. Ira M. son of Weill

Cornell Medical College, who will present the results of the American

trial at the meeting and is a consultant to Vertex.

But the numbers are pretty much what Wall Street expected. And many

more people getting the three drugs stopped treatment because of side

effects than those getting the standard therapy. The trials' cure

rates for the standard therapy are not yet available.

The Romark data come from a study in Egypt of only 96 previously

untreated patients. Alinia, also known as nitazoxanide, had

previously been tested there as a treatment for parasites.

Of the patients who received Alinia along with the two standard

drugs, 79 percent had no detectable virus 12 weeks after the end of

treatment. That compared with 43 percent in the control group

receiving the standard treatment. While the total treatment lasted

the usual 48 weeks, the patients received the two standard drugs for

only 36 weeks.

The patients had type 4 hepatitis C, which is rare in the United

States. Some experts said type 4 was slightly easier to treat than

type 1. Romark, which is based in Tampa, Fla., and has about $20

million in annual revenue, has begun a trial in the United States.

The drug's effectiveness against hepatitis C was discovered almost by

accident. When tested as a treatment for parasites, the drug showed

signs it was countering liver infection.

Dr. Keeffe, who is researching how the Romark drug works, said that

because Alinia is already on the market and appears to have few side

effects, some doctors might prescribe it off label to treat hepatitis

C. It costs about $30 a day for the two pills needed. It is legal for

doctors to prescribe medicines for uses other than those the Food and

Drug Administration has approved, but a drug maker cannot promote its

medicines for those off-label uses before formal approval.

“We're all looking for better therapy for our patients, and this

looks like a very benign thing to do,†Dr. Keeffe said.

But Dr. son of Cornell said that would be “very premature.â€