Replies to post #54169 on Biotech Values

[DewDiligence]

More on Telaprevir and a mysterious
drug for HCV that may actually work.

http://www.nytimes.com/2007/11/02/business/02drug.html

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2 Winning Drug Tests, One Expected and One a Surprise

November 2, 2007
By ANDREW POLLACK

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. [The 79% figure is an SVR12 rate rather than an SVR rate, and the treatment group had genotype-4 HCV, which is common in the Middle East.]

“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.”

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. Jacobson 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 [i.e. the SVR rates from the SoC control arms] 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. Jacobson of Cornell said that would be “very premature.”
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[ThomasS]

VRTX: I'd say it's pretty good for a 24 week treatment in that Genotype.
Regardless, we need an outright vaccine or better Inf such as ZGEN's candidate... as we are still in the HCV treatment Rock Ages.

[DewDiligence]

VRTX off 9% at the open (eom).

[DewDiligence]

Telaprevir Data Fails to Excite Investors

[This is a rewrite of the newswire in msg #54169 with added remarks from analysts about how the data fell short of the lofty expectations.]

http://www.reuters.com/article/marketsNews/idUKN0255798620071102

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Fri Nov 2, 2007 10:51am EDT
By Toni Clarke

BOSTON, Nov 2 (Reuters) - Vertex Pharmaceuticals Inc (VRTX) released data on Friday showing its experimental hepatitis C drug is more effective than existing treatments and works in half the time, but causes significantly more side effects.

The results disappointed investors who sent the stock down more than 10 percent in early trading.

Data to be presented at the Annual Meeting of the American Association of the Study of Liver Diseases, showed the drug, telaprevir, in combination with standard treatment, eradicated the hepatitis C virus in more than 60 percent of patients who took it as part of a 24-week treatment regime.

Typically, not more than 50 percent of patients taking standard treatments see their virus eradicated and the typical length of treatment is 48 weeks.

Still, some investors had hoped to see cure rates closer to 70 percent, and since telaprevir is Vertex's most important experimental product, the disappointment sent Vertex's shares plunging.

"From an expectations point of view this is negative, especially when you consider the competitive landscape," said Rachel McMinn, an analyst at Cowen & Co.

In a mid-stage trial conducted in the United States known as PROVE 1, 61 percent of 79 patients who took telaprevir in combination with the standard treatments, pegylated interferon and ribavirin, saw the virus fall to undetectable levels and remain undetectable six months after treatment was stopped.

But 13 percent of patients discontinued the trial in the first 12 weeks because of side effects. The most common side effect was rash.

"The takeaway here is that it looks like telaprevir is an approvable drug," said Jason Kolbert, an analyst at Susquehanna Financial. "It clearly has efficacy, but it also clearly has toxicity."

In a separate mid-stage trial known as PROVE 2, conducted in Europe, 65 percent of patients who took part in the 24-week treatment regime -- which consisted of 12 weeks of triple therapy followed by 12 weeks of standard treatment -- saw the virus eradicated and stay eradicated after three months.

Those results were below expectations.

"The expectation on the Street was 70 percent plus," said Ding Ding, an analyst at Maxim Group.

In the PROVE 1 trial, the relapse rate was 2 percent. In PROVE 2 it was 14 percent. Patients who take standard therapy typically relapse at a rate of 20 percent to 30 percent, Vertex said.

The difference in relapse rate between the two trials stemmed from the fact the PROVE 1 trial results were based only on patients who were least likely to relapse in the first place, based on the rapidity of their initial response to the drug.

In the PROVE 2 trial, which did not just measure patients who saw a quick drop in virus after starting therapy, 14 percent of patients relapsed.

Telaprevir is one of a new class of drugs that inhibits a hepatitis C protease, an enzyme essential for the virus to replicate. Vertex's partner on the drug is Tibotec Pharmaceuticals Ltd, a unit of Johnson & Johnson (JNJ) that is based in Ireland.

Shares of Vertex fell $3.64, or 11.5 percent, to $28.00 in early trading on the Nasdaq.
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