Firm's future hinges on new hepatitis drug

For one of Boston's biggest biotechnology companies, the future hinges on a potential billion-dollar drug and a liver-research conference about to kick off 3,700 miles away in Spain.

At the annual meeting of the European Association for the Study of the Liver , starting this week in Barcelona, perhaps the most hotly anticipated presentation will be Vertex Pharmaceuticals Inc.'s unveiling of new data from human trials of its pill for hepatitis C.

Between 3 million and 4 million Americans are thought to be infected with hepatitis C, a virus that can lead to cancer and liver failure. It spread through the blood supply before broad screening began in the early 1990s. Infected people can harbor the disease without symptoms for over a decade.

Vertex's pill, which mounts a new type of attack on the virus, has achieved dramatic results in early tests on a handful of infected patients. In most, it appeared to eradicate the virus in a matter of weeks. On Saturday evening the company plans to unveil details of a bigger trial on 250 patients, comparing those on standard therapy with others who got therapy plus Vertex's drug.

Dr. Raymond Chung , a liver specialist at Massachusetts General Hospital , said Saturday's results won't tell the whole story, but he called them an important indicator of how well the drug might work. Along with a handful of similar drugs in development, he said, "You're seeing the leading edge of a real shift in treatment."

Currently, patients are prescribed a year's worth of interferon injections plus pills, an $18,000 course of therapy that causes symptoms like a severe flu, and cures half of those who endure it.

Chung said he was particularly interested in one "shoot-the-moon" strategy that Vertex was employing. In a multipart trial of the drug, one group was taken off treatment after just three months -- far less time than the current standard of a year. On Saturday, Vertex expects to reveal whether the virus bounced back in those short-term patients, or whether they managed to stay virus-free long enough to be considered cured.

Even if Vertex's pill can't eradicate the virus in 12 weeks, Chung said, it could still be important if it boosts longer-term interferon treatment.

In a reflection of how crucial the drug is for the company, Vertex's stock has risen and fallen sharply in the past year with disclosures of even tiny pieces of medical data. It jumped nearly 20 percent one day last fall when some early positive news came out on a trial; it slid in December when the company revealed that 9 percent of clinical-trial patients had to discontinue treatment because of a serious rash and other side effects. Last week, the stock rose 10 percent, simply on anticipation that this weekend's results would be positive.

With 700 local employees and an 18-year history in Cambridge, Vertex is a pillar of the local biotechnology landscape. But its ups and downs suggest how important a single disease, and a single product, can be to even a well-established biotech company.

Vertex has only one product on the market, a minor AIDS drug sold by GlaxoSmithKline PLC. The company loses money every year, paying far more for research than it books in revenue, but one big success could change that equation. Although its hepatitis C drug is unlikely to reach the market before 2009, analysts estimate it could rack up sales of $1 billion a year.

Attracted by that potential market, a number of other firms are taking aim at hepatitis C, many in Boston. Enanta Pharmaceuticals Inc. , a private company in Watertown, recently signed a deal with Abbott Laboratories of Illinois to develop drugs similar to the one Vertex is testing.

Idenix Pharmaceuticals Inc., which is majority-owned by Swiss drug giant Novartis AG, is testing a drug with a slightly different mechanism, as is the New Haven company Achillion Pharmaceuticals Inc.

The promise of a fast-acting new treatment for hepatitis C has already paid off for Vertex, which signed an international sales deal with Johnson & Johnson last summer. But it also carries risks. A hepatitis C drug being developed by a European company caused toxic side effects in animals. And after unsuccessful trials, Coley Pharmaceutical Group of Wellesley suspended tests of its experimental hepatitis C drug in January, laying off 33 of its 150 employees.

This week's presentation is only the first in a series of findings expected from Vertex. Still in the wings: a larger trial involving 320 patients, and a third trial with 440 patients who weren't cured by standard therapy.

If the company succeeds, it will also face another question: What to do about the tens of millions of people with hepatitis C in developing nations who can't afford the steep price of therapy.

Vertex said it has a deal with Johnson & Johnson to start an international foundation dedicated to hepatitis C treatment.

"It's something that we've talked about extensively, and having a partner that shared our view of the importance of that was key," said Vertex spokesman Michael Partridge , although he said it was too early to discuss details of the program.

Stephen Heuser can be reached at sheuser@globe.com.