HGS's Lupus Drug Shows Promise in Latest Trials
Md. Firm Gets Step Closer to Applying For FDA Approval

By Mike Musgrove
Washington Post Staff Writer
Monday, July 20, 2009

An experimental drug that aims to treat people afflicted with the autoimmune disease lupus showed promising results in its latest round of tests, giving its Rockville-based developer hope that it may have a financial success on its hands.

Human Genome Sciences is set to report the new results on Monday. The drug, called Benlysta, is the first new treatment for lupus in decades to show potential this far into tests with human patients.

"This is just a wonderful outcome, we're delighted," said David C. Stump, HGS's head of drug development. "Patients have been looking for a new medication for 50 years."

If the next round of tests go well, HGS plans to file for Food and Drug Administration approval early next year. The drug could become available late in 2010. The company has not determined how much it will charge for the treatment.

Wall Street analysts believe the drug could be worth billions of dollars for the firm and its partner in developing it, GlaxoSmithKline, if Benlysta makes it to the market.

Given a history of failure in developing drugs for lupus, many Wall Street analysts did not anticipate favorable news.

"We believe odds are against a robust data set," wrote analyst Jason Kolbert of ThinkEquity in a note to investors last week. Kolbert predicted that an unlikely positive announcement from HGS could eventually send the company's stock, which had been trading at under $3 for much of the year, to over $15.

Even facing widespread skepticism, HGS shares rose Friday in anticipation of Monday morning's announcement. After spending the week priced at around $2.50 per share, the stock shot up to $3.63 before closing at $3.32.

The Lupus Foundation of America estimates that about 1.5 million Americans have some form of the disease. Currently, doctors typically use chemotherapy and steroids for lupus, treatments that can have harsh side effects.

"This is a big deal for a lot of people," said Sandra C. Raymond, the organization's chief executive, speaking before the results of the tests were known. In just the past year, lupus victims have seen their hopes dashed three times as other experimental drugs failed to pass their testing processes.

Lupus is a disease that waxes and wanes unpredictably, making drug testing particularly difficult, said Dr. Gary S. Gilkeson, professor of medicine at the Medical University of South Carolina. "It's not like cholesterol, cancer or diabetes, where there's something easy to measure," he said.

Gilkeson, who participated in previous failed trials for experimental drugs designed to treat the disease, said there was concern in the lupus community that another failure or two in this area could discourage drugmakers from trying to develop medicines to treat the disease.

Though the disease's causes are still not entirely understood, the HGS drug is designed to target a protein that becomes overactive in lupus patients and can cause the body to attack its own organs.

For its latest round of testing, 865 patients located in 13 countries outside the United States were given different doses of the drug, or a placebo, for a one-year period. Among those given the placebo, 44 percent experienced meaningful improvement in their symptoms. But among those who took a high dose of Benlysta, 58 percent saw improvement. Fifty-two percent of those who took a low dose improved.

The results were positive enough to meet guidelines for success that HGS had established with the FDA.

HGS's president and chief executive, H. Thomas Watkins, said he is optimistic about the drug's chances in the next round of tests, which will involve a similar number of patients in the United States and Western Europe.

The company has spent hundreds of millions of dollars to develop the drug over the past several years.

"It's a big wager, with no guarantee of success," he said. "We're happy to make those investments, particularly when you get results like this."

One press account earlier this year likened the fight against lupus to cornering a hyperactive cat, a description Watkins and Stump recalled at the end of a phone interview Friday afternoon.

"The cat has been cornered," Watkins said.

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