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Lupus drug makers reach final test stage
Sunday, July 05, 2009

WASHINGTON -- In the high-stakes, high-risk, high-failure business of developing drugs, there are two events that test an executive's intestinal fortitude. One: The first time a drug is tried by a human. Is he still alive? Two: Getting results of final-stage testing. Please, let's hope the tests worked.

Around eight years ago, Human Genome Sciences survived the first event, when patients were first given the firm's experimental lupus treatment. Sometime in July, and many hundreds of millions of dollars later, they will gather in a hotel conference room to see how they endured the second crucial event -- final human testing results.

David Stump, the firm's head of drug development, is hoping that the first PowerPoint slide the biostatistician shows will say success.

But many on Wall Street predict that the suburban Rockville, Md., company will fail. Lupus, a complicated autoimmune disease in which the body attacks its organs, is known as the drug industry's black hole, where even the world's top scientists go to fail.

There hasn't been a new lupus drug approved in 50 years. Not long ago, biotech behemoth Genentech stumbled with its own candidate.

"No one has any real conviction that HGS's drug is going to work," said Geoffrey Porges, a Sanford C. Bernstein analyst. "Nothing has really ever worked in lupus. It's a very difficult disease."

The difficulty in going after lupus is that the disease waxes and wanes, heightening the possibility of a placebo effect, and is a moving target involving many organs. In cancer, drug developers generally target stationary tumors with a laser focus; treating lupus is like trying to corner a hyper cat.

In key mid-stage testing in 2005, HGS fell short of the goal line. But when executives took a closer look at the data, they saw that their drug, now called Benlysta, was successful in treating a subset of patients who had biologic indicators in their blood showing that the disease was active.

HGS Chief Executive Thomas Watkins said Wall Street's reservations did not bother him. He pointed to recent positive results from an extension of the last study, which showed that for patients who stayed on the drug, after four years there was sustained improvement in the disease.

First published on July 5, 2009 at 12:00 am
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