Lupus drug succeeds in clinical trial: company

Human Genome Sciences Inc said on Monday its experimental drug to treat lupus was successful in a late-stage clinical trial.

The results, which were announced just after midnight on Sunday, showed patients who took the drug, Benlysta, showed an improvement in the symptoms of their disease compared to those taking a placebo.

Lupus is a complex autoimmune disease that causes the immune system to attack the body’s own tissue and organs, including the joints, kidneys, heart, lungs, brain, blood or skin. Symptoms include achy joints, fever, arthritis, kidney damage, chest pain and skin rash, among others.

It is estimated that 1.5 million people in the United States and 5 million worldwide are affected by lupus, according to the Lupus Foundation of America.

Results of the 52-week trial - the first of two requested by U.S. regulators - showed 57.6 percent of patients taking a high dose of Benlysta experienced an improvement in their symptoms, compared with 43.6 percent who took a placebo.

The result met the main goal of the clinical trial.

Of patients who took a low dose of the drug, which is administered once a month intravenously, 51.7 percent showed improvement in their symptoms.

The main goal of the trial was to show a four-point or greater improvement in disease symptoms as measured by a scale known as Selena Sledai. A four-point reduction on a scale of 10 constitutes a good, or meaningful, response. The lower the score the less disease activity a patient has. All patients entering the trial had a score of six or higher.

The trial also required that patients did not experience a worsening of their disease in any organ beyond the originally affected one. The trial met all of these goals at both doses.

Data from the 867-person trial, known as BLISS-52, take the company one step closer to being the first to have a new lupus drug approved in 50 years. There are multiple drugs that are approved for other indications and used to treat lupus, but no drug has been approved specifically for lupus in decades.

Human Genome’s drug is not expected to treat all patients. It is aimed at a subgroup of patients who have mild to moderate disease. That is because an earlier trial failed to meet its main goal, but investigators noticed that a subset of patients did very well.

The late-stage, or Phase III, trials were designed in conjunction with the U.S. Food and Drug Administration, to test those patients most likely to benefit from the drug.

The initial market for the Benlysta, therefore, consists of some 300,000 patients, said Thomas Watkins, the company’s chief executive, in an interview. Roughly 150,000 patients in the United States stand to benefit from the drug, if it is approved.

The second of the two late-stage trials is due to be reported in November. There are no substantive differences between the two trials, the company said. The first was conducted in Asia, Latin America and Eastern Europe. the second in Europe and North America.

If the second, confirmatory trial, replicates the findings in this trial, Human Genome and GlaxoSmithKline would aim to file for approval of the drug by early 2010, according to David Stump, the company’s head of research and development.

If the agency gives it a priority review, the drug could be on the market by the end of next year. Priority review is given to drugs that meet unmet medical need. Without priority review it could be on the market by early 2011.

Benlysta is designed to inhibit BLyS, a naturally occurring protein in the body that exists to keep B-cells functioning normally. B-cells make antibodies that prevent infection. In patients with lupus B-cells are overstimulated, producing antibodies known as auto-antibodies that attack the body.

Analysts had not held out much hope for the drug given disappointing results from a previous trial.

By Toni Clarke
BOSTON (Reuters)