Human Genome Sciences Novel Lupus Drug Cuts Symptoms In Trial
The 865-patient study, known as Bliss-52, found Benlysta was more effective at easing pain, hair loss and skin rash than a placebo, according to a statement from Rockville, Maryland- based Human Genome and London-based Glaxo.
Positive results were unexpected by analysts who cited disappointing data in earlier clinical trials and a history of failures for treatments of lupus, which triggers the immune system to attack healthy cells. Ten of 12 analysts rated Human Genome a “hold” or “sell” as of July 17, according to Bloomberg data. No cure exists for the disease, which affects about 5 million people worldwide.
“The lupus community has really been on tenterhooks waiting for a positive announcement,” Sandra Raymond, chief executive of the Lupus Foundation of America, a Washington, D.C.-based patient advocacy group, said in a July 17 telephone interview, before the results were announced. “Since lupus was first discovered over a century ago, there has never been a drug specifically developed for lupus.”
Patients in the study received one of two doses of the drug or a placebo. At the lower dose, 51.7 percent of patients showed improved symptoms compared with 43.6 percent who took the placebo, Human Genome said in a statement. At a higher dose, 57.6 percent had improved symptoms compared with 43.6 percent on a placebo, according to the companys statement.
Criteria for Success
“We have met the criteria in this study for success,” said Thomas Watkins, Human Genomes president and chief executive, in a telephone interview. “Now we need to do it a second time in the other study.”
Human Genome is conducting a separate, 76-week trial of Benlysta. Both studies are being conducted under a “special protocol assessment” from the U.S. Food and Drug Administration, which allows a drugmaker to work with the agency to design a late-stage clinical study with scientific goals that, if met, would be sufficient for marketing approval.
Results of the 76-week trial are expected in November, Watkins said.
Benlysta and an experimental drug for hepatitis C are Human Genomes lead products. The company also is conducting late- stage studies of novel treatments for anthrax, Type 2 diabetes and heart disease. Human Genome closed at $3.32 on July 17 in Nasdaq Stock Market composite trading and has fallen 49 percent in the past 12 months.
Peak Sales
Benlysta could reach peak sales of $600 million worldwide in the “unlikely event” it reaches the market, Jim Birchenough of Barclays Capital said in a July 6 note to clients.
Lupus has been a challenging illness for researchers and drug developers trying to design clinical trials because patients symptoms vary, the illness effects different organs in the body and symptoms tend to come and go, said Raymond.
“Each patient is like a snowflake, with no two the same,” she said.
Prevalence in Women
Lupus causes the immune system to attack healthy cells and tissues by mistake, damaging joints, skin, blood vessels, heart and lungs. It is most common in women ages 15 to 45 and more prevalent in African Americans, Hispanics, Asian and American Indians. About 1.5 million people in the U.S. have the disease, according to the U.S. Department of Health and Human Services.
Steroids are used in lupus patients to relieve joint pain and other symptoms of the disorder.
Cytoxan, approved in 1959, is a cancer therapy that may work to suppress the immune systems attack on the body in lupus patients. The medicine is sold generically. Imuran, an immune system suppressant developed by GlaxoSmithKline for use in kidney transplant patients, also may help lupus patients. It has been approved in generic versions since 1999.
Roche Holding AGs Genentech unit and Biogen Idec Inc.s said in March that their jointly marketed drug Rituxan failed a lupus trial. It was the second time a late-stage study of Rituxan in lupus failed.