New Lupus Drug Heads to FDA for Approval

An experimental lupus drug has just met another key goal in its journey to becoming the first approved treatment for the disease in over 50 years. The target of the new drug, known as Benlysta, is to suppress the response of the body’s immune system to lupus in an attempt to control the actions of a protein that becomes overactive in lupus patients.

Hopes were that the study data from a clinical program called Bliss-76 would confirm the optimistic results of a prior late-stage study. In actuality, the indications of the Bliss-76 data were that a 10-milligram dose of Benlysta, accompanied by therapy with steroids, resulted in improvement among 43 percent of lupus patients taking a higher dose, as well as a 40.6 percent of those taking a lower dose. This is compared to improvements observed in only 33.8 percent of patients taking a placebo.

Makers of the drug, Human Genome Sciences (HGS) based in Rockville, Maryland, and GlaxoSmithKline based in the United Kingdom, now plan to seek the regulatory approval of the U.S. Food and Drug Administration in the early months of 2010. If approved, Benlysta could be available on the market by late next year.

In a statement, HGS President and CEO H. Thomas Watkins said, “The Bliss-76 results confirm our view that Benlysta has the potential to become the first new approved drug in decades for people living with systemic lupus.” He then added, “We take great pride in the innovation and scientific rigor that has made it possible to bring Benlysta to this point.”

The Bliss-76 study was the second of two late-stage studies, and involved 865 patients who were treated and monitored over a period of one year. In October of this year, the first late-stage study, called Bliss-52, involved over 800 patients in Asia, South America and Eastern Europe, and has positive results in meeting several key goals.

Lupus is an inflammatory, autoimmune condition, in which the body’s defense system against pathogens attacks the body’s own tissue causing health issues such as rashes, mouth sores, arthritis, and kidney damage among other problems. Because the illness manifests itself differently in each patient, it is difficult to develop effective treatments against the disease. According to Watkins, “We’ve got a good chance to redefine the standard of care for patients living with lupus.”

While one recent study estimated that 322,000 Americans most likely have the most common form of lupus known as systemic lupus erythematosus, the Lupus Foundation of America estimates that about 1.5 million Americans have some form of the disease. According to Sandra C. Raymond, the organization's president and chief executive, “Individuals with lupus and their families have waited more than 50 years to hear that it is possible to develop therapies that control the disease.” She went on to say, “We believe that this is a significant first step in developing the full arsenal of therapies and personalized treatment lupus requires.”

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A new experimental drug holds promise for Lupus treatment

A new experimental drug, Benlysta, being developed by Human Genome Sciences holds promise as a treatment for Lupus.

Nearly 1.5 million Americans suffer from the autoimmune disorder that can cause chronic joint pain, fatigue, even organ damage.

Lupus strikes women more than men and can be confounding to treat because it often impacts 50 people in 50 different ways.

The last drug approved for the treatment of Lupus by the FDA was in the 1950s.

"There wasn't any research for Lupus just because it was considered a rare disease and therefore it wouldn't be of financial benefit to commercial sponsors to test drugs," says Dr Ellen Ginzler, Chief of Rheumatology, Suny Downstate Medical Center.

Benlysta is now giving new hopes to doctors and patients alike.

"It is an antibody to one of the proteins that regulates inflammation. It is an antibody to something called B cell lymphocyte stimulator (BLyS)," explains Ginzler.

BLyS is a naturally occurring protein which is normally involved in the survival of antibody producing B cells. When present in excess it can lead to the development of autoimmune diseases like lupus, where the body's immune system starts to attack its own tissues and organs.

According to Human Genome Sciences, a recent study involving 850 patients found half of the patients on Benlysta experience a reduction in their symptoms, including fatigue, joint pain and skin rashes.

A lot of questions about the experimental treatment remain to be answered warns Lupus specialist Tammy Otset of UC Medical Center.

"It's not a magic bullet. It's not going to cure Lupus but hopefully it will be something better in our armor."

Hopes are running high that Benlysta will become first FDA-approved treatment for lupus in 50 years.

Source : www.sawfnews.com

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Lupus drug Benlysta reduces symptoms in late stage testing

Human Genome Sciences Inc. and glaxosmithkline Plc said their experimental lupus drug reduced patients’ symptoms in a yearlong study. Benlysta is an investigational drug and the first in a new class of drugs called BLyS-specific inhibitors. No new drug for lupus has been approved by regulatory authorities in more than 50 years. Belimumab is being developed by HGS and GSK under a co-development and commercialization agreement entered into in August 2006. Lupus an autoimmune disease, is a chronic, often debilitating, and sometimes fatal illness that affects an estimated five million people worldwide.

In the double-blind, placebo-controlled, multi-center trial, known as BLISS-52, both dose levels of Benlysta(TM) plus standard of care were more effective than a placebo plus standard of care in people with serologically active systemic lupus. A total of 867 participants at 90 clinical sites in 13 countries, primarily in Asia, South America and Eastern Europe, were enrolled in the trial. Among patients who received 1 mg of Benlysta for every kilogram of body weight, the company said 51.7 percent experienced a significant improvement. Of patients who took a larger dose, 10 mg per kilogram, 57.6 percent had a significant improvement.

“This is the first drug shown to be effective in ameliorating the signs and symptoms of lupus in decades,” said Daniel J. Wallace, MD, clinical professor of medicine at the David Geffen School of Medicine at UCLA. “It represents a breakthrough for finally utilizing a methodology that enables researchers to demonstrate disease improvement. This will benefit lupus patients and their doctors.”

Study results also showed that belimumab was generally well tolerated, with adverse event rates comparable between belimumab and placebo treatment groups. Results of a second Phase 3 trial of Benlysta(TM), known as BLISS-76, are expected later this year. That trial has enrolled 826 participants at 133 clinical sites in 19 countries, primarily in North America and Europe. BLISS-52 and BLISS-76 are the largest clinical trials ever conducted in people with lupus.

Source : chattahbox.com

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Human Genome Seeks To Surprise Doubters With Lupus-Drug Data

Human Genome Sciences Inc. (HGSI) is hoping to surprise an army of doubters on Monday when it discloses late-stage clinical data on the experimental drug Benlysta in treating lupus, a notoriously hard-to-treat autoimmune disease that hasn't seen a new therapy in decades.

The Rockville, Md., drug maker, which has joined with GlaxoSmithKline PLC ( GSK), believes it has a better chance at success than previous failed therapies because it used lessons from a failed Phase II trial to help design the Phase III one. Although the company worked with the Food and Drug Administration on the trial's design, skeptics note that the trial's main goal, or endpoint, has never been used before and they have doubts about the trial's ability to succeed.

"Lupus has been a difficult disease to study, and a lot of recent drug failures have made people skeptical that anything will work," said Barry Labinger, the company's executive vice president and chief commercial officer.

Lupus occurs when the body attacks itself, causing inflammation and tissue damage virtually anywhere in the body and making it difficult to treat. Lazard Capital Markets projects that a new treatment represents a $2.9 billion annual market opportunity.

The coming data are the first of two similar trials, with the second expected in November. If this first trial fails, analysts see the stock dropping to near $1 but possibly exceeding $10 on successful data.

With the stock currently at $2.53, investors could be willing to trade a minimal investment for the chance to get huge short-term returns.

But, like many on Wall Street, Barclays analyst Jim Birchenough is advising clients to exercise caution as he calculates an imposing 85% chance of failure.

Furthermore, Human Genome could face debt issues after a failure, notes UBS analyst Maged Shenouda. The company has $397 million in debt coming due by 2012, he said, and $340 million in projected year-end cash.

Regardless of the outcome, Executive Vice President Labinger expressed confidence in the company's future, noting other products in development.

Attempts to treat Lupus have created a long list of failures, including disappointment from Rituxan, sold by Biogen Idec Inc. (BIIB) and Roche Holding AG (RHHBY), and Riquent from La Jolla Pharmaceutical Co. (LJPC), which is now winding down its operations.

More pessimism comes from Benlysta's Phase II failure that led to a complex Phase III design that attempts to isolate the patient group that responded in the previous trial.

The latest study includes only patients who have certain immune-system antibodies, which may indicate more disease activity. These patient made up about 75% of the Phase II study. Furthermore, the trial's endpoint uses a combination of several disease-activity measures that have never been used in a study before.

A retrospective analysis of the failed Phase II study shows it would have succeeded using the new endpoint, which gives Benlysta an edge that previous therapies didn't have, Labinger said.

After working with the FDA, Human Genome obtained a special protocol assessment, which is an agreement with the agency on a study's design that meets regulatory requirements for a new drug application and could lead to a drug's approval.

Although Birchenough acknowledges that the endpoint may be legitimate, he argues that there have been "few, if any" cases where a retrospective subset analysis of a failed Phase II trial led to subsequent success in a late-stage trial.

Researchers have long struggled with the design of lupus clinical trials because almost every patient has different symptoms and, accordingly, gets different treatments including anti-inflammatory drugs, steroids, anti-malarials and immunosuppressants.

This can make it difficult to isolate a population that is more likely to respond to any particular drug, and to measure a response in any group.

"No matter how far you whittle down a subpopulation, you are inevitably going to see another population develop," Birchenough said.

Source : news.morningstar.com

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Cancer Drug May Offer New Hope for Lupus Patients

The cancer drug Rituxan may offer the first new treatment approved for lupus in 50 years, suggests a small study by U.K. researchers at Imperial College London.

The 22-month study included 20 people with lupus nephritis, a severe kidney disorder caused by the disease lupus. After treatment with Rituxan, 60 percent showed significant signs of improvement, the researchers found. But the drug was not effective in people of African ancestry or in those with very low levels of albumin protein in their blood.

Rituxan targets hyperactive B cells, which contribute to kidney inflammation in people with lupus. If these study results can be repeated in larger trials, Rituxan might be approved to treat lupus, the researchers said.

"This is very welcome news to the 40 percent of lupus patients who are suffering with kidney involvement in their battle with systemic lupus erythematosus, an autoimmune disease, and who until now had very little hope of a breakthrough for lupus nephritis," Virginia T. Ladd, president and executive director of the American Autoimmune Related Diseases Association, said in an association news release.

People with lupus experience a wide range of problems, including muscle pain, extreme fatigue and inflammation of the joints, skin, major organs and central nervous system.

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