Human Genome Sciences Inc. (HGSI) and GlaxoSmithKline Plc (GSK, GSK.LN) are seeking to take a significant step next week in getting regulatory approval for the first new treatment to fight the autoimmune disease lupus in more than 50 years.

A U.S. Food and Drug Administration panel will decide Tuesday whether the companies' drug Benlysta should be approved by the agency in early December.

The drug showed surprise success in two late-stage studies last year--transforming Human Genome's stock market value--as analysts project annual sales eventually exceeding $1 billion a year. The drug is widely expected to get FDA approval because of its strong data and the lack of alternatives for patients.

"It is a beacon of hope. There hasn't been, in the history of the disease, a drug aimed specifically at lupus," said Sandra Raymond, president of the Lupus Foundation of America.

Lupus, which primarily strikes women of childbearing age, occurs when the body attacks itself, causing inflammation and tissue damage virtually anywhere in the body. The Lupus Foundation of America estimates that 1.5 million Americans have the disease, although Human Genome initially plans to target about 200,000 patients with moderate or severe forms of the disease.

Because the disease could cause many symptoms in different patients, physicians traditionally use different treatments, including anti-inflammatory drugs, steroids, anti-malarials and immunosuppressants.

Leerink Swann projects that world-wide sales of the drug will exceed $5 billion in 2020. The drug's success has produced consistent speculation that Glaxo, or another large drug maker, would acquire the company.

Shares of Human Genome closed at $26.48 Thursday; they had traded below $1 just months prior to the initial Phase III data on the drug last year. The stock ended up 8.8% ahead of Friday's expected release of briefing documents related to the panel's deliberations.

RBC Capital Markets recently projected that Human Genome's stock would rise above $30 on a positive recommendation from the panel, but could drop 50% to 60% if it gets a negative vote. The firm gives only a 5% to 10% chance of a rejection.

In late-stage trials, Benlysta patients showed a greater response to the drug, compared with placebo, in a combination of several disease-activity measures, including flares and symptom severity. Human Genome worked with the FDA on the studies' complex design that attempted to isolate the patient group that responded in a failed mid-stage trial.

The panel next week is expected to discuss the drug's complicated late-stage trials, along with the drug's safety profile, including the potential for an increased risk of infection and slight increase in some types of cancer. Most believe the benefits of the drug will outweigh those concerns and result in strong warnings on the drug's label, as has occurred on most rheumatoid arthritis treatments.

Benlysta is an antibody that blocks the activity of a protein required for the development of certain B cells, part of the immune system, that produce antibodies thought to play a key role in lupus and some other autoimmune diseases.

The nature of the disease makes it difficult to treat and a challenge to design clinical trials because almost every patient has different symptoms and, accordingly, gets different treatments. Numerous efforts over the years to develop new therapies have led to a string of major disappointments.

Current companies pursuing lupus research include UCB SA (UCB.BT) and Immunomedics Inc. (IMMU), Cephalon Inc. (CEPH) and ImmuPharma Plc (IMM.LN), and Astrazeneca Plc. (AZN, AZN.LN).

While some companies have drugs in development, there is hope that the clinical success of Benlysta and its potential approval will attract even more research in lupus by proving it is a viable area for major drug companies.

"It shows companies that it is possible to show a therapeutic benefit in lupus," said Chaim Putterman, chief of rheumatology at Albert Einstein College of Medicine and Montefiore Medical Center. He said he expects Benlysta to get widespread use and is hopeful that other medications in development will show similar benefits.

"It encourages other companies to begin to look at lupus as a potential disease of interest," he said.

-By Thomas Gryta, Dow Jones Newswires; 212-416-2169; thomas.gryta@dowjones.com
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