By Thomas Gryta
Of DOW JONES NEWSWIRES
NEW YORK (Dow Jones)
The success of Benlysta, being co-developed by the companies, has transformed the Rockville, Md.,-based Human Genome, which has seen its stock soar from 45 cents in March to Friday's close of $18.69 and a market value exceeding $2.5 billion. The study confirms July's surprise success and positions the companies to file for regulatory approval, which could come as soon as late 2010, with analysts projecting annual sales eventually exceeding $1 billion a year.
Despite Human Genome's recent run, many have projected that the outcome of the trial would lead to another significant move in the stock, something that has led to heavy activity in the options markets as well.
Many analysts expressed confidence in the second study, a sharp contrast from prior to the first study, but fear of past failures of lupus treatments seem to be keeping investors hesitant.
The interest in the stock is being driven by Benlysta's success, but also of the continued speculation that GlaxoSmithKline will acquire the company in order have full rights to the drug.
Lupus occurs when the body attacks itself, causing inflammation and tissue damage virtually anywhere in the body, making it difficult to treat and a challenge to design clinical trials.
The Lupus Foundation of America estimates that 1.5 million Americans have the disease, although Human Genome has estimated that 300,000 to 350,000 patients in the U.S. would benefit from a treatment like Benlysta.
Physicians traditionally use different treatments including anti-inflammatory drugs, steroids, anti-malarials and immunosuppressants. Numerous efforts to develop new therapies has led to a string of disappointments.
Like the first trial, the main goal of the study, designed along with the Food and Drug Administration, was a never-used combination of several disease-activity measures, including the drug's effect on flares and symptom severity.
The study showed the success in patients at a higher dose, based on a patient's weight, when used with standard care, in comparison to the placebo group that received standard care alone.
According to those measures, Benlysta showed that 43.2% of patients responded to the drug at the higher dose and 40.6% responded at the lower dose, as compared to a 33.8% response rate from placebo.
Despite the results, the lower dose group did not achieve statistically significant improvement. In the first study, both dose groups met that goal.
In the study, Benlysta had a similar rate of patient discontinuations caused by side effects across all treatment groups, with overall adverse event rates comparable to placebo.
As planned, the 826-patient study will continue for 24 more weeks and the companies hope to present the full details of the data in the first half of 2010.
The two Benlysta trials were virtually identical, with the key difference being patient location, a point that has worried some on Wall Street. The first trial enrolled patients in Asia, Latin America and Eastern Europe, while the second drew from the U.S. and Western Europe.
The concern is that differences in genetics and previous treatments among the groups would add an unknown variable, an uncertainty amplified by the previous lupus failures.
The company had defended the study, saying that there is no evidence that ethnicity or previous treatments cause patients to react differently, citing statistical measures that show Benlysta's strong and sustained effectiveness in the first year-long trial.
Benlysta blocks the activity of a naturally occurring protein required in 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.
-Thomas Gryta; Dow Jones Newswires; 212-416-2169; thomas.gryta@dowjones.com