Via Scoop.it – ALS Lou Gehrig’s Disease
Biogen Idec Inc. Chief Executive Officer George Scangos was skeptical when he learned company researchers were pursuing a new treatment for Lou Gehrig’s disease. No one knows the cause of the illness that killed the famous baseball player more than 70 years ago, and no medicine for it has been shown to slow its advance for long. Scangos changed his mind after he took charge two years ago and reviewed data about dexpramipexole. The compound, which may slow disease progression, is now in the final stages of clinical trials required for U.S. Food and Drug Administration approval, with results expected this year. “I don’t know any disease that’s in more need of therapy than ALS,” said Scangos, whose company is the world’s largest maker of drugs for multiple sclerosis, in a telephone interview. “It’s certainly risky, but the data speaks for itself. So we made a calculated bet.” There’s one drug on the market, Paris-based Sanofi’s Rilutek, and it provides only a modest benefit in reducing ALS’s progression. Rilutek has U.S. sales of about $50 million, according to Eric Schmidt, an analyst with Cowen & Co. in New York. The market for dexpramipexole, if it’s approved, may top $1 billion a year, Schmidt estimates. The company paid $80 million in cash and stock for a licensing deal in August 2010 with closely held Knopp Biosciences, which first developed the drug, and will pay an additional $265 million if certain regulatory and sales goals are met. The compound looked promising enough in earlier trials by Knopp to attract the attention of Al Sandrock, Weston, Massachusetts-based Biogen’s head of neurology research and a former physician who worked with ALS patients. It’s designed to improve functioning of the mitochondria, the energy producers in cells, and to provide protection to neurons under stress.
Via www.businessweek.com
ALS Research News 