Biogen Drug for Lou Gehrig’s Disease Flops in Trial

In a blow to both a high-flying biotech and the relatively few patients with Lou Gehrig’s disease, a Phase III clinical trial being run by Biogen Idec (BBIB) showed that its dexpramipexole for treating amyotrophic lateral sclerosis failed to demonstrate any efficacy in improving either function or survival.

As a result, further study is being discontinued, a move that also disappointed investors, who sent Biogen shares down 6 percent in early trading. The failure comes almost exactly a year after Biogen CEO George Scangos told an investor conference the drug “is far from a long shot.” His declaration helped propel Biogen stock more than 30 percent since then.

Biogen shares immediately fell on the news and then mostly recovered.

BIIB Chart

BIIB data by YCharts

ALS, which is also named for the famous New York Yankees baseball player Lou Gehrig, affects roughly 30,000 people each year in the US, but there is no cure. The disease causes nerve damage that leads to muscle weakening and, eventually, respiratory failure. About five of every 100,000 people worldwide are affected.

For this reason, there was considerable interest in the Biogen drug, especially after Phase II trials showed that, after just 12 weeks, patients taking the highest dose experienced slowed progression of the disease by roughly 35 percent compared with those given a placebo.

To read the remainder of this article, go to Pharmalot.

Ed Silverman, a contributing editor of YCharts, is the founder and editor of Pharmalot. He previously reported on the pharmaceutical industry and other business topics for the Star-Ledger of New Jersey, New York Newsday and Investor’s Business Daily. He can be reached at



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