Biogen Idec’s ALS Drug Fails Phase III Trial

1/3/13Follow @cathyarnst

A lot of patients’ hopes were dashed today with the news that Biogen Idec’s (NASDAQ: BIIB) closely watched drug dexpramipexole for amyotrophic lateral sclerosis (ALS) failed in a late-stage clinical trial. The Weston, MA-based company said it will discontinue further development of the once-promising drug.

Biogen said the drug neither slowed the loss of muscle function nor prolonged the lives of patients with ALS, often called Lou Gehrig’s disease. Nor did it show any efficacy in secondary endpoints of the clinical trial, or work in any sub-group of patients—about a big a failure as a company can have with a Phase III trial.

Biogen’s stock dropped as much as 8 percent in early trading, and by mid-day was down 2.07 percent to $147.93 a share.

“As a physician who has treated people with ALS, I hoped with all my heart for a different outcome,” Douglas Kerr, Director of Neurodegeneration Clinical Research at Biogen, said in the press release. “While these results were not what we expected, we hope these data will provide a foundation for future ALS research.” The double blind, randomized trial, named EMPOWER, enrolled 943 patients in 11 countries and started in March 2011.

The ALS community knew that the EMPOWER was risky, given that ALS drugs have failed in more than 20 clinical trials, and that a Phase II study of dexpramipexole reported in Nature in late 2011 showed mixed results. At the January 2012 J.P. Morgan Conference in San Francisco, Biogen CEO George Scangos was reported as saying that the drug was “far from a long shot,” but some analysts and medical experts were still skeptical.

There are about 30,000 Americans with ALS, which attacks nerve cells that control muscle movement and typically results in death within a few years of diagnosis. The cause is not known and there is only one mildly effective drug to ease symptoms, riluzole (Rilutek), made by Sanofi (NYSE: SNY).

Biogen licensed dexpramipexole in August 2010 from Knopp Biosciences, based in Pittsburgh, for $80 million in upfront payments and up to $265 million in milestones. The Massachusetts company said it will continue to search for ALS drugs, noting that it has several other research programs underway for the disease. In December Biogen announced a research collaboration with Duke University and HudsonAlpha Institute to sequence the genomes of as many as 1,000 people with ALS over the next five years.

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