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Trial Fails to Show Biogen Idec ALS Drug Efficacy

Brain silhouettes (Research.gov)

(Research.gov)

Biogen Idec, a biotechnology company in Weston, Massachusetts, reported the clinical trial of a drug to treat amyotrophic lateral sclerosis (ALS) did not meet its main objectives. Based on these results, the company decided to stop further development of dexpramipexole, the drug tested in the trial.

ALS, also known as Lou Gehrig’s disease, is a rapidly progressive neurological disease, usually fatal, that attacks the nerve cells responsible for controlling voluntary muscles, characterized by the gradual degeneration and death of motor neurons. National Institute of Neurological Disorders and Stroke says ALS is one of the most common neuromuscular diseases worldwide, and people of all races and ethnic backgrounds are affected. As many as 20,000 to 30,000 people in the U.S. have ALS, says the agency, and an estimated 5,000 people in the U.S. are diagnosed with the disease each year.

The trial, a phase 3 randomized study of the drug’s safety and effectiveness against ALS, enrolled 943 patients at 81 sites in 11 countries, who received either dexpramipexole (150 milligram tablets, twice a day) or a placebo. The study’s main indicator of effectiveness is a joint ranking measure on an index called Combined Assessment of Function and Survival. The investigators also recorded individual indicators of functional decline, survival and respiratory decline, which did not show improvement for patients receiving dexpramipexole versus the placebo.

“As a physician who has treated people with ALS, I hoped with all my heart for a different outcome,” noted Douglas Kerr, Biogen Idec’s neurodegeneration research director. “While these results were not what we expected, we hope these data will provide a foundation for future ALS research.”

Biogen Idec says it will discontinue development of dexpramipexole as an ALS therapy. The company says however, it will continue research into other ALS treatments. In December, Biogen Idec announced a collaboration with researchers from Yale, Harvard, Columbia, and Rockefeller universities to identify new drug targets for ALS, a $10 million, three-year project. In July 2012, the company began a project with labs at Duke University and HudsonAlpha Institute in Huntsville, Alabama to sequence the genomes of up to 1,000 patients with ALS over five years.

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Hat tip: MedCity News

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