Biogen Idec will discontinue work on its experimental drug dexpramipexole to treat the nerve cell disease amyotrophic lateral sclerosis after a Phase III trial failed to be both efficacious and prolong survival.   

Even among subpopulations, the drug failed to demonstrate efficacy, the company said. Dexpramipexole, which had been in-licensed from Knopp Biosciences, had been touted as a breakthrough following positive mid-stage results.

“We share the disappointment of members of the ALS community, who had hoped that dexpramipexole would offer a meaningful new treatment option,” said Douglas Williams, executive vice president of research and development at Biogen Idec. “Nevertheless, the Empower trial represents a significant contribution to ALS research, and Biogen Idec is committed to advancing ALS science. We continue to work with researchers around the world to understand the causes of ALS and find potential treatments for people with ALS.”

The Empower Phase III trial of 943 people with ALS, also known as Lou Gehrig’s disease, sought a primary endpoint of joint rank analysis of function and survival. In addition, the trial individually evaluated functional decline, survival and respiratory decline, among other measures. The only other available treatment for the disease is Sanofi’s Rilutek (riluzole), though its benefits have been described as modest.  

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

Biogen Idec has several programmes underway in ALS, which causes gradual paralysis through the degeneration of nerves that control muscles. These programmes include a research collaboration with Duke University and HudsonAlpha Institute to sequence the genomes of up to 1,000 people with ALS over the next five years, and a research collaboration with several academic research centres to identify new approaches to treating ALS. The company has also committed funds to the University of Massachusetts Medical School ALS Champion Fund to drive awareness and support basic research into ALS.  

Detailed results of the Empower trial will be presented at a future medical conference.