Dexpramipexole Fails in ALS

Susan Jeffrey

January 04, 2013

Biogen Idec reports that topline results of a phase 3 trial investigating dexpramipexole in patients with amyotrophic lateral sclerosis (ALS) failed to show a benefit on primary or secondary outcomes.

The trial, called EMPOWER and including 943 patients with ALS, failed to meet the primary outcome, a joint rank analysis of function and survival called the Combined Assessment of Function and Survival. Other endpoints, including functional decline, survival, or respiratory decline, and subgroup analyses also failed to show efficacy with treatment, overall or for any subpopulation.

"Based on these results, Biogen Idec will discontinue development of dexpramipexole in ALS," a statement from the company said.

"We share the disappointment of members of the ALS community, who had hoped that dexpramipexole would offer a meaningful treatment option," Douglas E. Williams, PhD, executive vice president of research and development at Biogen Idec, said in the company statement.

"I am deeply saddened by this news and know how disappointing it is for the thousands of people living with ALS and their families," said Jane H. Gilbert, president and CEO of The ALS Association in a statement. "On behalf of The ALS Association, I want to assure the ALS community that we are doing everything we can to work with companies to advance treatments and bring them to patients as soon as possible."

Dexpramipexole had been given a fast-track designation from the US Food and Drug Administration (FDA). It has also been granted orphan drug status by both the FDA and the European Medicines Agency.

In their statement, the company reassures the community that they remain committed to research in the ALS space. Efforts include the recent establishment of a research collaboration with Duke University and HudsonAlpha Institute to sequence the genome of up to 1000 people with ALS over the next 5 years, collaboration in a research consortium with several leading research centers to identify new treatments, as well as "significant funds" committed to the University of Massachusetts Medical School ALS Champion fund to increase awareness and support basic and clinical science research into ALS and other neurodegenerative diseases.

"As a physician who has treated people with ALS, I hoped with all my heart for a different outcome," said Douglas Kerr, MD, PhD, 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."

Riluzole (Rilutek, sanofi-aventis) has shown modest benefits in a measure of survival time and is currently the only approved treatment for ALS.