Biogen Idec has announced that dexpramipexole, an experimental drug for the treatment of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease, did not pass the Phase 3 clinical trial.
The drug was not able to slow the loss of muscular function or lengthen the lifespan of patients with the condition. Therefore, Biogen has decided to cease experimentation on this therapy.
ALS, a progressive neurodegenerative disorder, affects an estimated 30,000 people in the United States. The disease attacks the nerves that are responsible for controlling muscles, which normally results in paralysis and death after just a few years.
Douglas E. Williams, Ph.D., Executive Vice President of Research and Development at Biogen Idec, stated:
“We share the disappointment of members of the ALS community, who had hoped that dexpramipexole would offer a meaningful new treatment option. 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 company plans to explain the full results at a medical conference in the near future.
There were 943 patients with ALS involved in EMPOWER, which was a “randomized, double-blind, placebo-controlled Phase 3 trial”, at 81 locations in 11 countries. Half the participants took the drug dexpramipexole and the other half received a placebo.
A joint rank examination of survival and function, referred to as the Combined Assessment of Function and Survival (CAFS), was the main endpoint. The researchers also individually analyzed functional, survival and respiratory decline.
Unfortunately, the primary endpoint was not met and there was no improvement seen in the individual elements of function or survival.
Douglas Kerr, M.D., Ph.D., Director of Neurodegeneration Clinical Research at Biogen Idec, said:
“As a physician who has treated people with ALS, I hoped with all my heart for a different outcome. While these results were not what we expected, we hope these data will provide a foundation for future ALS research.”
Biogen Idec is currently conducting a study over the next 5 years with Duke University and HudsonAlpha Institute to sequence the genomes of over 1,000 patients with the disease. The experts hope to gain further knowledge on the genetic causes of ALS. The team will work with some of the best researchers around the world who have experience with the disease and its linked genes. Previous research from 2011 discovered a mutation on the gene SIGMAR1 associated with the development of juvenile ALS.
A research consortium was just developed by Biogen along with with many important academic research centers in order to discover novel methods to treat ALS.
The company also contributed to the University of Massachusetts Medical School ALS Champion Fund. The knowledge of the condition will expand as a result of the funding, while supporting research into possible therapies for ALS and other neurodegenerative diseases.
Written by Sarah Glynn