Weston, Mass.-based Biogen Idec has decided to discontinue development of dexpramipexole in amyotrophic lateral sclerosis (ALS), after top-line results of EMPOWER, its phase III trial, did not meet its primary endpoint, a joint rank analysis of function and survival, and no efficacy was seen in the individual components of function or survival.
The trial also failed to show efficacy in its key secondary endpoints. Additional analyses of multiple subpopulations failed to demonstrate any efficacy among these groups.
"We share the disappointment of members of the ALS community, who had hoped that dexpramipexole would offer a meaningful new treatment option,” said Douglas E. Williams, Ph.D., executive vice president of R&D, 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 company intends to present detailed results at a future medical conference.
EMPOWER was a randomized, double-blind, placebo-controlled phase III trial which enrolled 943 people with ALS at 81 sites in 11 countries. Patients were randomized on a one-to-one basis to receive either dexpramipexole or placebo. The primary endpoint was a joint rank analysis of function and survival, known as the Combined Assessment of Function and Survival (CAFS). In addition to CAFS, the trial individually evaluated functional decline, survival and respiratory decline, among other measures.
“As a physician who has treated people with ALS, I hoped with all my heart for a different outcome,” said Douglas Kerr, M.D., Ph.D., 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 programs underway in ALS. The company recently entered into 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 in an effort to gain a deeper understanding about the fundamental genetic causes of the disease. Duke and HudsonAlpha will work with several world-class researchers who have deep expertise and experience with ALS and the genes associated with the disease.
Biogen Idec also recently created a research consortium in collaboration with several leading academic research centers to identify new approaches to treating ALS. Each of the centers involved in this consortium brings different scientific and technical expertise and a shared goal of improving the understanding of ALS from a basic science perspective.
In addition, Biogen Idec has committed significant funds to the University of Massachusetts Medical School ALS Champion Fund. The funding will drive awareness of ALS and support basic and clinical science research into potential treatments for ALS and other neurodegenerative diseases.