Biogen Idec CEO George Scangos gambled big that dexpramipexole could buck the long losing streak of experimental drugs for ALS and go on to score positive results in Phase III. But today the company had to acknowledge that Scangos--in his first big setback after taking the helm--lost that wager. Despite promising mid-stage results, dexpramipexole flunked out of the late-stage study with no sign of efficacy and is now being relegated to the scrap heap of failed therapies.
Its stock ($BIIB) quickly lost $8.25 a share, or 5.5%.
Scangos--credited with turning around the floundering biotech--had decided to continue work on dexpramipexole following a pipeline review shortly after he took over. Biogen had in-licensed the drug from Knopp Biosciences. And as he told analysts at JP Morgan a year ago, he felt that the program had bright prospects as a potential blockbuster. Investigators signed up 943 ALS patients at 81 sites, but they failed to respond as the treatment failed the primary endpoint as well as key secondary endpoints.
"Nevertheless, the EMPOWER trial represents a significant contribution to ALS research, and Biogen Idec is committed to advancing ALS science," noted Biogen R&D chief Doug Williams. "We continue to work with researchers around the world to understand the causes of ALS and find potential treatments for people with ALS."
Biogen has made a big commitment to the ALS field which will continue despite the Phase III flop. The company is involved in a collaboration with Duke University and HudsonAlpha Institute to sequence the genomes of 1,000 ALS patients and recently established a research consortium of academic groups to further study the disease as it seeks to blaze new pathways in the clinic.
"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."
- here's the press release
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