By Cyndi Root
Biogen Idec captured the spotlight recently, announcing that it was skipping Phase 2 and moving its experimental drug for Alzheimer’s disease (AD) into Phase 3 clinical trials. The company announced the move at the Deutsche Bank BioFEST conference, stating that BIIB037 showed both a reduction in amyloid plaques and an increase in cognition. According to CNBC, Douglas E. Williams, Biogen’s research chief, said that the company is planning to move “aggressively” and advance to Phase 3. The announcement created a stir due to the lack of effective Alzheimer’s treatments on the market and in the pipeline.
BIIB037 is thought to bind to amyloid plaques, thereby eliminating the toxic substances that form in the brains of patients with AD. Alzforum, a community site, has published details on the agent, including its profile, mechanism of action, and study results. BIIB037 is a monoclonal antibody developed by Neurimmune in Schlieren, Switzerland. Biogen has been collaborating with the company since 2007. In March 2014, Eisai and Biogen Idec entered into a partnership agreement, wherein Eisai acquired an option to jointly develop the agent.
A Phase 1 study of 56 people with mild to moderate AD were assessed periodically for two years. With MRI imaging studies, investigators documented amyloid-related imaging abnormalities (ARIAs) in the safety and pharmacokinetics study. Since side effects were mild or moderate, the safety monitoring board authorized a dose increase from 30 to 60 mg/kg. Dosing showed little variability among patients and no plasma spikes.
The Phase 1b study began in 2012 and is titled, “A Randomized, Double-Blinded, Placebo-Controlled Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB037 in Subjects With Prodromal or Mild Alzheimer's Disease.” The primary outcome measure for the 194 people is the number of participants with adverse events. Interim data reported in December 2014 suggest that BIIB037 is effective in reducing amyloid plaques and shows a cognitive benefit.
Are Amyloid Plaques the Wrong Target?
In a review of the Alzheimer’s pipeline, a study suggests that given the magnitude of the disease, clinical trials are scarce and many agents have failed. A study by researchers at Georgetown University Medical Center in Washington has provided a clue to the failures of amyloid plaque-targeted treatments. They say that amyloid plaques are not the trouble, but rather, tau protein dysfunction causes neurodegeneration.