Biogen, a global biotechnology company based in Cambridge, Mass., has manufactured a drug that has successfully slowed mental function decline in patients with Alzheimer’s disease.
Alzheimer’s disease is the most common form of dementia, known to slowly destroy memory and thinking skills. Currently, the disease affects 5.2 million people across America, costing an estimated total of $226 billion in direct costs. Biogen could reap billions of dollars a year from the production of the drug if the successful results can be repeated in larger trials.
The drug, called Aducanumab, aims to counter Alzheimer’s by binding to buildups known as amyloid plaques and ridding them from the brain. Amyloid plaques are widely known to be a contributing cause to dementia; however, the extent to which the plaques contribute to dementia is controversial and unknown.
Nevertheless, this breakthrough offers hope in a new and innovative approach to curing Alzheimer’s.
“Alzheimer’s disease is a veritable graveyard of failed development efforts so a tempered approach is advised here,” Chris Raymond, an analyst with Robert W. Baird, wrote in a research note. “That said, this is obviously a huge unmet need, and as BIIB037 approaches phase 3, we think it’s plausible that an increasing premium for this opportunity has to start working its way into the stock.”
In 2012, Biogen started the study in 166 patients with Alzheimer’s disease who also tested positive for an amyloid plaque scan. In order to maximize chances of success, the drug was given in large doses (roughly 4 grams for a 150lb person) and given only to those with a positive scan for amyloid plaque in the brain.
The 166 patients were randomly assigned to be given the drug or the placebo. The drug not only slowed mental deterioration, but also reduced plaque in the brain.
“It would be kind of hard to get those results by chance,” said Dr. Rachelle S. Doody, consultant to Biogen and director of the Alzheimer’s Disease and Memory Disorders Center at Baylor College of Medicine.
One way to measure cognition is the 30-point mini-mental state exam (MMSE). Patients receiving the placebo dropped an average of 3.14 points a year, but those taking the drug dropped only an average of 0.58 points for a high dose and an average of 0.75 a medium dose in one year.
The results shattered expectations, and Biogen plans to continue onto larger clinical trials in a hope to achieve the same successful results.
“We’re going to try to replicate what we’ve seen in this relatively small study,” Biogen’s head of research and development, Doug Williams, said in a presentation at Deutsche Bank’s BioFEST conference in Boston. “Based on these results, we’re planning very aggressively to start the phase 3 program.”
However, several setbacks still remain. The drug comes with several major side effects, the most significant one being a localized swelling in the brain. This is a common side-effect in most drugs that aim to reduce amyloid plaques, but effects appear to be more significant in Aducanumab.
“We believe we have a safety window to work with,” Williams said. Biogen is continuing the study and will experiment with different doses to reduce possible side effects.
Although several more clinical trials and procedures remain, there is much hope for this new drug.
As Raymond writes, “Bottom line—too early to pop the champagne corks just yet, but definitely a positive development.”