Eisai Co Ltd and Biogen Inc on Tuesday said their experimental Alzheimer's drug significantly slowed the cognitive and functional decline in a large trial of patients in the early stages of the disease, marking a rare win in a field littered with failed drugs.
The drug, lecanemab, slowed the progress of the cognitive decline disease by 27% compared with a placebo, meeting the study's main goal, and potentially offering hope for patients and their families desperate for an effective treatment.
It's not a huge effect, but it's a positive effect," said Ronald Petersen, director of the Mayo Clinic Alzheimer's Disease Research Center in Rochester, Minnesota.
Eisai, leader of the 50-50 partnership's lecanemab program, is seeking FDA approval under an accelerated pathway, with a decision expected in early January.
The company said it will seek approval in Japan and Europe this year.
The results of the 1,800-patient trial support a long-held hypothesis that removing sticky deposits of a protein called amyloid beta from the brains of people with early-onset Alzheimer's could delay the progression of the debilitating disease, Isai said.
Some scientists have challenged the so-called amyloid theory, particularly in the US in 2021. After the Food and Drug Administration gave controversial approval to AduHelm, based on its ability to remove plaque rather than evidence that it helped slow cognitive decline. The decision came after the FDA's own panel of outside experts advised against approval.
Patient advocacy groups applauded the news of the positive lecanemab trial results.
"This is important because it shows that each of these drugs is different ... I expect the FDA to approve the drug in January," said George Vradenburg, chairman of USAAgainst Alzheimer's.
The Phase III trial evaluated the drug's ability to reduce cognitive and functional decline based on the Clinical Dementia Rating-Sum of Boxes (CDR-SB), a numerical scale used to quantify the severity of dementia in patients in areas such as memory, orientation, judgment and problem solving and personal care.
According to the Alzheimer's Association, the number of Americans with Alzheimer's is expected to rise to 13 million by 2050. Globally, without effective treatment, this figure will rise to 139 million by 2050, according to Alzheimer's Disease International.