SWITZERLAND — Roche Holding’s much-anticipated Alzheimer’s disease drug failed in two large studies, a new disappointment in a research field riddled with failures.
The experimental drug, gantenerumab failed to slow patients’ clinical decline in people with early Alzheimer’s disease, according to the Swiss pharmaceutical company.
Additionally, its removal of beta-amyloid, a protein that builds up to form plaques in the brains of people with Alzheimer’s disease, was lower than expected. Neither study met its primary research goal.
The setback will be a new challenge for Roche’s head of diagnostics, Thomas Schinecker, who will be promoted in March.
He will succeed Severin Schwan, the CEO who has led a successful campaign to diversify Roche’s traditional focus on cancer.
A long list of study failures has plagued the quest to develop an Alzheimer’s drug that targets beta-amyloid or other molecules. However, researchers claim that amyloid is only one component of Alzheimer’s disease.
On a positive turn of events, Biogen scored a surprise trial success with an experimental Alzheimer’s drug co-developed with Eisai in September, restoring confidence in the beta-amyloid approach among industry executives and researchers.
Biogen and Eisai reported that their drug candidate lecanemab slowed progression of the brain-wasting disease by 27% compared to a placebo in a large trial of patients in the early stages of Alzheimer’s.
Among other differences in the molecules and trial designs, the Swiss company’s compound was primarily targeting larger amyloid structures, whereas Biogen’s lecanemab was primarily targeting earlier stages of amyloid build-up.
Roche released only the main findings of the trials. It intends to present detailed data on Nov. 30 at the Clinical Trials on Alzheimer’s Disease conference in San Francisco.
The FDA controversially approved aducanumab, another monoclonal antibody developed by Biogen, to treat Alzheimer’s disease last year despite no clear evidence of cognitive benefit.
Two incomplete phase III trials showed that the drug could clear amyloid from the brain, but only one subset of participants experienced a slowing in cognitive decline.
Eli Lilly expects to release results for its candidate, donanemab, next year.
According to the World Health Organization, Alzheimer’s disease affects the majority of the 55 million people worldwide who suffer from dementia. Dementia is expected to affect 78 million people by 2030.
Despite the large number of people affected worldwide, there is no cure. While current treatments manage symptoms, they offer no hope of recovery.
Part of the difficulty in finding dementia treatments stems from the fact that dementia is a complex health problem with more than 50 underlying causes.
Dementia can be thought of as an umbrella term for a variety of conditions that cause parts of the brain to deteriorate gradually.
The majority of current drug treatments are aimed at the pathology of Alzheimer’s disease, the most common form of dementia.
Only 0.1% of antibodies circulating in the bloodstream are thought to enter the brain, which includes therapeutic antibodies currently used in clinical trials.