Daily Alzheimer’s pill slows brain shrinkage and improves cognition – study
An Alzheimer’s pill was linked to slower brain shrinkage and improved cognition over nearly three years in early disease, a new analysis suggests.
The benefits seen in people with early Alzheimer’s disease after a year of treatment with blarcamesine were sustained over nearly three years, according to new analysis from Anavex Life Sciences.
Patients treated with the investigational oral medication in a Phase 2b/3 study had less loss of brain volume, a sign of nerve cell damage, along with improvements in cognition, daily function and disease severity, one-year data showed.
A new analysis from that trial and its long-term extension found those trends continued, and patients also experienced slower disease progression over three years. The results were presented at the 2026 International Conference on Alzheimer’s and Parkinson’s Diseases in Copenhagen and online.
Christopher U. Missling, president and chief executive of Anavex, said: “These results indicate that blarcamesine’s clinical effects are biologically coherent with MRI-based measures of neuroprotection. The consistent relation between structural preservation of brain volume and functional outcomes further drives our dedication to developing a novel disease-modifying Alzheimer’s treatment with our oral blarcamesine.”
Alzheimer’s is caused by the build-up of toxic clumps of misfolded proteins in brain cells, which disrupt their function and eventually lead to their death. As these cells are lost over time, connections between them break down, leading to symptoms including brain shrinkage and impaired cognitive function.
Given as a once-daily oral capsule, blarcamesine is designed to enhance autophagy, a recycling process cells use to remove unneeded or toxic proteins, including those involved in Alzheimer’s. By doing so, the therapy aims to protect brain cells and slow disease progression.
The global Phase 2b/3 trial evaluated blarcamesine in 508 patients aged 60 to 85 with mild cognitive impairment due to Alzheimer’s, an early stage of memory and thinking problems that is more serious than normal ageing but not as severe as dementia. Participants were randomly assigned to receive daily doses of 30mg or 50mg of blarcamesine or a placebo for 48 weeks.
Results showed that both doses significantly slowed cognitive decline, as measured by the Alzheimer’s Disease Assessment Scale-Cognition, and cognitive and functional worsening, as assessed with the Clinical Dementia Rating Scale Sum of Boxes.
The therapy also significantly reduced brain shrinkage in several key regions. According to updated data, these included the whole brain by 37.7 per cent, total grey matter by 63.5 per cent and the temporal lobe by 69.2 per cent. Grey matter helps process information, while the temporal lobe plays an important part in memory and language.
After completing the trial, participants could enter an open-label extension study in which all patients received blarcamesine and were monitored for long-term outcomes.
After nearly four years, patients who started treatment earlier maintained significantly better cognitive scores on the Alzheimer’s Disease Assessment Scale-Cognition than those who initially received a placebo. Similar benefits were seen in daily functioning, as measured by the Alzheimer’s Disease Cooperative Study-Activities of Daily Living scale. Overall, the therapy was well tolerated, with most side effects mild to moderate. The most common drug-related side effect was dizziness, which did not typically last long.
The new analyses from these trials showed that reductions in brain atrophy across all three key regions after about one year of treatment with blarcamesine were consistently associated with improvements in cognition, daily functioning and disease severity.
In a subgroup of patients known as ABCLEAR3, whose genetic profile is associated with an enhanced response to blarcamesine, the association between brain volume changes and cognition was about 78 per cent stronger than in the overall study population.
Over nearly three years of follow-up, treatment with blarcamesine delayed disease progression by 77 weeks, nearly 18 months, compared with a matched external control group from the Alzheimer’s Disease Neuroimaging Initiative, a large research database tracking people with Alzheimer’s who were not receiving the therapy.
Timo Grimmer, a member of the Anavex scientific advisory board and national coordinating investigator for the study, who presented the data at the conference, said: “The patient-friendly oral administration, the manageable side effects, and the clinical efficacy, particularly in the genetically defined ABCLEAR3 population, make blarcamesine, in conjunction with the associated biomarker signal, a promising drug candidate for patients with early-stage Alzheimer’s disease.”
Grimmer said researchers believe “these new results will contribute to the growing body of scientific data demonstrating the long-term beneficial effect of blarcamesine in early Alzheimer’s disease.”
Anavex is also exploring blarcamesine’s potential in other neurological diseases, including Parkinson’s, Rett syndrome and fragile X syndrome.
