Anavex's Landmark Phase IIb/III Blarcamesine Trial Results Presented at Alzheimer's Conference
Anavex Life Sciences Corp. has announced promising results from its Phase IIb/III clinical study of blarcamesine (ANAVEX®2-73), a once-daily oral treatment for early Alzheimer's disease (AD). The data, presented at the 2024 Alzheimer's Association International Conference by Dr. Marwan Noel Sabbagh, revealed that blarcamesine significantly slowed disease progression in patients, showing a reduction in clinical decline by 38.5% and 34.6% in the 50 mg and 30 mg treatment groups, respectively, compared to a placebo.
The study utilized the ADAS-Cog13 cognitive assessment as its primary endpoint, which is a recognized measure for early AD trials. The results showed statistically significant improvements in cognitive function for both dosage groups. Although the functional co-primary endpoint, ADCS-ADL, trended positively, it did not achieve statistical significance, likely due to its design being more suited to advanced Alzheimer's stages. However, the key secondary endpoint, CDR-SB, showed significant improvements in both dosage groups.
Biomarker analysis supported these findings, with significant reductions in brain atrophy observed. Specifically, blarcamesine reduced whole brain atrophy by 37.6%, total grey matter loss by 63.5%, and expansion of lateral ventricles by 25.1%. These markers are crucial as they indicate the preservation of brain structure in AD patients.
Dr. Sabbagh emphasized the excitement around these results, highlighting blarcamesine’s potential due to its oral administration and favorable safety profile. Unlike other anti-amyloid treatments, blarcamesine did not lead to adverse neuroimaging outcomes such as hemorrhage or Amyloid-related imaging abnormalities (ARIA). This aspect could make blarcamesine more accessible and less burdensome for patients, potentially easing some complexities within the current healthcare system for AD.
In terms of safety, the study noted that common adverse events like dizziness were mostly mild to moderate and manageable. Such side effects occurred during both the titration and maintenance phases of treatment but were less frequent with slower titration and nighttime dosing.
Blarcamesine’s unique mechanism of action, which involves modulating SIGMAR1 and muscarinic receptors, provides an innovative approach to treating AD. This mechanism promotes autophagy, aiding in the clearance of protein aggregates that are characteristic of Alzheimer's pathology. Dr. Juan Carlos Lopez-Talavera, Head of Research and Development at Anavex, noted that this precision medicine approach is particularly tailored to slowing disease progression and maintaining patients' cognitive functions.
Anavex plans to submit a regulatory application for blarcamesine to the European Medicines Agency (EMA) in the fourth quarter of 2024. If approved, blarcamesine could offer a new, convenient treatment option for early Alzheimer's patients, potentially complementing existing therapies.
The study reaffirms previous findings that blarcamesine can address the complex pathophysiology of Alzheimer's through a compensatory process involving SIGMAR1 activation. Christopher U. Missling, President and CEO of Anavex, expressed gratitude to all participants and contributors to the study, underlining the potential of blarcamesine to meet the high unmet needs of AD patients.
Data from this study will be published in a forthcoming peer-reviewed journal, and Anavex continues its commitment to developing novel therapeutics for neurodegenerative diseases.
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