Anavex’s Oral Drug Blarcamesine Demonstrates Disease-Modifying Potential in Early Alzheimer’s Disease

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Anavex’s Oral Drug Blarcamesine Demonstrates Disease-Modifying Potential in Early Alzheimer's Disease

Anavex Life Sciences Corp. (Nasdaq: AVXL) has announced results from the ANAVEX2-73-AD-004 Phase IIb/III clinical trial evaluating blarcamesine (ANAVEX2-73) for the treatment of early Alzheimer’s disease (AD). The study, which enrolled 508 participants, met its co-primary endpoints, demonstrating significant improvements in cognitive function and clinical progression.

Blarcamesine is an orally available small molecule that enhances autophagy through SIGMAR1 activation and restoration of cellular homeostasis. In the randomized, double-blind, placebo-controlled trial, participants received either blarcamesine (30 mg or 50 mg) or placebo once daily for 48 weeks. The co-primary outcomes were assessed using the Alzheimer’s Disease Assessment Scale-Cognitive Subscale (ADAS-Cog13) and the Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) scales.

The results showed that blarcamesine significantly slowed clinical progression by 36.3% at 48 weeks compared to placebo on the ADAS-Cog13 scale (p=0.008). Additionally, the secondary endpoint, the Clinical Dementia Rating-Sum of Boxes (CDR-SB), showed significant improvement with blarcamesine treatment (p=0.010). These findings were supported by biomarker data, including increased plasma Aβ42/40 ratio (p=0.048) and reduced whole brain atrophy (p=0.002) in the blarcamesine group.

The safety profile of blarcamesine was favorable, with no associated neuroimaging adverse events. The most common treatment-emergent adverse event was transient dizziness, which was mostly mild to moderate in severity.

Christopher U. Missling, Ph.D., President and Chief Executive Officer of Anavex, stated, “We are very encouraged by the results of this study, which demonstrate the potential of blarcamesine as a novel treatment for early Alzheimer’s disease. These findings, along with the positive safety profile, support the continued development of blarcamesine as a potential disease-modifying therapy for AD.”

Anavex is dedicated to developing novel therapeutics for neurodegenerative, neurodevelopmental, and neuropsychiatric disorders, including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, and other central nervous system diseases. The company’s lead drug candidate, blarcamesine, has shown promise in multiple clinical trials for various indications.

The successful completion of the ANAVEX2-73-AD-004 trial represents a significant milestone for Anavex and provides hope for patients and families affected by Alzheimer’s disease. With no currently approved oral disease-modifying treatments for AD, blarcamesine could potentially fill a critical unmet need in the management of this devastating condition.
Anavex plans to submit these findings to regulatory authorities and engage in discussions regarding the next steps in the development of blarcamesine for Alzheimer’s disease. The company remains committed to advancing its pipeline of innovative therapies to improve the lives of patients with neurological disorders.