Anavex Life Sciences Corp., a clinical-stage biopharmaceutical company, has presented promising results from its Phase IIb/III trial of Blarcamesine (ANAVEX®2-73) for early Alzheimer’s disease at the 2024 Alzheimer’s Association International Conference.
The study demonstrates that this once daily oral Alzheimer’s treatment significantly slowed clinical decline in patients with early-stage Alzheimer’s disease.
The trial evaluated Blarcamesine, an orally administered drug candidate, in two dosage groups (30 mg and 50 mg) compared to placebo.
Results showed that Blarcamesine significantly reduced clinical progression by 34.6% in the 30 mg group and 38.5% in the 50 mg group over 48 weeks, as measured by the primary cognitive endpoint ADAS-Cog13.
Dr. Marwan Noel Sabbagh, Professor of Neurology at Barrow Neurological Institute and Chairman of the Scientific Advisory Board, presented the data. He emphasized the excitement surrounding these results, particularly noting the objective slowing of neurodegeneration markers.
Anavex Life Sciences’ Blarcamesine demonstrated efficacy on multiple fronts:
- Cognitive Function: The primary endpoint ADAS-Cog13 showed statistically significant improvement in both dosage groups compared to placebo at 48 weeks.
- Clinical Decline: The key secondary endpoint CDR-SB, which measures clinical decline, also showed significant improvement in both dosage groups.
- Brain Atrophy: Blarcamesine significantly slowed brain atrophy in key regions, including a 37.6% reduction in whole brain atrophy, 63.5% in total grey matter, and 25.1% in lateral ventricles.
- Biomarkers: The study reported benefits on amyloid-beta and brain volume, two underlying pathological hallmarks of Alzheimer’s disease.
The safety profile of Blarcamesine appears favorable, with no reported neuroimaging adverse events such as Amyloid-related imaging abnormalities (ARIA) or hemorrhage. The most common treatment-emergent adverse event was dizziness, which was mostly mild to moderate and transient.
Dr. Juan Carlos Lopez-Talavera, Head of Research and Development at Anavex Life Sciences, highlighted the company’s precision medicine approach. He noted that blarcamesine’s mechanism of action, which improves autophagy (a key clearance mechanism for protein aggregates), uniquely positions the company to develop innovative solutions for Alzheimer’s disease.
The potential impact of this once-daily oral Alzheimer’s treatment extends beyond its clinical efficacy. As a small molecule that can be administered orally, Blarcamesine could offer several advantages over existing therapies:
- Convenience: Once-daily oral administration could improve patient compliance and reduce the burden on healthcare systems.
- Accessibility: The oral route of administration may make the treatment more accessible to a broader patient population.
- Safety Profile: The absence of neuroimaging adverse events suggests that routine MRI monitoring may not be necessary, potentially simplifying treatment protocols.
- Complementary Approach: Given its differentiated mechanism of action, Blarcamesine could potentially be used in combination with other Alzheimer’s treatments, such as anti-beta amyloid monoclonal antibodies.
Anavex Life Sciences plans to submit a regulatory application to the European Medicines Agency (EMA) in Q4 2024. This move could potentially bring this novel treatment option closer to patients in Europe.
It’s important to note that while these results are promising, Blarcamesine is still an investigational drug. Further research and regulatory approvals will be necessary before it can be made available to patients.
The development of Blarcamesine represents a significant step forward in Alzheimer’s disease research. As an orally administered, once-daily treatment that targets multiple aspects of the disease process, it offers a potentially transformative approach to managing early Alzheimer’s disease.
Anavex Life Sciences’ innovative approach to Alzheimer’s treatment, focusing on upstream processes like autophagy, could open new avenues for addressing this complex and devastating disease.
As research continues and regulatory processes unfold, the medical community and patients alike will be watching closely to see if this once-daily oral Alzheimer’s treatment can deliver on its early promise.
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Disclaimer: The information provided above is for informational purposes only and does not constitute medical advice. Before starting or discontinuing any medication, including investigational treatments like Blarcamesine, it is essential to consult with your healthcare provider.
Only a licensed physician can provide the appropriate diagnosis and treatment options based on your medical condition.