In December 2023, Anavex Life Sciences Corp., a biopharmaceutical company specializing in treatments for neurodegenerative and neurodevelopmental diseases, announced a significant milestone for its Alzheimer’s disease drug, blarcamesine. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed that the drug is eligible for submission under the European Medicines Agency’s centralized procedure for a Union Marketing Authorisation.
This eligibility is crucial because it allows Anavex to submit a single Marketing Authorisation Application (MAA) that, once approved, would enable blarcamesine to be marketed across all European Union member states.
Background and Mechanism of Action
Blarcamesine, also known by its investigational code ANAVEX 2-73, is a small molecule drug under development for neurodegenerative and neurodevelopmental disorders. Originating from the research of Anavex Life Sciences, blarcamesine is designed to target the sigma-1 receptor (S1R) and muscarinic receptors in the central nervous system. S1R plays a crucial role in cellular homeostasis and neuroprotection, and its activation is believed to modulate key pathways involved in neurodegeneration, mitochondrial function and the cellular stress response.
Blarcamesine’s mechanism of action is centered on the modulation of these receptors to restore cellular balance, reduce oxidative stress, and enhance synaptic plasticity. These effects make it a promising candidate for treating conditions such as Alzheimer’s disease, Parkinson’s disease and Rett syndrome. In preclinical and clinical trials, blarcamesine has shown potential to improve cognitive function, mitigate inflammation and reduce the accumulation of amyloid-beta plaques—hallmarks of Alzheimer’s pathology.
One of the distinguishing features of blarcamesine is its oral bioavailability and favorable safety profile, which has been demonstrated across multiple studies. In Alzheimer’s clinical trials, blarcamesine has been shown to improve cognitive scores in some patients while stabilizing disease progression, suggesting it may modify the underlying disease process rather than simply treating symptoms.
Currently, blarcamesine is in various stages of clinical development for Alzheimer’s disease, Parkinson’s disease, and Rett syndrome. While further studies are needed to confirm its efficacy and safety in larger populations, early results indicate its potential to become a novel therapeutic agent for managing complex neurological diseases by targeting the S1R pathway.
Scalable and Easily Administered
Anavex’s President and CEO, Dr. Christopher U. Missling, expressed his gratitude for the CHMP’s decision. He highlighted the need for scalable, easily administered treatment options like oral blarcamesine, which does not require complex procedures. This is particularly important for Alzheimer’s disease, where accessible and straightforward treatment options are vital.
Alzheimer’s disease affects around 7 million people in Europe, with this number expected to rise significantly in the coming years. Blarcamesine could potentially play a pivotal role in addressing this growing population, offering an accessible treatment alternative. Unlike some other therapies, blarcamesine does not require invasive diagnostic tools, such as amyloid PET scans or spinal taps, to confirm eligibility for treatment.
This expands the drug’s potential market compared to monoclonal antibodies, which are limited to patients who meet specific amyloid criteria. According to a National Institutes of Health (NIH) analysis, only a small percentage of Alzheimer’s patients qualify for treatments that rely on these criteria.
Efficacy in Clinical Trials
The effectiveness of blarcamesine has been demonstrated in the ANAVEX®2-73-AD-004 trial, a placebo-controlled Phase 2b/3 study. This trial showed significant improvements in dementia symptoms for patients taking the drug, along with reductions in amyloid build-up and brain volume loss, which are both associated with the progression of Alzheimer’s disease.
Anavex plans to release the results from this Phase 2b/3 clinical trial in a peer-reviewed journal soon, providing further insight into the drug’s potential as a treatment for Alzheimer’s disease.
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