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Anavex Life Sciences Corp. recently released promising study results for the treatment of Parkinson’s disease dementia patients. The 48-week open-label extension of its Phase 2 study, Anavex 2-73-PDD-EP-001 (NCT04575259), assessed the investigational agent Anavex 2-73, known as blarcamesine. The study demonstrated positive outcomes in meeting primary and secondary objectives within the target cohort. Anavex Life Sciences is a clinical-stage biopharmaceutical company specializing in therapies for neurodevelopmental and neurodegenerative disorders.
The extension phase of the study experienced delays due to the COVID-19 pandemic. The resulting average delay of approximately 41 weeks after the double-blind period led to reduced enrollment rates and a "drug holiday" for participants. Despite these challenges, all efficacy endpoints, such as Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III and Clinical Global Impression-Improvement (CGI-I), consistently improved upon resumption of treatment.
Blarcamesine, or Anavex 2-73, is a promising investigational drug developed by Anavex to target specific receptors in the brain associated with cognitive function and neuronal health. The primary focus of blarcamesine’s mechanism of action lies in its ability to modulate sigma-1 receptors. These receptors are known to have a crucial role in various cellular processes, including neuroprotection, neurotransmitter regulation, and neuronal signaling. By interacting with sigma-1 receptors, blarcamesine aims to restore cellular homeostasis and support neuronal survival.
One of the most exciting aspects of blarcamesine is its potential therapeutic application in Alzheimer’s disease and other neurodegenerative disorders such as Parkinson’s. Preclinical studies have demonstrated that blarcamesine can mitigate cognitive decline and reduce pathological features associated with neurodegeneration. This includes the reduction of amyloid-beta plaques and tau protein tangles, hallmark indicators of Alzheimer's disease.
Additionally, blarcamesine has shown promise in improving synaptic plasticity, which is essential for learning and memory processes. By enhancing synaptic function and neuronal connectivity, blarcamesine may offer a novel approach to treating cognitive impairment in neurodegenerative diseases.
Ongoing clinical trials will assess blarcamesine’s potential to improve cognitive function and quality of life in patients with Alzheimer's disease and other cognitive disorders. Early results from these trials have shown encouraging outcomes, opening the way for further investigation and potential regulatory approval.
Blarcamesine represents a groundbreaking approach to addressing cognitive decline in neurodegenerative disorders by targeting sigma-1 receptors and restoring neuronal function. While more research is needed to fully understand the range of its therapeutic potential, blarcamesine holds promise as a future treatment option for individuals suffering from Alzheimer's disease and related conditions.
Assessment
Christopher U. Missling, Ph.D., president and CEO of Anavex, commented on the findings. “It is encouraging that the patients’ clinical symptoms consistently improved longitudinally over time during the extension phase under active Anavex 2-73 treatment," he said. These observations suggest potential for Anavex 2-73 to address the urgent unmet need for managing symptoms of Parkinson’s disease.
Over the study's extension period (from OLE baseline to Week 48), improvements were recorded in various measures. Changes included a mean reduction of minus 2.25 (SE, 6.656) in MDS-UPDRS total score and a reduction of minus 0.7 (SE, 0.309) in CGI-I score. Notably, there were also mean changes of minus 1.2 (SE, 0.537) in Montreal Cognitive Assessment and minus 0.524 (SE, 0.620) in REM Sleep Behavior Disorder Screening Questionnaire scores. Specific MDS-UPDRS scores showed improvements, including mean changes of minus 3.95 (SE, 4.067) in Part III scores and minus 2.20 (SE, 5.314) in combined Part II and III scores.
In a prior report, patients on high-dose blarcamesine showed significant improvements compared to the placebo group at the end of the double-blind period. This included a relative improvement of 18.9% in MDS-UPDRS total score. Additionally, gene expression levels related to neurodegenerative processes were notably restored by blarcamesine treatment.
Given the study's limitations, notably its small sample size, caution is advised in interpreting these results. Participants who completed the 48-week extension were granted continued access to blarcamesine through a compassionate use Special Access Scheme. The upcoming six-month Parkinson’s disease study by Anavex will further examine major endpoints, including MDS-UPDRS II and III and CGI-I.
Earlier data from the Phase 2 proof-of-concept study underscored blarcamesine’s impact on increasing sigma-1 receptor mRNA expression, aligning with observed clinical efficacy measures. These findings correlated with improvements in cognitive and motor assessments, supporting blarcamesine's potential therapeutic benefit.
The Phase 2 trial comprised 132 Parkinson’s disease dementia patients randomized to blarcamesine 30 mg, 50 mg, or a placebo for up to 14 weeks. Patients were allowed to continue their existing anti-Parkinson’s medications during the study period. The observed improvements in Parkinson’s disease dementia patients indicate blarcamesine’s potential to address the progression of Parkinson’s symptoms, addressing a critical unmet need within this patient population. Notably, blarcamesine demonstrated positive effects on REM sleep behavior disorder without impairing sleep.
These findings contribute to the evolving understanding of blarcamesine’s potential role in addressing neurodevelopmental and neurodegenerative diseases like Parkinson's disease dementia. Continued research will be crucial in validating these outcomes and determining the broader implications for patients living with these conditions.
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Contact Info:
Name: Christopher Missling
Email: Send Email
Organization: Anavex Life Sciences Corporation
Address: 630 5th Avenue, 20th floor, New York, NY, 10111 USA
Phone: 1-844-689-3939
Website: https://www.anavex.com/
Release ID: 89131553
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