Anavex Life Sciences Corporation is focused on developing innovative small molecule therapeutics aimed at treating various central nervous system (CNS) disorders, including Alzheimer's disease, Parkinson's disease, schizophrenia, and rare conditions like Rett syndrome. The company's proprietary SIGMACEPTOR™ platform is designed to produce orally available compounds that target the Sigma-1 receptor (SIGMAR1). By activating SIGMAR1, Anavex aims to restore neural cell homeostasis and enhance neuroplasticity, potentially unlocking the body's natural defenses against neurodegeneration. Their lead candidate, ANAVEX®2-73 (blarcamesine), has shown promise in clinical trials, demonstrating the ability to improve autophagy, a critical process for clearing misfolded proteins and protein aggregates associated with neurodegenerative diseases.

In addition to ANAVEX®2-73, the company is advancing ANAVEX®3-71, which acts as a dual SIGMAR1 receptor agonist and M1 positive allosteric modulator. This compound has exhibited a dose-dependent pharmacodynamic effect on EEG biomarkers in schizophrenia patients, indicating its potential to address various symptom domains without the side effects commonly associated with traditional antipsychotics. Anavex's research highlights the interconnectedness of neurodegenerative and neuropsychiatric disorders, suggesting that their therapeutic approaches may offer comprehensive benefits across multiple CNS conditions. The ongoing clinical studies aim to further validate the efficacy and safety of these investigational drugs, with the goal of providing new treatment options for patients worldwide.

United States

Anavex Life Sciences Corp., is a clinical-stage biopharmaceutical company focused on developing treatments for debilitating neurodegenerative and neurodevelopmental diseases. The Company’s lead product candidate, ANAVEX®2-73 is undergoing four mid- to late-stage clinical trial programs for CNS diseases where no good treatment options exist, or current treatments are inadequate. These include Alzheimer’s disease (Phase 2b/3) and Parkinson’s disease dementia (Phase 2), which are neurodegenerative diseases that are on the rise due to aging populations in developed nations. Additionally, Anavex has two ongoing Phase 2 trials for Rett syndrome, a neurodevelopmental disease that mostly affects young female girls. Anavex has received Orphan Drug Designation from both the FDA and EMA for Rett syndrome. ANAVEX®2-73 has potential to treat other CNS disorders, given its ability to restore upstream cellular homeostasis. Anavex has received funding from Rett Syndrome Foundation, as well as Michael J Fox Foundation supporting Anavex’s studies. Anavex has identified a novel biomarker signature (genetic variants) that have potential to determine which patients will benefit the most from ANAVEX®2-73, which targets the Sigma-1 receptor (SIGMAR1), a natural cell signaling pathway that is activated to restore cellular homeostasis.

Cardiovascular Diseases

Central Nervous System (CNS)

Genetic Diseases

Nervous System Diseases

Psychological Diseases

Anavex Life Sciences Corporation

ANAVEX®2-73 (BLARCAMESINE)

ANAVEX®3-71 (AF710B)

Parkinson's disease

ANAVEX®2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) &nbsp;protein, which serves as a molecular chaperone and functional modulator &nbsp;involved in restoring homeostasis. In a Phase 2a Alzheimer’s disease &nbsp;(AD) study, ANAVEX®2-73 (blarcamesine) has shown dose dependent &nbsp;improvement in exploratory endpoints of cognition (MMSE) and activities &nbsp;of daily living (ADCS-ADL). Full genomic analysis of ANAVEX®2-73 (blarcamesine) Phase 2a study in AD patients was performed. The ANAVEX®2-73 (blarcamesine) Phase 2 PDD study design includes genomic biomarkers identified in the ANAVEX®2-73 (blarcamesine) Phase 2a AD study. Studies of ANAVEX®2-73 (blarcamesine) in a disease modifying animal model of Parkinson’s disease indicates that ANAVEX®2-73 (blarcamesine) &nbsp;is well tolerated, induces significant motor recovery (p&lt;0.05), &nbsp;induces neurohistological restoration (p&lt;0.05) and reduces microglial &nbsp;activation (p&lt;0.05), a potential biomarker of Parkinson’s disease. &nbsp;Behavioral patterns were completely normal, meaning no signs of either &nbsp;dystonia or stereotypic behaviors were detected in animals receiving the &nbsp;treatment. These studies were funded by The Michael J. Fox Foundation &nbsp;for Parkinson’s Research.
<a href="https://www.anavex.com/proof-of-concept-controlled-phase-2-clinical-trial-data-evaluating-anavex2-73-blarcamesine-in-parkinsons-disease-dementia-presented-at-ctad-2020-conference/">Source</a>

ANAVEX 2-73 (Blarcamesine) 

Rett syndrome

ANAVEX®2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) &nbsp;protein, which serves as a molecular chaperone and functional modulator &nbsp;involved in restoring homeostasis. In a Phase 2a Alzheimer’s disease &nbsp;(AD) study, ANAVEX®2-73 showed dose dependent improvement in exploratory &nbsp;endpoints of cognition (MMSE) and activities of daily living &nbsp;(ADCS-ADL). The ANAVEX®2-73 Phase 2 Rett syndrome study design includes &nbsp;genomic biomarkers of response identified in the ANAVEX®2-73 Phase 2a AD &nbsp;study. Studies of ANAVEX®2-73 (blarcamesine) in a mouse model of Rett syndrome, carrying a heterozygous Mecp2-null &nbsp;mutation and presenting with neurological impairments that mimic the &nbsp;human disorder, demonstrated improvements at both young ages (~6 weeks) &nbsp;and adulthood (7 months). Chronic oral administration of ANAXEX®2-73 (blarcamesine) &nbsp;led to significant improvements in multiple motor, sensory, and &nbsp;autonomic phenotypes of relevance to Rett syndrome, including motor &nbsp;coordination and balance, acoustic and visual responses, hindlimb &nbsp;clasping (resembling the characteristic hand stereotypies), and episodes &nbsp;of apnea. Notably, these changes were dose related and also evident in &nbsp;animals at an advanced stage of pathology.
<a href="https://www.anavex.com/anavex-life-sciences-announces-exceeding-of-enrollment-target-for-the-anavex2-73-blarcamesine-u-s-phase-2-rett-syndrome-clinical-trial/#_ftnref3">Source</a>

ANAVEX 2-73 (blarcamesine) (RS-002)

ANAVEX®2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring homeostasis. In a Phase 2a Alzheimer’s disease (AD) study, ANAVEX®2-73 showed dose dependent improvement in exploratory endpoints of cognition (MMSE) and activities of daily living (ADCS-ADL). The ANAVEX®2-73 Phase 2 Rett syndrome study design includes genomic biomarkers of response identified in the ANAVEX®2-73 Phase 2a AD study. Studies of ANAVEX®2-73 (blarcamesine) in a mouse model of Rett syndrome, carrying a heterozygous Mecp2-null mutation and presenting with neurological impairments that mimic the human disorder, demonstrated improvements at both young ages (~6 weeks) and adulthood (7 months). Chronic oral administration of ANAXEX®2-73 (blarcamesine) led to significant improvements in multiple motor, sensory, and autonomic phenotypes of relevance to Rett syndrome, including motor coordination and balance, acoustic and visual responses, hindlimb clasping (resembling the characteristic hand stereotypies), and episodes of apnea. Notably, these changes were dose related and also evident in animals at an advanced stage of pathology. These experiments were sponsored by Rettsyndrome.org.

ANAVEX 2-73 (blarcamesine) (2-73-RS-003) 

Alzheimer's disease

ANAVEX®2-73 (blarcamesine) activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring homeostasis. S1R activation has demonstrated ability to reduce key pathophysiological signs of Alzheimer’s disease: beta amyloid, hyperphosphorylated tau, and increased inflammation. In the Phase 2a 57-week Alzheimer’s disease (AD) ANAVEX®2-73-002 (ClinicalTrials.gov NCT02244541) study, ANAVEX®2-73 (blarcamesine) has shown dose dependent improvement in exploratory endpoints of cognition (MMSE) and function (ADCS-ADL). Its open-label extension study ANAVEX®2-73-003 (ClinicalTrials.gov NCT02756858) for an additional 208 weeks included full genomic analysis of patients with Alzheimer’s disease treated with ANAVEX®2-73 (blarcamesine). A 48-week Phase 2b/3 study ANAVEX®2-73-AD-004 (ClinicalTrials.gov NCT03790709) of ANAVEX®2-73 (blarcamesine) in 450 patients with early Alzheimer’s disease is ongoing and currently over 60% enrolled.

ANAVEX 2-73 (Blarcamesine)

Schizophrenia

ANAVEX 3-71 (previously AF710B) is a preclinical drug candidate &nbsp;targeting both sigma-1 and M1 muscarinic receptors demonstrating &nbsp;neuroprotection and improved cognition in Alzheimer’s disease models. &nbsp;&nbsp;It is a CNS-penetrable mono-therapy that bridges treatment of both &nbsp;cognitive impairments with potential disease modifications. &nbsp;ANAVEX 3-71 &nbsp;is effective in very low doses against the major Alzheimer’s hallmarks &nbsp;in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and &nbsp;tau pathologies, and has also demonstrated beneficial effects on &nbsp;neuroinflammation and mitochondrial dysfunctions
<a href="https://www.anavex.com/u-s-fda-grants-orphan-drug-designation-to-anavex-3-71-for-the-treatment-of-frontotemporal-dementia/">Source</a>

Anavex Life Sciences Corporation

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