Initially, biopharmaceutical company Anavex Life Sciences (“Anavex”) developed Anavex 2-73 (blarcamesine) for patients with Alzheimer’s disease. Now, the company has expanded its exploration of the therapy to determine its efficacy for both Rett Syndrome and Parkinson’s disease (PD). According to an article in Parkinson’s News Today, data from the Phase 2 ANAVEX 2-73-PDD-001 clinical trial highlights the drug’s efficacy in treating motor and non-motor symptoms related to Parkinson’s disease dementia.

Anavex 2-73

During the Phase 2 clinical trial, researchers evaluated the safety, efficacy, and tolerability of Anavex 2-73. According to the ALZ Forum, Anavex 2-73 is:

[a small-molecule] agonist of the intracellular sigma-1 chaperone protein [and] a mixed ligand for sigma1/muscarinic receptors. Blarcamesine reportedly binds the sigma-1 receptor in the high nanomolar and the muscarinic receptor in the low micromolar range [and] has been reported to have memory-preserving and neuroprotective effects in mice.

Altogether, 132 patients enrolled in this proof-of-concept study. Patients, between ages 50-85, were diagnosed with Parkinson’s disease dementia. During the trial, patients received either 30 or 50mg Anavex 2-73 or a placebo for a 14-week period. The trial aimed to determine safety, changes or improvements in cognitive function, motor ability changes, and sleep quality. Findings included:

• Anavex 2-73 improved sigma-1 receptor (SIGMAR1) levels. Now, SIGMAR1 can be considered a response biomarker. Overall, the therapy works by activating SIGMAR1, which plays a role in nerve cell growth and plasticity; cellular stress response; and neuroinflammation.
• This study saw similar responses to a prior study. In short, Anavex 2-73 improved cognitive skills, lessened sleep interruptions or REM sleep behavior disorder, and lowered oxidative stress and neuroinflammation.
• 50mg Anavex 2-73 significantly reduced both motor and non-motor PD symptoms. Thus, Anavex believes that its therapy has the potential to reverse PD progression and symptoms.

Moving forward, Anavex will run an open-label extension study and will present data from this most recent study to the FDA.

Parkinson’s disease (PD)

In patients with Parkinson’s disease (PD), a progressive central nervous system (CNS) disorder, dopamine-producing (dopaminergic) neurons begin to die. Normally, these neurons contribute to communication between the brain and body, resulting in movement. But due to a mixture of genetic and environmental factors, these neurons die in PD, inhibiting communication and movement. Altogether, PD occurs in five stages. Within the first two stages, patients present with mild symptoms, such as muscle rigidity and tremors on one or both sides of the body. Balance loss and movement difficulties solidify in stage three, with the patient becoming unable to live independently by stage four. In the final stage, patients may experience hallucinations or delusions. In many cases, PD affects older individuals (50+). When symptoms appear, they include:

• Muscle stiffness and rigidity
• Tremors or shaking in one or both hands
• Loss of balance and coordination
• Impaired posture
• Slowed movement
• Changes in speech, such as slurring or stuttering
• Apathy or anxiety
• Incontinence
• Hallucinations or dementia
• Sleep disturbances