Anavex's neuro drug slows decline in Parkinson's disease as it boosts levels of its biomarker

A neurologist reviews brain scans
Establishing the relationship between efficacy and biomarker is particularly salient after Biogen’s Aduhelm scored an FDA approval on its ability to reduce the amount of beta-amyloid in the brain rather than its impact on the cognitive symptoms of Alzheimer’s disease. (Getty Images/Chinnapong)

Anavex’s Parkinson’s disease treatment beat placebo at slowing the progression of motor and non-motor symptoms, a benefit that correlated with an increase in SIGMAR1, the drug’s target and the biomarker the company will use to forecast its efficacy. Anavex will now submit the phase 2 data to the FDA to figure out the drug’s path forward. 

The company tested two doses of the treatment, known as blarcamesine or Anavex2-73, against placebo in 132 patients with Parkinson’s disease dementia. Patients were allowed to keep taking their usual Parkinson’s meds. 

After 14 weeks of treatment, the higher dose, 50 mg, boosted patients’ scores on the Movement Disorder Society’s Unified Parkinson Disease Rating Scale, a measure of various aspects of Parkinson’s disease, by nearly 11 points or 18.9% from baseline. Patients taking placebo saw their scores worsen by 3.53 points. An improvement of 7.1 points on that scale is considered clinically meaningful.

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This improvement, as well as improvements in cognitive assessments, were linked to an increase in mRNA expression of SIGMAR1, or the sigma-1 receptor, which is involved in the transport of lipids and proteins within the cell and plays a role in the development of neurodegenerative diseases. Anavex2-73 is designed to activate SIGMAR1 and the company is developing the drug for Alzheimer’s disease, Rett syndrome, infantile spasms, Angelman’s and Fragile X syndrome. 

The company’s stock opened 20% higher Monday morning than its closing price on Friday. 

Besides hustling the data to the FDA to determine the drug’s next steps in Parkinson's, Anavex plans to present the data at a medical meeting later this year. 

The Parkinson’s trial is the second placebo-controlled study to link SIGMAR1 mRNA expression with a clinical benefit, said Anavex CEO Christopher Missling, Ph.D. in a statement. “We believe that the easily accessible predictive biomarker combined with the observed efficacy is a consistent explanation of the efficacy in this second largest CNS indication with unmet medical need. This data further strengthens the foundation of ANAVEX®2-73 as a cross-platform CNS drug.” 

RELATED: Biogen, fresh off coveted FDA nod for Aduhelm, must now navigate ethics minefield for phase 4 trial 

Establishing the relationship between efficacy and biomarker is particularly salient after Biogen’s Alzheimer’s treatment Aduhelm (aducanumab) scored an FDA approval on its ability to reduce the amount of beta-amyloid in the brain rather than its impact on the cognitive symptoms of Alzheimer’s. 

Although Biogen may now market Aduhelm in the U.S., the FDA requires the company to conduct a post-approval confirmatory study to prove that the drug, in fact, slows the progression of the disease in addition to reducing amyloid. If the new study doesn't confirm a clinical benefit, the FDA could pull the drug from the market.