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Anavex encouraged by scientific data confirming sigma-1 receptor agonist extends survival in ALS (Lou Gehrig’s disease)

a clinical stage biopharmaceutical company developing novel drug candidates to treat Alzheimer’s, diseases of the central nervous system (CNS) and various types of cancer, has announced the findings of a research report published in the current issue of peer-reviewed scientific journal . The Company is encouraged by the report because its results point to potential for ANAVEX 2-73 to treat ALS, also known as Lou Gehrig’s disease. Data showed that sigma-1 receptor (S1R) agonists are effective in suppressing motor neuron degeneration and symptom progression in ALS animal models.

“The findings from this new study are very encouraging and may increase the scope of the therapeutic potential of our sigma-1 receptor agonists, including ANAVEX 2-73,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We are excited to initiate pre-clinical exploration of our S1R agonist drug candidate ANAVEX 2-73 in ALS models.”

(ALS) is a devastating and usually fatal neurodegenerative disease characterized by progressive degeneration of motor neurons in the CNS. Most people with ALS die from respiratory failure within three to five years from the onset of symptoms. Recently, mutations in the gene of the sigma-1 receptor have been identified as a cause of familial juvenile ALS.

The Neuroscience Letters report describes how the S1R agonist “SA4503″ protected motor neuronal cells against induced cell death in both in vitro and in vivo animal models. One important mechanism by which motor neuronal injury is caused involves inhibition of specific components of the mitochondrial electron transfer chain. Therapeutic measures aimed at protecting mitochondrial respiratory chain function may be useful in related familial and possibly other forms of ALS.

ANAVEX 2-73, developed to treat Alzheimer’s through potential disease modification, may have a similar beneficial effect on respiratory cell mitochondria, which could be valuable in treating ALS. The drug candidate recently demonstrated its ability to repair mitochondrial functionality in the hippocampus, the part of the brain involved with learning, memory and emotions. Mitochondrial dysfunction has been consistently reported as an early cause of Alzheimer’s disease.

The report, entitled “SA4503, a sigma-1 , suppresses motor neuron damage in in vitro and in vivo amyotrophic lateral sclerosis models,” was authored by Dr. Hideaki Hara and colleagues from the Department of Molecular Pharmacology, Department of Biofunctional Evaluation, Gifu Pharmaceutical University, Gifu, Japan.


SA4503, a sigma-1 receptor agonist, suppresses motor neuron damage in in vitro and in vivo amyotrophic lateral sclerosis models, Yoko Ono, Hirotaka Tanaka, Masafumi Takata, Yuki Nagahara, Yasuhiro Noda, Kazuhiro Tsuruma, Masamitsu Shimazawa, Isao Hozumi, Hideaki Hara, Neuroscience Letters (2014) DOI: 10.1016/j.neulet.2013.12.005.

Anavex Life Sciences Corp