Forward momentum in Fragile X

Anavex reports positive preclinical results with ANAVEX 2-73 in Fragile X

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NEW YORK—Biopharmaceutical company Anavex Life Sciences has announced that ANAVEX 2-73, an experimental drug for Alzheimer’s disease, has shown in a new preclinical study to have an effect on autism-like symptoms in mice with Fragile X syndrome, the most common form of inherited intellectual disability and most frequent single-gene cause of autism.
The positive data of ANAVEX 2-73 in an exploratory study was presented June 6 at the Gordon Research Conference for Fragile X and Autism-Related Disorders in Mount Snow, Vt.
Anavex is currently conducting a Phase 2a trial of ANAVEX 2-73 in Alzheimer’s and has received orphan designation from the U.S. Food and Drug Administration for Rett syndrome, according to lead investigator Dr. Michael Tranfaglia, medical director and chief scientific officer of FRAXA Research Foundation. The unique mechanism of action of the drug, a Sigma-1 receptor agonist, restores endoplasmic reticulum mitochondria.
Study researchers report that dosing of ANAVEX 2-73 for 14 days in a Fragile X syndrome mouse model was followed by a battery of behavioral tests linked to pathophysiological signs of autism-related disorders and Fragile X syndrome: hyper-locomotion, associative learning and marble burying.
Treatment with ANAVEX 2-73 significantly improved all behaviors tested. ANAVEX 2-73 significantly reversed the hyperactivity and impairment in learning and memory to the same levels observed in vehicle-treated wild-type mice. ANAVEX 2-73 also yielded a partial effect in the marble burying experiment. The study was sponsored by FRAXA, via the FRAXA Drug Validation Initiative, and performed by Fraunhofer Chile Research.
“The ANAVEX 2-73 data in an array of behavioral paradigms in a validated mouse model of Fragile X is very encouraging,” Tranfaglia states.
The results are promising for both Fragile X syndrome and autism spectrum disorders, since there is an overlap in the clinical as well as in the underlying molecular pathology of the two disorders, Tranfaglia says, adding that Anavex “would be very interested in continuing the work with ANAVEX 2-73.”
A potential finding in the search for treating autism, the breakthrough study also underscores the fact that mitochondrial dysfunction is known to be a central factor in many intellectual disability-related diseases, including Parkinson’s disease, Alzheimer’s disease, Down syndrome, autism, Fragile X syndrome and Rett syndrome.
“Our previous results in Rett syndrome, together with the new data in another neurodevelopmental disorder, Fragile X, support our hypothesis that ANAVEX 2-73 has a mechanism of action that could be of interest to investigate clinically in rare disorders like Rett syndrome, Fragile X syndrome and other autism spectrum disorders, in addition to neurodegenerative diseases such as Alzheimer’s disease,” states Christopher U. Missling, president and CEO of Anavex.
Additionally, The Michael J. Fox Foundation for Parkinson’s Research has awarded Anavex a research grant to develop ANAVEX 2-73 for the treatment of Parkinson’s disease to fully fund a preclinical study, which could justify moving ANAVEX 2-73 into a Parkinson’s disease clinical trial.
The poster of this preclinical data, titled “Sigma-1 Receptor Agonists as Potential Treatment Options for Autism Spectrum Disorders: Pre-clinical Studies with ANAVEX 2-73 in a Fragile X Model,” is slated to soon be available on the publications page of the Anavex website, according to the company.
Fragile X syndrome affects approximately one in 4,000 males and one in 6,000 females. Autism is much more common in boys with this condition than in girls. According to the Centers for Disease Control and Prevention, a national parent survey found that 46 percent of males and 16 percent of females with Fragile X syndrome have been diagnosed or treated for autism spectrum disorders.

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