Promising Results of Preclinical Study of ANAVEX®2-73

One of the most exciting kinds of work that FRAXA does is guiding an experimental new  treatment from early animal-model studies  until it is ready for trials in people with Fragile X. From an initial idea, through the development process, to clinical trials, FRAXA helps out all along the way.

We are excited to share that Anavex Life Sciences announced today that preclinical data of the ANAVEX®2-73 (blarcamesine) study in Fragile X syndrome were published in the peer-reviewed journal, Scientific Reports. The study evaluated ANAVEX®2-73 in Fmr1 knockout (KO) mice. The drug was able to reverse hyperactivity, restore associative learning, and reduce anxiety and perseverative behaviors. These results show promise for ANAVEX®2-73 as a potential treatment for Fragile X syndrome.

Promising Results

This is an exciting study for many reasons. The investigation used chronic administration, as opposed to a single dose, which provided additional evidence in favor of the clinical use of ANAVEX®2-73. The observation of normalization of pAkt and BDNF levels (important biomarkers of neuronal function) after ANAVEX®2-73 administration in a brain region critical for cognition and behavior is also an important finding for Fragile X syndrome and other central nervous system disorders.

This drug works by an entirely different mechanism from other drugs in development for Fragile X. ANAVEX®2-73 is a sigma agonist, which means it boosts the the activity of the sigma receptor. Boosting sigma results in very powerful anti-inflammatory effects and cell protective effects. Interestingly, Luvox, an available drug which also boosts sigma activity, shows promise in treating some symptoms of Fragile X.

“Testing novel drugs that can safely improve the symptoms of Fragile X syndrome is a high priority,” Walter E. Kaufmann, MD, Chief Medical Officer of Anavex, “The present findings support the viability of SIGMAR1 as a therapeutic target in Fragile X syndrome, and the clinical potential of ANAVEX®2-73 (blarcamesine) in Fragile X syndrome and other neurodevelopmental disorders.”

Next steps for Anavex

Anavex, with the continued support of FRAXA Research Foundation, will initiate a double-blind, placebo-controlled Phase 2/3 of ANAVEX®2-73 in Fragile X syndrome. The next phase of the trial will look for reversal of hyperactivity and restoration of associative learning as well as reduction in anxiety-like and perseverative behaviors in humans. Additional outcomes could include a reduction in anxiety-like and perseverative behaviors, too. “We look forward to initiating a double-blind, placebo-controlled Phase 2/3 ANAVEX®2-73 study in Fragile X syndrome,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “We are intrigued about the clear preclinical data providing potential to expand the therapeutic profile of ANAVEX®2-73 into the largest portion of addressable market of autism spectrum disorder, Fragile X syndrome. This is further evidence of the potential of ANAVEX®2-73 as a platform technology of precision medicine.”

What does this mean for families?

There is hope.

FRAXA continues to fund research that is making a difference. The FRAXA-Drug Validation Initiative (DVI) continues to accelerate preclinical validation of candidate treatments, helping us to get to the next steps of the drug trials faster. We are on the right track.

The next phase for Anavex will be Phase 2 double-blind, placebo-controlled study. There are good indications that the results from the next phase will also be promising for both Fragile X syndrome and Autism.

Anavex got its Start in Fragile X research through FRAXA-DVI

In 2016 we shared the exciting news about a potential new treatment from Anavex that was showing promise for Fragile X syndrome, that was funded by FRAXA. The results from the initial study were made possible through the utilization of FRAXA-DVI to determine the drug’s potential. FRAXA tested this new drug at FRAXA-DVI at a time when the company would not otherwise have tried it on Fragile X. Today’s update from Avanex is another example of the difference FRAXA makes in accelerating Fragile X research.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine), successfully completed a Phase 2a clinical trial for Alzheimer’s disease and recently a Phase 2 proof-of-concept study in Parkinson’s disease dementia and a Phase 2 study in adult patients with Rett syndrome. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and antidepressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at You can also connect with the company on Twitter, Facebook, Instagram and LinkedIn.


Global Leader in Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.

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Global Leader in Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.

Explore Current Research Grants
Help Fund the Cure

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