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.

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.”

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.”

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.