Israel-based biopharmaceutical firm Alcobra has submitted a protocol to the US Food and Drug Administration (FDA) for a Phase IIb clinical trial of its proprietary drug candidate Metadoxine Extended Release (MDX) for treatment of patients with Fragile X Syndrome (FXS), a genetic condition that is the most widespread single-gene cause of autism.

The planned multi-centre, randomised, placebo-controlled Phase IIb MDX clinical trial will be carried out in the US.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

The submission of protocol is under an IND and is supported by strong, positive data secured from multiple earlier pre-clinical trials on metadoxine.

Alcobra said that results from the pre-clinical trials showed significant improvement in behavioural and cognitive outcomes based on evaluations of memory, learning, and social interaction.

“The positive findings reported from the preclinical studies, together with information that is known on the mechanism of action of metadoxine, suggest that MDX may be helpful in treating cognitive symptoms in these patients.”

In addition, metadoxine treatment carried out in a validated mouse model of Fragile X Syndrome, showed improved levels of certain Fragile X-associated blood and brain biological markers that may have a role in learning and memory, while simultaneously reducing the number of immature brain connections and levels of abnormally increased protein.

The FDA granted orphan drug designation to metadoxine in December 2013 for the treatment of Fragile X Syndrome.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Rush University Medical Center professor of biochemistry, neurological sciences and pediatrics Elizabeth Berry-Kravis said patients with Fragile X Syndrome currently have limited treatment options, with no FDA approved medications.

“The positive findings reported from the preclinical studies, together with information that is known on the mechanism of action of metadoxine, suggest that MDX may be helpful in treating cognitive symptoms in these patients,” said Berry-Kravis, and principal investigator in the trial.

“This Phase IIb study should provide us with important insights into the potential role of MDX in this condition.”

FXS causes intellectual disability, behavioural and learning challenges and various physical characteristics.

Behavioural characteristics can include ADHD, autism and autistic behaviors, social anxiety, stereotypic movements, poor eye contact, sensory disorders and increased risk for aggression.

According to the US Centers for Disease Control and Prevention (CDC), one in 4,000 males and one in 8,000 females have FXS.

To date, the FDA has not approved any drugs specifically for the treatment of Fragile X Syndrome or its symptoms.