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.

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.

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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.