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Alcobra Ltd. Submits Protocol to FDA for Phase IIb Clinical Study of Metadoxine Extended Release (MDX) in the Treatment of Fragile X Syndrome

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April 10, 2014

Alcobra Ltd., an emerging biopharmaceutical company primarily focused on the development and commercialization of its proprietary drug candidate Metadoxine Extended Release (MDX), to treat cognitive dysfunctions, such as ADHD and Fragile X Syndrome, today announced that it has submitted a protocol to the FDA for a Phase IIb clinical trial for its MDX product candidate for the treatment of patients with Fragile X Syndrome.

The planned Phase IIb MDX clinical trial will be a multi-center, randomized, placebo-controlled study, conducted primarily in the US. The protocol submission is under an IND and is supported by strong, positive data collected from multiple earlier pre-clinical trials on metadoxine. Results from these studies demonstrated significant improvement in behavioral and cognitive outcomes based on evaluations of memory, learning, and social interaction. Furthermore, in a validated mouse model of Fragile X Syndrome, metadoxine treatment was shown to result in 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. In December 2013, the FDA granted “Orphan Drug” designation to Metadoxine in the treatment of Fragile X Syndrome.

Elizabeth M. Berry-Kravis, MD, PhD, Professor of Biochemistry, Neurological Sciences and Pediatrics at Rush University Medical Center in Chicago, Illinois and Principal Investigator in this study commented, “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. This Phase IIb study should provide us with important insights into the potential role of MDX in this condition.”

About Fragile X Syndrome

Fragile X syndrome (FXS) is a genetic condition that causes intellectual disability, behavioral and learning challenges and various physical characteristics. Behavioral characteristics can include ADHD, autism and autistic behaviors, social anxiety, stereotypic movements, poor eye contact, sensory disorders and increased risk for aggression. Fragile X Syndrome is the leading known genetic cause of autism, accounting for about 2-5% of cases. Fragile X Syndrome represents an unmet medical need and a rare disease, as defined by the Orphan Drug Act. According to the U.S. Centers for Disease Control and Prevention (CDC), approximately one in 4,000 males and one in 8,000 females have Fragile X Syndrome. The FDA has not approved any drugs specifically for the treatment of Fragile X Syndrome or its symptoms.

About Alcobra Ltd.

Alcobra Ltd. is an emerging biopharmaceutical company primarily focused on the development and commercialization of a proprietary drug candidate, MDX (Metadoxine Extended Release (MG01CI)), to treat cognitive dysfunctions including Attention Deficit Hyperactivity Disorder (ADHD) and Fragile X Syndrome.  MDX has completed Phase II studies to treat Attention Deficit Hyperactivity Disorder. The company was founded in 2008 and is headquartered in Tel Aviv, Israel. For more information please visit the Company’s website, www.alcobra-pharma.com, the content of which is not incorporated herein by reference.

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