Alcobra Announces FDA Clearance of Protocol for Phase IIb Study of Metadoxine Extended Release (MDX) in the Treatment of Frag...
Thursday 8 May 2014
Alcobra Ltd. (Nasdaq:ADHD), 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 FDA has approved the protocol for the Company's planned Phase IIb clinical trial of MDX for the treatment of Fragile X Syndrome. The trial is expected to begin enrolling patients shortly.
The Phase IIb study will be a multi-center, randomized, placebo-controlled study, conducted primarily in the US, and is supported by positive data collected from multiple earlier pre-clinical studies. Results from these pre-clinical studies demonstrated significant improvement in behavioral and cognitive outcomes based on evaluations of memory, learning, and social interaction. 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. The FDA granted "Orphan Drug" designation to Metadoxine in the treatment of Fragile X Syndrome in December 2013.
"Given the lack of FDA approved therapies, there is a substantial unmet need for treatment options for Fragile X Syndrome," said Dr. Yaron Daniely, President and Chief Executive Officer of Alcobra. "We are pleased that the FDA has approved the protocol for our Phase IIb study, and we expect it to provide important insights into the potential role of MDX in this and related conditions."
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.
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