UPDATE: Alcobra Issues Protocol to FDA for Phase IIb Study of Metadoxine ER for Treatment of Fragile X Syndrome

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."