The multi-center, randomized, placebo-controlled, Phase IIb study, will be conducted primarily in the US and patient enrollment is expected to begin in the near future.
The study is supported by data collected from multiple earlier pre-clinical studies which demonstrated significant improvement in behavioral and cognitive outcomes based on evaluations of memory, learning, and social interaction.
According to Alcobra, 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.
Alcobra president and CEO Dr Yaron Daniely noted that given the lack of FDA approved therapies, there is a substantial unmet need for treatment options for Fragile X Syndrome.
"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," Dr Daniely added.
In December 2013, Metadoxine has received Orphan Drug designation from the FDA for the treatment of Fragile X Syndrome.
Image: Chemical structure of salt pyridoxine-pyrrolidone carboxylate. Photo: Courtesy of Fluidcarl