Alcobra Ltd., an emerging biopharmaceutical company, has reported positive results from a new study in Fmr1 knock-out mice, a validated animal model of Fragile X Syndrome. The study revealed that normalization of certain over-activated biological markers was evident in the blood and brains of test mice following treatment with Metadoxine.

This finding raises the possibility of using this effect as a screening tool in future clinical investigations for identifying patients who are more likely to respond to treatment and monitoring their response over time.

Improved cognitive and social functions were associated with reduction of blood levels of over-activated Akt and Extracellular signal-related Kinase (ERK) in test mice. The Akt and ERK pathways are thought to play a critical role in impaired synaptic plasticity underlying learning and memory in Fragile X Syndrome patients.

Additional findings from this new study showed that Metadoxine treatment prevented the overabundance of poorly shaped, immature neurons in the hippocampal region of the brain and reduced exaggerated new protein synthesis in the same brain region. Overabundance of immature neurons and increased protein synthesis have been implicated in the pathophysiology of Fragile X Syndrome and are presumed to be responsible for impaired learning and memory.

Dr Yaron Daniely, president and chief executive officer of Alcobra commented, "The possibility of identifying a target population that may be more responsive to therapy using a simple blood test is a valuable milestone. The findings in this study are exciting, replicating and validating previously reported findings using Metadoxine in this model. We are excited to be moving forward this year with a clinical study evaluating MG01CI in Fragile X Syndrome."

Alcobra previously demonstrated benefits on behavioral outcomes, including contextual fear conditioning (a method of assessing learning and memory) and social impairment in Fmr1 knock-out mice. These findings were reported at the FRAXA Investigators Meeting in Southbridge, MA in September 2013. These studies were funded in part by the FRAXA Research Foundation.

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 represents an unmet medical need and a rare disease, as defined by the Orphan Drug Act.

Alcobra Ltd. is an emerging biopharmaceutical company primarily focused on the development and commercialization of a proprietary drug candidate, MG01CI, to treat cognitive dysfunctions including Attention Deficit Hyperactivity Disorder (ADHD) and Fragile X Syndrome.