Fragile X Clinical Trial Results of metadoxine by Alcobra

Fragile X Clinical Trial Results of metadoxine by Alcobra

Metadoxine for Fragile X – a glimmer of hope Alcobra just announced results from their trial of a proprietary formulation of metadoxine in Fragile X Syndrome subjects, and there is a glimmer of hope in their announcement http://www.alcobra-pharma.com/releasedetail.cfm?ReleaseID=919218. While the drug did not show any beneficial effect on the primary outcome measure (essentially, an ADHD scale) it did result in some statistically significant improvement on 2 secondary measures. One of those showing improvement, a subscale of the Vineland Adaptive Behavior Scale (VABS) which measures daily living skills, is a well validated outcome measure which has been used in other Fragile X trials (though none of the other drugs resulted in improvement.) The other outcome measure showing improvement was the  KiTAP distractibility test. In both cases, the improvement was not only statistically significant, but also likely clinically meaningful; in other words, it was a relatively large effect. The folks at Alcobra rightly note that this is the best showing to

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FDA Grants Orphan Drug Status to Alcobra’s Metadoxine for Fragile X Syndrome

“We are pleased that the FDA has granted Orphan Drug status to Metadoxine, the active ingredient in MG01CI, in Fragile X,” said Dr. Yaron Daniely, President and Chief Executive Officer of Alcobra Ltd. “We recently achieved positive results from a preclinical study of Fragile X and believe this outcome supports investigation in clinical trials which we plan to initiate in 2014. We recently raised approximately $38 million in a secondary offering, and we believe we now have enough cash to fund the Company through planned submissions of NDA and sNDA filings for MG01CI for adult ADHD, pediatric ADHD and Fragile X Syndrome.” In September this year, Alcobra announced positive findings from a pre-clinical study of Fragile X Syndrome. The study showed significant improvement in cognitive and social functioning following treatment with MG01CI in a valid animal model of Fragile X Syndrome (FMR1 knock-out mouse model). The study was funded in

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