Rare Disease Report

Phase 2b Study in Fragile X Syndrome Begins

AUGUST 20, 2014
James Radke

Good news for patient with Fragile X syndrome. Alcobra Ltd. announced they have enrolled the first patient in a Phase IIb fragile X syndrome study of Metadoxine Extended Release (MDX).
The Phase IIb trial is a multi-center, randomized, placebo-controlled study being conducted in 11 clinical sites, primarily in the United States.  The study will compare MDX vs placebo for 6 weeks in 60 adolescent and adult subjects with fragile X syndrome.  The primary outcomes measures will be:
1) the Attention Deficit Hyperactivity Disorder (ADHD) Rating Scale (ADHD RS-IV, and
2) symptoms of Fragile X syndrome as measured by the inattentive subscale of ADHD RS-IV.

Dr. Yaron Daniely, President and Chief Executive Officer of Alcobra  said, "We are pleased to start enrolling patients into our Phase IIb clinical study for MDX in fragile X syndrome," adding,  "Our interest in Fragile X Syndrome is based on a comprehensive data set demonstrating significant improvements in learning, social and cognitive functions in a pre-clinical model of the disease. In addition, data supporting MDX modulation of the GABA/glutamate circuit and its rapid and significant effect on attentional capabilities in the ADHD population suggest a potential benefit in the Fragile X population. Our goal is to complete patient enrollment and report top-line data in the 4th quarter of 2014."

Elizabeth Berry Kravis, MD, PhD, Associate Professor of Biochemistry, Neurological Sciences, and Pediatrics at Rush University Medical Center, and the Principal Investigator of the Phase IIb study stated, "We expect that this study will give us important insights into the efficacy and safety of MDX in fragile X syndrome."
The study is currently recruiting patients. For more information, visit http://clinicaltrials.gov/ct2/show/NCT02126995

About Fragile X Syndrome

Fragile X syndrome is a rare genetic condition that causes intellectual disability, behavioral and learning challenges and various physical characteristics. Behavioral characteristics may 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 2-6% of cases. At present, there are no FDA approved drugs specifically to treat fragile X syndrome or its symptoms.

About Alcobra Ltd.

Alcobra Ltd. is a pharmaceutical company primarily focused on the development and commercialization of a proprietary drug candidate, MDX to treat cognitive disorders including ADHD and fragile X syndrome.  The company was founded in 2008 and is headquartered in Tel Aviv, Israel.

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