Alcobra Reports Data From Phase 2 Clinical Trial Of MDX For Fragile X Syndrome

(RTTNews) - Alcobra Ltd. (ADHD) reported that its Phase 2 clinical study of MDX for the treatment of FXS did not meet the primary endpoint of change from baseline to week 6 of the inattentive subscale of the Attention Deficit Hyperactivity Disorder Rating Scale or ADHD RS-IV.

The company noted that the difference between treatment groups was not statistically significant. However, MDX did achieve statistical significance, in the Intent-to-Treat or ITT population, on two secondary endpoints, including the Vineland Adaptive Behavior Scale or VABS Daily Living Skills Domain, and the computerized cognitive Test of Attentional Performance for Children Distractibility subscale. In the study, MDX was generally well tolerated and no safety concerns were identified.

According to the company, there are no approved medications to treat FXS. MDX has received an Orphan Drug designation from the U.S. Food and Drug Administration or FDA for the treatment of FXS.

Alcobra said it plans to discuss these trial results with the FDA before finalizing the design of the next study of MDX in FXS.

The purpose of the randomized, double-blind, placebo-controlled, multi-center Phase 2 clinical trial was to assess the efficacy and safety of MDX compared to placebo in 62 adolescent and adult patients with FXS, a rare neurogenetic condition and the leading known genetic cause of autism. Efficacy measures included both behavioral and cognitive scales.