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Alcobra (ADHD)’s Fragile X Syndrome Drug Fails During Mid-Stage Trial – Courtesy (Biospace)
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June 24, 2015
By Alex Keown, BioSpace.com Breaking News Staff

TEL AVIV, Israel – Israel-based Alcobra Ltd. (ADHD) said its drug treating Fragile X, a genetic disorder that could lead to autism, failed to meet its primary endpoint in a mid-stage trial.

The company’s stock dropped this morning in response to the news. Shares of Alcobra (ADHD) were down about 13 percent, hitting a low of $6.76 per share.

The drug, MDX (Metadoxine Extended Release), did not achieve statistical significance from baseline to week 6 of the inattentive subscale of the Attention Deficit Hyperactivity Disorder Rating Scale. The difference between the MDX-treated group and placebo was not statistically significant (p=0.21), favoring placebo, the company said.

However, MDX did achieve statistical significance, in the Intent-to-Treat (ITT) population, on two secondary endpoints, including the Vineland Adaptive Behavior Scale (VABS) Daily Living Skills Domain (p=0.044), and the computerized cognitive Test of Attentional Performance for Children (KiTAP) Distractibility subscale, the company said.

Patients who received MDX during the trial showed improvements in adaptive behavior and cognition, which suggests a meaningful finding, Elizabeth Berry Kravis, the lead investigator and a professor of Neurology at Chicago-based Rush University Medical Center said in a statement.

The average IQ for participants in the study was 57 for subjects taking MDX and 52 for subjects taking placebo, with a wide overall range of 18 to 107. Autism spectrum disorder, as classified by the Autism Diagnostic Observation Schedule, was present in over 80 percent of the 40 subjects who participated in the study.

MDX received an Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Fragile X Syndrome. There are currently no approved drugs to treat the genetic disorder.

Fragile X Syndrome is caused by changes in the fragile X mental retardation 1 (FMR1) gene, according to the U.S. Centers for Disease Control. Fragile X affects both males and females, however, females typically show milder symptoms than males. It is unknown how many people are afflicted with Fragile X Syndrome, but the CDC estimates that about one in 5,000 males are born with the disorder.

Alcobra said it plans to discuss trial results with the FDA before finalizing the design of the next study of MDX for Fragile X Syndrome.

Yaron Daniely, president and chief executive officer of Alcobra, said he was encouraged the drug met its secondary endpoint.

“FXS is a difficult disease to treat and study, as demonstrated by the lack of approved treatments. Our findings suggest a clinically meaningful advance for patients and caregivers affected by FXS and we plan to meet with the FDA to determine next steps in advancing our research,” Daniely said in a statement.

MDX was generally well tolerated and no safety concerns were identified. The most common side effects were an upper respiratory infection and irritability.

When used to treat adolescent patients with attention deficit disorder, MDX met primary endpoints in a Phase II study, thecompany said in March.



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