Alcobra’s Only Drug Falters in Phase II Trial for Fragile X Syndrome
Israeli drugmaker Alcobra’s only drug, MDX, failed a Phase II trial to treat Fragile X Syndrome, which can cause autism and ADHD.
During a six-week trial of 62 adolescent and adult patients, MDX (metadoxine extended-release) did not meet its primary endpoint of improving patients’ attentiveness scores on the Attention Deficit Hyperactivity Disorder Rating Scale. The results from the group using the drug were not significantly different from the placebo arm, the company says.
Alcobra notes the results could have been skewed because most of the patients took other ADHD drugs at the same time, potentially increasing the effectiveness of the placebo. The firm also backed away from its primary endpoint, saying the ADHD rating scale is limited when used by patients with varying intellectual capabilities.
Instead, Alcobra touted the results of its secondary endpoints — statistical significance on the Vineland Adaptive Behavior Scale and the cognitive Test of Attentional Performance for Children, saying they are better indicators of efficacy.
There are no approved medications for Fragile X Syndrome, which affects about 50,000 people in the U.S. MDX received an orphan drug designation from the FDA in December 2013. The company says it will discuss the trial results with the agency before finalizing the design of its next study.
This is the third setback for MDX since October, when it failed a Phase 3 study in adults with the condition. In March, the drug failed to improve attentiveness versus placebo among pediatric ADHD patients in another trial. — Kellen Owings