Alcobra's only drug fails mid-stage trial for genetic disorder

By Anjali Rao Koppala (Reuters) - The only drug in Alcobra Ltd's pipeline failed a mid-stage trial testing it against a type of genetic disorder that can cause autism and ADHD, sending the company's shares down as much as 19 percent on Wednesday. Israel-based Alcobra said the drug, MDX, aimed at treating Fragile X Syndrome (FXS), was not statistically different from a placebo in reducing symptom score on a rating scale for attention deficit hyperactivity disorder (ADHD). The company's stock plunged 28 percent before markets opened, but regained some ground to trade down 16 percent by Wednesday afternoon. The selloff represents an oversold opportunity for the stock and it is unlikely most investors ascribe significant value to Alcobra's FXS program as they are focusing on the company's planned ADHD studies, Piper Jaffray's Charles Duncan wrote in a research note. Alcobra is also testing MDX for ADHD in adults and children. The drug cleared a late-stage ADHD trial in October, but only after excluding data from patients who reacted positively to a placebo. The company said on Wednesday that MDX met the FXS study's secondary goals on two scales, VABS and KiTAPS, which assess a patient's behavior, cognition and attentiveness. Alcobra plans to discuss the results with the U.S. Food and Drug Administration and could suggest having VABS scores as the main goal of further studies, the company said on a conference call. Alcobra's failure follows in the footsteps of other drugmakers such as Novartis AG and Roche Holding AG, whose experimental FXS drugs have also failed key studies, leading the developers to abandon the programs. FXS affects about 50,000 Americans and can cause behavioral and learning challenges, according to the National Institutes of Health. No cure is currently available. Other drugmakers testing compounds to treat FXS include Marinus Pharmaceuticals and Australia's Neuren Pharmaceuticals Ltd. Both treatments are in mid-stage studies. (Reporting by Anjali Rao Koppala in Bengaluru; Editing by Don Sebastian and Simon Jennings)