Anavex Life Sciences (AVXL) on Tuesday said its treatment had a positive effect on patients with Rett syndrome. However, AVXL stock dropped after an inaccurate impression. Anavex’s trial is a Phase 2b study, comparing the experimental drug to placebo in patients with ALS.
It’s designed to assess the effectiveness of Anavex 2-73 as a symptomatic treatment of ALS, not a disease-modifying one that would treat the underlying molecular defect. The study found that, over the course of one year, patients who received the drug made significantly greater improvements in their motor skills and daily living abilities than those receiving a placebo. Most Rett syndrome patients are diagnosed between ages 1 and 4.
The disorder causes severe cognitive impairment, delayed development, seizures, breathing problems, and an inability to speak. Those results, while not statistically significant, show that the drug “might have a benefit,” said Dr. Roger Rosenberg, chief of geriatrics at North Shore-LIJ Health System in New Hyde Park, N.Y., who was not involved in the study. ANAVEX 2-73, is one of a growing number of experimental compounds being developed to improve the lives of people who have a disease that strikes children.