Zynerba Pharmaceuticals (NASDAQ:ZYNE) reported positive topline data from its Phase 2 trial of Zygel for the treatment of autism spectrum disorder (ASD).
Zygel is a cannabidiol-based gel that provides controlled drug delivery into the bloodstream through the skin.
The trial, known as BRIGHT, enrolled 37 patients who received weight-based doses of 250 mg or 500 mg daily of Zygel, for 14 weeks. All five subscales of the Aberrant Behavior Checklist showed statistically significant improvements at 14 weeks of treatment from baseline.
“I am very impressed with the improvements my patients made over the 14-week treatment period while receiving Zygel; the reduction in irritability, communication deficits, and repetitive movements were especially noteworthy since some of these are core autistic behaviors,” Helen Heussler, the study’s principal investigator, said in a statement.
“The magnitude of effect on autistic behaviors in this trial is significant, including hyperactivity and stereotypy, which are among the most difficult behaviors to improve with therapeutic intervention,” she added.