Zynerba shares up on rare disease trial success

Dive Brief:

  • Zynerba Pharmaceuticals’ shares shot up by 52.5% after news on Thursday that its lead drug, ZYN002 cannabidiol gel met its primary endpoint in its FAB-C Phase 2 trial in children and adolescents with Fragile X syndrome.
  • In the open label study of the orphan drug, patients had a 46% improvement from baseline on the Anxiety, Depression and Mood Scale (p0.0001), and clinically meaningful improvements in all measures of the Aberrant Behavior Checklist.
  • The next step will be a meeting with the Food and Drug Administration in the first half of 2018, and a Phase 2/3 trial in pediatric and adolescent patients by the end of next year.

Dive Insight:

“These data are extremely promising, particularly the improvements in anxiety, social avoidance and irritability as measured by scales including ADAMS, ABC-FXS, and PARS-R. Tolerability is essential in these patients, so I’m very pleased to see that ZYN002 was well tolerated

... read more at: http://www.biopharmadive.com/news/zynerba-shares-up-on-rare-disease-trial-success/506093/