Abstract Details

Title Cannabidiol Transdermal Gel for the Treatment of Fragile X Syndrome: Post hoc Responder Analysis and Pattern of Efficacy on domains of the Aberrant Behavior Checklist-FXS (ABC-CFXS)

Topic Child Neurology and Developmental Neurology

Presentation(s) P15 - Poster Session 15 (12:00 PM-1:00 PM)

Poster/Presentation
Number 5-002

Objective This study evaluated the pattern of clinical efficacy of open-label ZYN002, experimental cannabidiol (CBD) transdermal gel, in the treatment of Fragile X Syndrome (FXS).

Background FXS is a neurodevelopmental condition characterized by repeat expansion in the FMR1 gene on the X chromosome. Previously, we documented open-label treatment with ZYN002 was well-tolerated and associated with improvement in behavioral symptoms and anxiety in patients with FXS. An improvement of at least 25% in individual domains of the Aberrant Behavior Checklist-FXS (ABC-C_FXS) has been established as an important criterion in defining positive responder status in contemporary studies in FXS. The pattern of response to ZYN002 across multiple ABC-C_FXS domains may help to predict its possible future clinical utility.

Design/Methods This 12-week, phase 2, open-label study assessed ZYN002 in participants aged 6-17 years, with full FMR1 mutation, who showed suboptimal clinical response to standard of care. We explored efficacy by assessing change on all ABC-C_FXS domains (social avoidance, irritability, socially unresponsive/lethargic, hyperactivity, stereotypy, inappropriate speech), creating responder analyses and “radar charts” to visualize change in each ABC-C_FXS domain.

Results Of the 20 enrolled participants, 18 completed the study. Maximal 25% responder rates on all domains of ABC-C_FXS on any visit ranged from 77.8 – 83%. Maximal 50% responder rates on all domains of ABC-C_FXS on any visit ranged from 55.6 – 77.8%. Baseline to endpoint “radar charts” showing all ABC-C_FXS domains, with each domain independently represented its own normalized single axis, provided visual evidence of meaningful change

Conclusions The majority of FXS patients treated with open-label ZYN002 met an important criterion for response on the ABC-C_FXS. Simultaneous visualization of change across all measured domains of the ABC-C_FXS (“radar charts”) provided evidence of more global, multi-domain reduction in behavioral symptom burden, and may provide evidence to support the efficacy of ZYN002 CBD transdermal gel in FXS.

Authors/Disclosures
Joseph M. Palumbo, MD (BioVie) PRESENTER	Dr. Palumbo has received personal compensation for serving as an employee of BioVie. An immediate family member of Dr. Palumbo has received personal compensation for serving as an employee of Merck Research Laboratories. Dr. Palumbo has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Stalicla SA. Dr. Palumbo has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Psychae Therapeutics Pty Ltd. Dr. Palumbo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Phocus Pharmaceuticals Inc.. Dr. Palumbo has received personal compensation in the range of $0-$499 for serving as an officer or member of the Board of Directors for BioVie. Dr. Palumbo has or had stock in BioVie. An immediate family member of Dr. Palumbo has or had stock in Merck Research Laboratories. The institution of Dr. Palumbo has received research support from United States Department of Defense. Dr. Palumbo has received intellectual property interests from a discovery or technology relating to health care. Dr. Palumbo has received intellectual property interests from a discovery or technology relating to health care. Dr. Palumbo has received intellectual property interests from a discovery or technology relating to health care.
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file

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Abstract Details