Zynerba Pharmaceuticals Presents Data Supporting FMR1 Methylation Status as a Correlate to Fragile X Syndrome Severity at the Virtual Joint 16th International Child Neurology Congress (ICNC) & 49th Annual Child Neurology Society (CNS) Meeting - Seite 2

Zynerba utilized psychometric analyses to determine what constitutes a clinically meaningful change from baseline as measured by subscales of the ABC-C_FXS. New results described today include the results of these analyses which support the definition of a clinically meaningful treatment response over 12 weeks of treatment as an improvement of three points or greater for the Social Avoidance subscale, nine points or greater for the Irritability subscale, and five points or greater for the Socially Unresponsive / Lethargic subscale. The Company determined that 58.2% of FMet patients receiving Zygel achieved a clinically meaningful change in their socially avoidant behavior compared to 40.6% of patients receiving placebo (statistically significant; p=0.031), and 40.3% of patients receiving Zygel achieved a clinically meaningful change in Irritability compared to 23.8% of patients receiving placebo (statistically significant; p=0.036).

Figure 1: Greater Percentages of Participants Achieved Meaningful Change in ABC-C_FXS Social Avoidance and Irritability with ZYN002 vs Placebo

https://www.globenewswire.com/NewsRoom/AttachmentNg/d8cf2270-883e-4618 ...

The authors of the poster concluded that:

• To our knowledge, CONNECT-FX is the largest controlled study ever performed in FXS.
• These results may represent an important step forward in biomarker-driven prediction of response in FXS and neuroscience.
  • Zygel was well tolerated, and the safety profile was consistent with previously reported clinical trials.
  • In the FMet group, Zygel was superior to placebo in multiple analyses, including:
    • Statistically significant mean change in Social Avoidance vs placebo;
    • Proportion of patients attaining threshold of clinically meaningful change in Social Avoidance and Irritability;
    • Caregiver reported improvements, including statistically significant improvements in Social Avoidance and Isolation, Social Interaction, and Irritable and Disruptive Behaviors.
• Zynerba will be meeting with the FDA in the fourth quarter of 2020 to discuss these data.

About Zynerba Pharmaceuticals, Inc.