On 30 June 2020 Zynerba Pharmaceuticals announced the top line results from the CONNECT-FX Trial of Zygel (CBD gel) in Fragile X syndrome. The study enrolled 245 participants with Fragile X syndrome at clinical sites in Australia, NZ and the US in a double blind randomized trial, following an earlier study in Australia.

Zygel did not achieve results with statistical significance in the major endpoint assessment criteria being measured compared to the participants on placebo. This means that participants using Zygel did not see significant improvement (in the primary and secondary endpoints) compared to those on placebo. Zygel did however achieve positive results of statistical significance for a large group of the study participants in improvement on a social avoidance scale measured in the study. This group included about 80% of the participants who had what was classified as a fully methylated full mutation.

Zynerba has stated that it intends to meet with the Federal Drug Administration in the US to discuss the outcome of the study on the basis that “The results from CONNECT-FX identified a significant patient population who responded well to Zygel and may provide […] a pathway towards licensure” of Zygel.

More will be known once Zynerba meets with the FDA in the coming weeks.

Zynerba will be discussing these results in more detail at the Fragile X Research Roundup event of the NFXF International Fragile X Conference Virtual Series on July 22.
Media release here:   https://zynerba.com/zynerba-pharmaceuticals-announces-top-…/