The results are in! Zynerba Pharmaceuticals released their top line results from their CONNECT-FX Trial on Zygel™, a CBD gel for Fragile X syndrome.

Linda Sorensen, NFXF Executive Director, remarks, “These top-line data show promise for Zygel in some individuals with Fragile X syndrome. We look forward to seeing the next steps taken by Zynerba, and continue to be thankful for their thoughtful partnership and dedication to bettering the lives of those living Fragile X.”

Dr. Randi Hagerman commented, “This study identified a key population of patients who might benefit from treatment of their behavioral symptoms of FXS with Zygel … Zygel has the potential to be an important therapeutic option for the most severely impacted patients with Fragile X.”

The study did not reach statistical significance in primary or secondary endpoints in the full analysis. It did achieve statistical significance in the primary endpoint in the ad-hoc analysis of participants with full methylation of the FMR1 gene (~80% of participants in the study).

This means that participants using Zygel did not see significant improvement (in the primary and secondary endpoints) compared to those on placebo. Researchers need to reach statistical significance in order to say they reached their endpoint. When Zynerba performed the pre-planned ad-hoc analysis, they did see a statistical significant improvement in individuals who have full methlyation of the FMR1 gene. This was a large group of the patients enrolled in the study, ~80%. Patients with genetically confirmed full mutation Fragile X and full methylation of their impacted FMR1 gene are generally the most severely impacted by the disorder. This group of patients in the analysis presented with higher anxiety, lower IQ, lower adaptive function, and more severe autism as compared to patients without a fully methylated FMR1 gene at the time of enrollment.

The next step for Zynerba is speaking with the FDA about these results as soon as possible.

Zynerba will be discussing these results in more detail on July 22 during our Fragile X Research Roundup of the NFXF International Fragile X Conference Virtual Series. Please visit our registration page if you haven’t already registered for the conference.

Thanks to all of you who participated in this pivotal trial! Thanks to our many NFXF Fragile X Clinic and Research Consortium (FXCRC) clinics who participated as well.

To learn more, read Zynerba’s press release, and listen to a replay of their Topline Results of Pivotal CONNECT-FX Trial in Fragile X Syndrome. (If you have any trouble with the replay, find their replays in their calendar here.)