Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) announced that Fragile X patients treated with Zygel showed a significant reduction in behavioral symptoms compared to those treated with a placebo in a recent Phase 3 trial.
Results from the Phase 3 CONNECT-FX study of Zygel for the treatment of behavioral symptoms in children and adolescents with Fragile X syndrome (FXS) were published in the Journal of Neurodevelopmental Disorders, the company said. Zygel, which contains cannabidiol, was previously granted orphan drug designation by the European Commission for the treatment of FXS.
The paper details how Zygel was well tolerated in patients with FXS and demonstrated efficacy with a favorable benefit-risk profile in patients with greater than 90% methylation of the FMR1 gene (in whom gene silencing is most likely, and the impact of FXS is typically most severe).
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“Children with Fragile X syndrome are dramatically impacted by debilitating behavioral and emotional challenges, including anxiety, social avoidance, irritability, inattention and aggression,” said Elizabeth Berry-Kravis, a professor in the department of pediatrics and neurological sciences at Rush University Medical Center.
“The results from CONNECT-FX offer hope for children with FXS and their caregivers as additional treatment options are needed for children who continue to struggle with this condition.”
CONNECT-FX was a randomized, double-blind, multinational 14-week study of 212 patients who were randomized to 12 weeks of Zygel or a placebo, as an add-on to standard of care. The primary endpoint assessed change in social avoidance measured by the aberrant behavior checklist–community edition FXS social avoidance subscale.
Although statistical significance for the primary endpoint was not achieved in the full cohort, the company noted, significant improvement was seen in patients with at least 90% methylation of FMR1. That group also achieved statistically significant improvements in caregiver global impression-change in social avoidance and isolation, irritable and disruptive behaviors and social interactions, the company said.
A follow-up phase 3 trial is already underway as well, the company said.
“CONNECT-FX provided evidence that a biologically identifiable and clinically responsive population of patients with FXS, who are defined by both full mutation and greater than or equal to 90% methylation of the FMR1 gene, benefited from treatment with Zygel,” Zynerba CEO Armando Anido.
“Given these findings, our follow-on pivotal Phase 3 trial, RECONNECT, is underway to confirm the results observed in this population. We believe having patients with complete methylation of their FMR1 gene as the primary analysis population improves our chance of success and we look forward to topline results from RECONNECT in the second half of 2023 and believe the results, if positive, will be sufficient to support the submission of a new drug application in the US and marketing authorization application in the EU for Zygel in patients with FXS.”
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