DEVON, PA — Zynerba Pharmaceuticals, Inc. (NASDAQ: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, announced that the Company has completed enrollment in its 14-week pivotal CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial.
The trial is evaluating the efficacy and safety of Zygel™ CBD Gel as a treatment for behavioral symptoms of Fragile X syndrome (FXS) in 210 children ages three through 17 with full mutation FXS. Topline results are expected late in the second quarter of 2020.
“We are excited about the potential for Zygel, if approved by the FDA, to be the first drug ever indicated to treat the behavioral symptoms of Fragile X, and completion of enrollment in this trial is an important step toward that goal,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “I want to thank everyone who participated in this achievement, particularly the patients in the study, their families, the clinicians involved in CONNECT-FX and their staff. We look forward to announcing the topline results of this trial late in the second quarter of this year.”
The multi-national, randomized, double-blind, placebo-controlled, 14-week CONNECT-FX trial is assessing the efficacy and safety of Zygel for the treatment of children and adolescents with FXS.
Two hundred and ten (210) patients with Fragile X syndrome, confirmed with the full mutation of the FMR1 gene, have been randomized at 21 clinical sites in the United States, Australia, and New Zealand.
The trial protocol targeted enrollment of at least 204 patients. Patients have been randomized 1:1 to either trial drug or placebo.
The primary endpoint is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale.
Key secondary endpoints are the change from baseline to the end of the treatment period in the ABC-CFXS Irritability subscale score and the ABC-CFXS Socially Unresponsive/Lethargic subscale score, and improvement in Clinical Global Impression (CGI-I) anchored to behavioral symptoms of FXS evaluated at the end of the treatment period.
Consistent with recent guidance from the FDA on capturing the voice of the patient in drug development, additional qualitative data on the clinical relevance of various FXS behaviors to caregivers and patients is also being collected.
Baseline Characteristics of Patients Randomized into CONNECT-FX
As intended and prospectively designed, the trial has successfully enrolled a more severely affected population than the population that was enrolled in the previously completed Phase 2 FAB-C trial, as measured by baseline behavioral symptoms, enabling the study to potentially demonstrate the anticipated full range of efficacy of Zygel in several behavioral domains.
The ABC-CFXS mean baseline scores for the patients randomized in the CONNECT-FX trial in comparison to the FAB-C trial are as follows (higher baseline scores denote more severe behaviors):
- Social Avoidance subscale (primary endpoint): 7.2 in CONNECT-FX vs 5.1 in FAB-C;
- Irritability subscale (key secondary endpoint): 28.1 in CONNECT-FX vs 18.2 in FAB-C;
- Socially Unresponsive/Lethargic subscale (key secondary endpoint): 13.2 in CONNECT-FX vs 8.7 in FAB-C;
- Hyperactivity subscale: 18.4 in CONNECT-FX vs 14.5 in FAB-C;
- Stereotypy subscale: 9.4 in CONNECT-FX vs 7.9 in FAB-C; and
- Inappropriate Speech subscale: 6.9 in CONNECT-FX vs 6.1 in FAB-C.
During screening, the caregivers of patients in the trial were informed that their participating child may have the opportunity to receive Zygel in an open label extension trial following the child’s compliant completion of CONNECT-FX, regardless of their child’s perceived response or actual blinded drug assignment in CONNECT-FX. To date, 97% of the 158 patients who have completed CONNECT-FX have enrolled in the open label extension trial.
One hundred and fifty seven (157), or 75%, of the enrolled patients are male and the mean age at randomization in the study is 9.8 years.
The Company expects to announce topline results of this study late in the second quarter of 2020.
If the results are positive, the Company expects to meet with the U.S. Food and Drug Administration (FDA) to determine acceptability of the data as a basis to submit its New Drug Application (NDA) for Zygel in FXS in the second half of 2020, with potential approval by mid-year 2021.
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