Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for orphan neuropsychiatric disorders, today announced
that the 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 paper titled, “A Randomized, Controlled Trial of
ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with
Fragile X Syndrome (CONNECT-FX),” details how Zygel was
well tolerated in patients with FXS and demonstrated efficacy with
a favorable benefit risk profile in patients with ≥90% methylation
of the FMR1 gene, in whom gene silencing is most likely, and the
impact of FXS is typically most severe. The article can be accessed
online at the Journal of Neurodevelopmental Disorders at
https://rdcu.be/c0sKz.
“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, M.D., Ph.D., Professor,
Department of Pediatrics and Neurological Sciences, 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 to evaluate the efficacy and safety of Zygel in
children and adolescents aged 3 to 17 years. A total of 212
patients were randomized to 12 weeks of Zygel (250 mg or 500 mg
daily [weight-based]) or placebo, as add-on to standard of care.
The trial was conducted at 21 investigational centers in the U.S.,
Australia and New Zealand. The primary endpoint assessed change in
social avoidance (SA) measured by the Aberrant Behavior
Checklist–Community Edition FXS (ABC-CFXS) SA subscale in the full
cohort of patients with full mutation FXS, regardless of the FMR1
gene methylation status. Ad hoc analyses assessed efficacy in
patients with ≥90% and 100% methylation of the promoter region of
the FMR1 gene, in whom FMR1 gene silencing is most likely.
Although statistical significance for the primary endpoint was
not achieved in the full cohort, significant improvement was
demonstrated in patients with ≥90% methylation of FMR1 (nominal
p=0.020), representing 80% of the overall study population. This
group also achieved statistically significant improvements in
Caregiver Global Impression-Change in SA and Isolation, Irritable
and Disruptive Behaviors, and Social Interactions (nominal
p-values: p=0.038, p=0.028, and p=0.002, respectively). Similar
results were seen in patients with 100% methylation of FMR1,
representing 65% of the study population. Zygel was generally well
tolerated. All treatment-emergent adverse events (TEAEs) were mild
or moderate. The most common treatment-related TEAE was application
site pain (Zygel: 6.4%; placebo: 1.0%).
“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,”
said Armando Anido, Chairman and Chief Executive Officer of
Zynerba. “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 U.S. and Marketing Authorization
Application in the EU for Zygel in patients with FXS.”
About Zygel
Zygel is the first and only pharmaceutically-manufactured
cannabidiol formulated as a patent-protected permeation-enhanced
clear gel, designed to provide controlled drug delivery into the
bloodstream transdermally (i.e., through the skin). Recent studies
suggest that cannabidiol may modulate the endocannabinoid system
and improve certain behavioral symptoms associated with
neuropsychiatric conditions. Zygel is an investigational drug
product in development for the potential treatment of behavioral
symptoms associated with Fragile X syndrome (FXS), 22q11.2 deletion
syndrome (22q) and autism spectrum disorder (ASD). The Company has
received orphan drug designation for cannabidiol, the active
ingredient in Zygel, from the FDA and the European Commission in
the treatment of FXS and the treatment of 22q. Additionally, Zygel
has been designated a Fast Track development program for treatment
of behavioral symptoms of FXS.
About Fragile X Syndrome (FXS)
Fragile X syndrome is a rare genetic developmental disability
that is the leading known cause of both inherited intellectual
disability and autism spectrum disorder, affecting 1 in 4,000 males
and 1 in 6,000 females. It is the most common inherited
intellectual disability in males and a significant cause of
intellectual disability in females, and the leading genetic cause
of autism spectrum disorder (ASD). The disorder negatively affects
synaptic function, plasticity and neuronal connections, and results
in a spectrum of intellectual disabilities and behavioral symptoms,
such as social avoidance and irritability. In the U.S., there are
about 78,000 people suffering with FXS, approximately 60% of whom
have complete methylation of the FMR1 gene.
FXS is caused by a mutation in FMR1, a gene which modulates a
number of systems, including important effects on the
endocannabinoid system, and most critically, codes for a protein
called FMRP. This protein helps regulate the production of other
proteins and plays a role in the development of synapses, which are
critical for relaying nerve impulses, and in regulating synaptic
plasticity. The FMR1 mutation manifests as multiple repeats of a
DNA segment, known as the CGG triplet repeat. In most neurotypical
people, the FMR1 gene correctly codes for the FMRP protein. As a
result, FMRP is produced at levels that enable control over
behaviors like social avoidance and anxiety. In people with full
mutation of the FMR1 gene, the CGG segment is repeated more than
200 times, and in most cases causes the gene to not function.
Methylation of the FMR1 gene also plays a role in determining
functionality of the gene. For patients with complete methylation,
no FMRP is produced. With no FMRP, the systems and processes that
are modulated by FMRP become dysregulated.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative
pharmaceutically-produced transdermal cannabinoid therapies for
orphan neuropsychiatric disorders. We are committed to improving
the lives of patients and their families living with severe,
chronic health conditions including Fragile X syndrome, 22q11.2
deletion syndrome and autism spectrum disorder. Learn more at
www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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Investor Contact
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
Media Contact
Laura MorganSam Brown Inc.Cell:
951.333.9110lauramorgan@sambrown.com
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