Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for orphan neuropsychiatric disorders, today announces
the publication of a paper entitled, “
Role of the
Endocannabinoid System in Fragile X Syndrome: Potential Mechanisms
for Benefit From Cannabidiol Treatment,” in the Journal of
Neurodevelopmental Disorders. The review of evidence suggests a
central role for the endocannabinoid system (ECS) in neuronal
development and cognitive function and in the pathogenesis of
Fragile X syndrome (FXS), and the potential role of cannabidiol as
a treatment for FXS. The article can be accessed online at the
Journal of Neurodevelopmental Disorders at https://rdcu.be/c25fu.
“This publication describes the potential of Zygel as a
treatment for Fragile X syndrome based on the central role the
endocannabinoid system plays in neuronal development and function,”
said Armando Anido, Chairman and Chief Executive Officer of
Zynerba. “The proposed mechanisms of action of cannabidiol are
supported by the results from our Phase 3 CONNECT-FX trial in
patients with Fragile X syndrome with greater than or equal to 90%
methylation of the FMR1 gene, benefited from treatment with Zygel.
These findings bolster our confidence in the design of the ongoing,
confirmatory, pivotal Phase 3 RECONNECT trial.”
FXS is caused by deficiency or absence of the FMR1 protein,
FMRP. The absence of FMRP disrupts ECS signaling, which has been
implicated in FXS pathogenesis. The ECS helps maintain neuronal
function and signaling and is thought to be disrupted in FXS.
Cannabidiol may help restore the function of the ECS in FXS
according to a review of data to date. Cannabidiol may also act as
an agonist on serotonin 5HT1A and other receptors which may
contribute to beneficial effects in people with FXS. Consistent
with these proposed mechanisms of action of cannabidiol in FXS, in
the Phase 3 CONNECT-FX trial, Zygel was associated with
improvements in measures of social avoidance, irritability and
social interaction, particularly in patients showing ≥90%
methylation of the FMR1 gene. Results from the CONNECT-FX study of
Zygel for the treatment of behavioral symptoms in children and
adolescents with FXS can be accessed online at the Journal of
Neurodevelopmental
Disorders at https://rdcu.be/c0sKz.
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 and 22q11.2
deletion syndrome. Learn more at www.zynerba.com and follow us on
Twitter at @ZynerbaPharma.
Cautionary Note on Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,”
“might,” “will,” “should” or other words that convey uncertainty of
future events or outcomes to identify these forward-looking
statements. Such statements are subject to numerous important
factors, risks and uncertainties that may cause actual events or
results to differ materially from the Company’s current
expectations. Management’s expectations and, therefore, any
forward-looking statements in this press release could also be
affected by risks and uncertainties relating to a number of other
factors, including the following: the Company’s cash and cash
equivalents may not be sufficient to support its operating plan for
as long as anticipated; the Company’s expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
incentive and other tax credit eligibility, collectability and
timing, and availability of and the need for additional financing;
the Company’s ability to obtain additional funding to support its
clinical development programs; the results, cost and timing of the
Company’s clinical development programs, including any delays to
such clinical trials relating to enrollment or site initiation;
clinical results for the Company’s product candidates may not be
replicated or continue to occur in additional trials and may not
otherwise support further development in a specified indication or
at all; actions or advice of the U.S. Food and Drug Administration,
the European Medicines Agency and other foreign regulatory agencies
may affect the design, initiation, timing, continuation and/or
progress of clinical trials or result in the need for additional
clinical trials; the Company’s ability to obtain and maintain
regulatory approval for its product candidates, and the labeling
under any such approval; the Company’s reliance on third parties to
assist in conducting pre-clinical and clinical trials for its
product candidates; delays, interruptions or failures in the
manufacture and supply of the Company’s product candidates the
Company’s ability to commercialize its product candidates; the size
and growth potential of the markets for the Company’s product
candidates, and the Company’s ability to service those markets; the
Company’s ability to develop sales and marketing capabilities,
whether alone or with potential future collaborators; the rate and
degree of market acceptance of the Company’s product candidates;
the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates; the extent to which health epidemics and
other outbreaks of communicable diseases, including COVID-19, could
disrupt our operations or adversely affect our business and
financial conditions; and the extent to which inflation or global
instability, including political instability, may disrupt our
business operations or our financial condition. This list is not
exhaustive and these and other risks are described in the Company’s
periodic reports, including the annual report on Form 10-K,
quarterly reports on Form 10-Q and current reports on Form 8-K,
filed with or furnished to the Securities and Exchange Commission
and available at www.sec.gov. Any forward-looking statements that
the Company makes in this press release speak only as of the date
of this press release. The Company assumes no obligation to update
forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press
release.
Investor Contact
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
Media Contact:Andrea CohenSam Brown Healthcare
CommunicationsCell: 917.209.7163andreacohen@sambrown.com
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